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KEYTRUDA® (pembrolizumab) in Combination With Chemotherapy
Merck Receives Positive EU CHMP Opinion for KEYTRUDA® (pembrolizumab) in Combination With Chemotherapy for Certain Patients With Locally Recurrent Unresectable or Metastatic Triple-Negative Breast Cancer Whose Tumors Express PD-L1 (CPS ≥10)
September 17, 2021 7:15 am ET
Recommendation Based on Data From Phase 3 KEYNOTE-355 Trial
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended approval of KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with chemotherapy for the treatment of locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC) in adults whose tumors express PD-L1 (Combined Positive Score [CPS] ≥10) and who have not received prior chemotherapy for metastatic disease.
The positive opinion is based on progression-free survival (PFS) and overall survival (OS) results from the Phase 3 KEYNOTE-355 trial, which showed that treatment with KEYTRUDA in combination with chemotherapy (nab-paclitaxel, paclitaxel or gemcitabine/carboplatin), as compared to chemotherapy alone, significantly improved PFS and OS in these patients. Overall survival data from KEYNOTE-355 will be presented at the European Society for Medical Oncology (ESMO) Congress 2021 on Sept. 19. The CHMP’s recommendation will now be reviewed by the European Commission for marketing authorization in the European Union.
“Triple-negative breast cancer grows and spreads faster than other types of breast cancer and consequently has a worse prognosis,” said Dr. Vicki Goodman, vice president, clinical research, Merck Research Laboratories. “This positive CHMP opinion is an important step forward in bringing a new immunotherapy treatment option with KEYTRUDA to appropriate patients in Europe with metastatic triple-negative breast cancer. Importantly, this treatment regimen can be used in combination with different chemotherapy agents. We look forward to the European Commission’s decision in the coming months.”
Merck is rapidly advancing a broad portfolio in gynecologic and breast cancers through an extensive clinical development program for KEYTRUDA and several other investigational and approved medicines across these areas.
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,600 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS ≥1)] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):
- who are not eligible for any platinum-containing chemotherapy, or
- who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
For more information, visit www.merck.com
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
Merck wins positive opinion in EU for KEYTRUDA plus chemotherapy for breast cancer
Sep. 17, 2021 7:35 AM ETMerck & Co., Inc. (MRK)By: Dulan Lokuwithana, SA News Editor3 Comments
- Merck (NYSE:MRK) announced that European Medicines Agency (EMA) recommended its anti-PD-1 therapy KEYTRUDA in combination with chemotherapy for a certain group of patients with locally recurrent unresectable or metastatic triple-negative breast cancer (TNBC).
- https://seekingalpha.com/news/3740757-merck-wins-positive-opinion-in-eu-for-keytruda-plus-chemotherapy-for-breast-cancer
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
GAVRETO® (pralsetinib)
Roche receives positive CHMP opinion for Gavreto® (pralsetinib) for the treatment of adults with RET fusion-positive advanced non-small cell lung cancer
- Gavreto showed robust and durable clinical responses in people with NSCLC with RET fusions
- If approved, Gavreto will be the first and only targeted treatment approved by the EMA that includes first-line treatment of people with RET fusion-positive advanced NSCLC
Basel, 17 September 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended the approval of Gavreto® (pralsetinib) as a monotherapy for the treatment of adult patients with rearranged during transfection (RET) fusion-positive advanced non-small cell lung cancer (NSCLC) not previously treated with a RET inhibitor.
“This positive CHMP opinion for Gavreto represents another important step towards our goal of providing effective therapeutics that target genomic drivers of disease for as many cancer patients as possible," said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “Advances in personalised medicine also underscore the importance of tumour genomic profiling to identify patients who may benefit from targeted therapies.”
RET alterations are key disease drivers in many cancer types, including NSCLC and multiple types of thyroid cancer. RET fusion-positive NSCLC affects approximately 37,500 people worldwide each year and the disease often affects those who least expect it;[1,2,3] RET fusion-positive NSCLC is often identified in younger people with a minimal to no history of smoking.[3] These cancers also typically represent a high unmet need, due to limitations associated with standard therapies.[4,5,6] Biomarker testing for these fusions is the most effective way to identify people with advanced NSCLC who are eligible for treatment with Gavreto. Gavreto is a highly selective, potent, and CNS-penetrant RET inhibitor and, together with Alecensa® (alectinib) and Rozlytrek® (entrectinib), is part of Roche’s portfolio of targeted treatments for NSCLC. Together, they offer personalised treatment options for almost one in ten people with advanced NSCLC.[7]
The CHMP recommendation is based on the results of the phase I/II ARROW study, in which Gavreto demonstrated rapid, potent, and durable clinical activity in patients with advanced RET fusion-positive NSCLC.[8] A final decision regarding the approval of Gavreto is expected from the European Commission in the coming months.
Gavreto has also shown activity across multiple solid tumour types, reflecting tumour-agnostic potential.[9] In September 2020, the U.S. Food and Drug Administration (FDA) approved Gavreto for the treatment of adults with metastatic RET fusion-positive NSCLC, and in December 2020 it was approved for the treatment of adult and paediatric patients 12 years of age and older with advanced RET-altered thyroid cancers. Gavreto has since been approved in Canada, mainland China and Switzerland. In the European Union, the MAA for Gavreto for the treatment of adults with RET fusion-positive NSCLC is ongoing, and a submission for RET-altered thyroid cancers is planned. Regulatory submissions for these indications are underway in multiple countries worldwide.
Blueprint Medicines and Roche are co-developing Gavreto globally, with the exception of certain territories in Asia, including China.* Blueprint Medicines and Genentech, a wholly owned member of the Roche Group, are commercialising Gavreto in the US and Roche has exclusive commercialisation rights for Gavreto outside of the US, with the exception of certain territories in Asia, including China.*
*CStone Pharmaceuticals retains all rights to the development and commercialisation of Gavreto in these territories under its existing collaboration with Blueprint Medicines.
About the ARROW study[10]
ARROW is an ongoing phase I/II, open-label, first-in-human study designed to evaluate the safety, tolerability and efficacy of Gavreto, administered orally in people with rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC), RET-mutant medullary thyroid cancer, RET fusion-positive thyroid cancer and other RET-altered solid tumours. ARROW is being conducted at multiple sites across the United States, Europe and Asia.
An update from the ARROW study was presented at the American Society of Clinical Oncology (ASCO) 2021 Virtual Scientific Programme, 4-8 June.[11] In 126 patients with RET fusion-positive NSCLC who previously received platinum-based chemotherapy, Gavreto demonstrated an overall response rate (ORR) of 62% (95% CI: 53%, 70%), a clinical benefit rate (CBR) of 74% (95% CI: 65%, 81%), and a disease control rate (DCR) of 91% (95% CI: 85%, 96%). Median progression-free survival (PFS) was 16.5 months (95% CI: 10.5 months, 24.1 months). In 68 treatment-naïve patients, the confirmed ORR was 79% (95% CI: 68%, 88%), the CBR was 82% (95% CI: 71%, 91%), and the DCR was 93% (95% CI: 84%, 98%). Median PFS was 13.0 months (95% CI: 9.1 months, not reached (NR)). In 25 treatment-naïve patients who were enrolled after eligibility criteria were revised, to allow candidates for platinum-based therapy, the confirmed ORR was 88% (95% CI: 69%, 98%), the CBR was 88% (95% CI: 69%, 98%), and the DCR was 96% (95% CI: 80%, 100%). Median PFS was not reached. In addition, Gavreto was well-tolerated; of the 471 ARROW trial patients across RET-altered tumour types, the most common (≥ 25%) treatment-related adverse events were neutropenia, increased liver enzymes (aspartate aminotransferase [AST] and alanine aminotransferase [ALT]), anaemia, white blood cell count decrease, high blood pressure (hypertension) and lack of energy (asthenia).
About Gavreto® (pralsetinib)
Gavreto is a once-daily, oral targeted treatment designed to selectively target rearranged during transfection (RET) alterations, including fusions and mutations, regardless of the tissue of origin. Preclinical data have shown that Gavreto inhibits primary RET fusions and mutations that cause cancer in subsets of patients, as well as secondary RET mutations predicted to drive resistance to treatment. Blueprint Medicines and Roche are co-developing Gavreto for the treatment of people with various types of RET-altered cancers.
The Roche Group, headquartered in Basel, Switzerland, is active in over 100 countries and in 2020 employed more than 100,000 people worldwide. In 2020, Roche invested CHF 12.2 billion in R&D and posted sales of CHF 58.3 billion. Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan. For more information, please visit www.roche.com.
All trademarks used or mentioned in this release are protected by law.
Blueprint Medicines, Gavreto and associated logos are trademarks of Blueprint Medicines Corporation.
Roche receives positive CHMP opinion for Gavreto for non-small cell lung cancer
Sep. 17, 2021 8:00 AM ET Roche Holding AG (RHHBY)
By: Jonathan M Block, SA News Editor
- The European Medicine Agency's Committee for Medicinal Products for Human Use ("CHMP") has issued a positive opinion on Roche's (OTCQX:RHHBY) Gavreto (pralsetinib) as a monotherapy for non-small cell lung cancer ("NSCLC").
- https://seekingalpha.com/news/3740769-roche-receives-positive-chmp-opinion-for-gavreto-for-non-small-cell-lung-cancer
- https://seekingalpha.com/symbol/RHHBY
- https://www.roche.com/
- https://www.gavreto.com/index.html
- https://seekingalpha.com/symbol/BPMC
- https://www.blueprintmedicines.com/
- https://www.blueprintmedicines.com/medicines/
PIQRAY® (alpelisib)
Novartis announces findings from a real-world study of alpelisib demonstrating clinical benefit in people with PIK3CA-Related Overgrowth Spectrum (PROS)
Sep 17, 2021
- PROS is a spectrum of rare disorders caused by PIK3CA mutations and is characterized by atypical, visible overgrowths and anomalies in blood vessels, the lymphatic system and other tissues
- At 24 weeks, 38% of patients achieved ≥20% reduction in the volume of the PROS lesions assessed in the primary endpoint analysis; no patients experienced disease progression or death
- Alpelisib is the first potential treatment to specifically address the root cause of PROS conditions
- EPIK-P1 study findings presented at ESMO Virtual Congress 2021 support FDA submission
Basel, September 17, 2021— Novartis today announced important findings from a real-world study evaluating the safety and efficacy of alpelisib for people living with PIK3CA-Related Overgrowth Spectrum (PROS) who received treatment daily for at least 24 weeks. Results from EPIK-P1 showed alpelisib effectively reduced volume of clinically significant PROS-related lesions and improved signs and symptoms in pediatric and adult patients. Results were presented at the European Society of Medical Oncology (ESMO) Virtual Congress 2021 [LBA23].
“There are few options available to manage PROS conditions, and they are mainly focused on addressing worsening symptoms. It is devastating for patients to be without treatments that address the underlying cause of PROS,” said Guillaume Canaud, MD, PhD, Necker-Enfants Malades Hospital – AP-HP, the Paris Descartes University, Inserm (INEM Institute Necker Enfants Malades – Centre for Molecular Medicine). “The EPIK-P1 findings show robust clinical benefit for adult and pediatric patients and a potential new path forward for those impacted by PROS conditions.”
In EPIK-P1, alpelisib reduced target lesion volume and improved PROS-related symptoms and manifestations. The primary endpoint analysis conducted at week 24 in patients with complete cases (n=32) showed 38% of patients achieving a response to treatment which was defined as 20% or greater reduction in the sum of PROS target lesion volume. Nearly three in four patients (74%) showed some reduction in target lesion volume, with a mean reduction of 13.7%, and no patients experienced disease progression at time of primary analysis.
About EPIK-P1
EPIK-P1 is a global, site-based retrospective non-interventional medical chart review of pediatric and adult male and female patients aged 2 years or older with PIK3CA-Related Overgrowth Spectrum (PROS) who received alpelisib via a compassionate use program. Primary endpoint is proportion of patients with response at 24 weeks, defined as achieving at least 20% reduction from index date in the sum of measurable target lesion volume via central imaging. Data were obtained from medical charts of 57 patients (39 pediatric, 18 adult) at seven sites in five countries. There were 32 complete cases, meaning there were complete scans for comparison at baseline and date of analysis, and one patient discontinued prior to week 24 due to lack of clinical efficacy.
Alpelisib is not approved by any regulatory authority for the treatment of PROS conditions.
Find out more at https://www.novartis.com.
Novartis' alpelisib shows robust clinical benefit in people with PROS conditions
Sep. 17, 2021 8:31 AM ETNovartis AG (NVS)By: Mamta Mayani, SA News Editor
- Novartis (NYSE:NVS) announces findings from a real-world study evaluating the safety and efficacy of alpelisib in people with PIK3CA-Related Overgrowth Spectrum (PROS) who received treatment daily for at least 24 weeks.
- https://seekingalpha.com/news/3740790-novartis-alpelisib-shows-robust-clinical-benefit-in-people-with-pros-conditions
- https://seekingalpha.com/symbol/NVS
- https://www.hcp.novartis.com/products/piqray/metastatic-breast-cancer/
- https://www.novartis.com/
VUMERITY® (diroximel fumarate)
CHMP Recommends VUMERITY® (diroximel fumarate) for Approval in the European Union as a Treatment for Relapsing-Remitting Multiple Sclerosis
SEPTEMBER 17, 2021 • INVESTOR RELATIONS
- VUMERITY is a next-generation oral fumarate with a well-characterized efficacy and safety profile
- Data from the Phase 3 EVOLVE-MS-2 study have demonstrated that treatment with VUMERITY results in low discontinuation rates due to its gastrointestinal (GI) tolerability profile
- Upon approval, VUMERITY will offer a new oral option for MS patients as they consider treatment initiation in the context of the COVID-19 environment
CAMBRIDGE, Mass., Sept. 17, 2021 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) today announced that the Committee for Medicinal Products for Human Use (CHMP), part of the European Medicines Agency (EMA), issued a positive opinion and has recommended granting marketing authorization for VUMERITY® (diroximel fumarate) in the European Union (EU). VUMERITY is a next-generation oral fumarate for the treatment of adults with relapsing-remitting multiple sclerosis (RRMS). An estimated 2.8 million people live with MS across the globe, with some European countries demonstrating the highest prevalence of MS in the world.1
“With MS, finding the right treatment option is as much about managing the clinical aspects of the disease as it is about how treatment fits into a person’s life,” said Simon Faissner, M.D., PhD, Assistant Professor at the Department of Neurology, Ruhr-University Bochum. “Today’s CHMP opinion is a crucial step forward in providing an oral therapeutic option that is easy to integrate into a patient’s daily life, which helps with ongoing care management.”
The CHMP’s positive opinion will now be referred to the European Commission (EC), which grants marketing authorizations for medicines in Europe.
The positive CHMP opinion was based on data from pharmacokinetic bridging studies comparing VUMERITY and TECFIDERA® (dimethyl fumarate) to establish bioequivalent exposure of monomethyl fumarate, the active metabolite, and relied in part on the well-established long-term safety and efficacy profile of TECFIDERA. The CHMP also assessed findings from EVOLVE-MS-2, a large, randomized, double-blind, five-week, multi-center Phase 3 study to evaluate the gastrointestinal (GI) tolerability of VUMERITY compared to TECFIDERA in patients with RRMS. In EVOLVE-MS-2, the rate of overall treatment discontinuation was lower in participants treated with VUMERITY compared to those treated with TECFIDERA (1.6% compared to 6%, respectively). The difference in the discontinuation rates due to GI tolerability was 0.8% for VUMERITY compared to 4.8% for TECFIDERA.
VUMERITY was first approved by the U.S. Food and Drug Administration in October 2019 and is currently the number one prescribed oral MS therapy in the country. Since its launch in the U.S., real-world data have reinforced the positive GI tolerability profile of VUMERITY and confirmed that the experience demonstrated in clinical trials is consistent with clinical practice.2 Biogen continues to file regulatory submissions in other countries.
About VUMERITY® (diroximel fumarate)
VUMERITY is an oral fumarate with a distinct chemical structure from TECFIDERA® (dimethyl fumarate), approved in the U.S. for the treatment of relapsing forms of multiple sclerosis in adults, to include clinically isolated syndrome, relapsing-remitting disease and active secondary progressive disease. Once in the body, VUMERITY rapidly converts to monomethyl fumarate, the same active metabolite of dimethyl fumarate providing similar efficacy and safety profiles.
VUMERITY is contraindicated in patients with known hypersensitivity to diroximel fumarate, dimethyl fumarate or to any of the excipients of VUMERITY; and in patients taking dimethyl fumarate. Serious side effects for VUMERITY are based on data from dimethyl fumarate (which has the same active metabolite as VUMERITY) and include anaphylaxis and angioedema, progressive multifocal leukoencephalopathy, which is a rare opportunistic viral infection of the brain that has been associated with death or severe disability, a decrease in mean lymphocyte counts during the first year of treatment, herpes zoster and other serious infections, liver injury and flushing. The most common adverse events, obtained using data from dimethyl fumarate (which has the same active metabolite as VUMERITY), were flushing, abdominal pain, diarrhea and nausea.
Please click here for Important Safety Information and full Prescribing Information, including Patient Information for VUMERITY in the U.S.
About TECFIDERA® (dimethyl fumarate)
TECFIDERA, a treatment for relapsing forms of multiple sclerosis (MS) in adults, is the most prescribed oral medication for relapsing MS in the world and has been shown to reduce the rate of MS relapses, slow the progression of disability and impact the number of MS brain lesions, while demonstrating a well-characterized safety profile in people with relapsing forms of MS. TECFIDERA is approved in 69 countries, and more than 500,000 patients have been treated with it, representing more than 1,000,000 patient-years of exposure across clinical trial use and patients prescribed TECFIDERA. Of these, 6,335 patients (14,241 patient-years) were from clinical trials.3
TECFIDERA is contraindicated in patients with a known hypersensitivity to dimethyl fumarate or any of the excipients of TECFIDERA. Serious side effects include anaphylaxis and angioedema, and cases of progressive multifocal leukoencephalopathy, a rare opportunistic viral infection of the brain which has been associated with death or severe disability, have been seen with TECFIDERA patients in the setting of lymphopenia. Other serious side effects include a decrease in mean lymphocyte counts during the first year of treatment, herpes zoster and other serious infections, liver injury and flushing. In clinical trials, the most common adverse events associated with TECFIDERA were flushing, abdominal pain, diarrhea and nausea.
For information on TECFIDERA prescribing information in the EU, please visit: https://www.ema.europa.eu/en/medicines/human/EPAR/tecfidera. Please click here for Important Safety Information and full Prescribing Information, including Patient Information for TECFIDERA in the U.S., or visit your respective country’s product website.
https://www.biogenoptions.com/en_us/home/treatment-overview/tecfidera.html
We routinely post information that may be important to investors on our website at www.biogen.com.
Biogen gets CHMP positive recommendation for multiple sclerosis treatment Vumerity
Sep. 17, 2021 9:28 AM ETBiogen Inc. (BIIB)By: Aakash Babu, SA News Editor1 Comment
- Biogen (NASDAQ:BIIB) announces that the Committee for Medicinal Products for Human Use (CHMP) of the EMA has issued a positive opinion for the approval of Vumerity (diroximel fumarate) in the EU.
- https://seekingalpha.com/news/3740837-biogen-gets-chmp-positive-recommendation-for-multiple-sclerosis-treatment-vumerity
- https://seekingalpha.com/symbol/BIIB
- https://www.vumerity.com/
Nucala (mepolizumab)
17 September 2021
GSK receives CHMP positive opinions recommending approval of Nucala (mepolizumab) in three additional eosinophil-driven diseases
For media and investors only
Issued: 17 September 2021, London UK
- If approved in Europe, mepolizumab would be the only treatment indicated for use in four eosinophil-driven diseases
- CHMP positive opinions advance efforts to provide the first targeted treatment for eosinophilic granulomatosis with polyangiitis (EGPA) and the first anti-IL-5 biologic treatment for patients with hypereosinophilic syndrome (HES) or chronic rhinosinusitis with nasal polyps (CRSwNP)
GlaxoSmithKline (GSK) plc today announced that the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued positive opinions recommending Nucala (mepolizumab), a monoclonal antibody that targets interleukin-5 (IL-5), for use in three eosinophil-driven diseases; hypereosinophilic syndrome (HES), eosinophilic granulomatosis with polyangiitis (EGPA) and chronic rhinosinusitis with nasal polyps (CRSwNP). The CHMP opinion is one of the final steps in the marketing authorisation procedure prior to approval decision by the European Commission.
The three positive opinions are based on data from pivotal studies investigating the role of targeted IL-5 inhibition with mepolizumab in these eosinophil-driven diseases. Eosinophil-driven diseases are inflammatory conditions associated with elevated levels of eosinophils, a type of white blood cell.
Mepolizumab is already approved for use in Europe as an add-on treatment for patients with severe eosinophilic asthma. Epidemiological, clinical, and pathophysiological studies strongly suggest that CRSwNP and asthma are closely linked and often coexist. Additionally, patients with EGPA frequently have severe asthma. This overlap across eosinophil-driven diseases underscores the importance of understanding the complex role of eosinophils in disease.
Mepolizumab has been studied in over 4,000 patients in a total of 41 clinical trials evaluating the role mepolizumab may play in targeting the underlying cause of inflammation and reducing eosinophils through IL-5 inhibition. Through ongoing research, GSK is committed to improving the lives of those living with disease associated with uncontrolled eosinophilic inflammation, continuously innovating in order to address the unmet needs in this broad patient group.
About mepolizumab
First approved in 2015 for severe eosinophilic asthma (SEA), mepolizumab is the first-in-class monoclonal antibody that targets IL-5. It is believed to work by preventing IL-5 from binding to its receptor on the surface of eosinophils, reducing blood eosinophils and maintaining them within normal levels. A normal level of blood eosinophils being less than 500 eosinophils/microlitre. The mechanism of action for mepolizumab has not been definitively established.
Mepolizumab has been developed for the treatment of diseases that are driven by inflammation caused by eosinophils. It has been studied in over 4,000 patients in 41 clinical trials across a number of eosinophilic indications and has been approved under the brand name Nucala in the US, Europe and in over 25 other markets, as an add-on maintenance treatment for patients with SEA. Mepolizumab is approved in 17 markets, including Europe and the US, for paediatric use in SEA from ages 6-17 years, with approval in an additional 7 markets for use in patients with SEA aged 12-17 years. The first approval for mepolizumab in CRSwNP was granted by the FDA in July 2021. In a total of 13 markets including the US, Japan and Canada it is approved for use in adult patients with EGPA. Mepolizumab was approved for use in HES in the US in September 2020, followed by Brazil in February 2021, Argentina in May 2021 and Canada in September 2021. Mepolizumab is currently being investigated in COPD. It is not currently approved for use in COPD anywhere in the world.
For further information please visit www.gsk.com/about-us.
European advisory group backs GlaxoSmithKline's Nucala in three eosinophil-driven diseases
Sep. 17, 2021 8:56 AM ETGlaxoSmithKline plc (GSK)
By: Mamta Mayani, SA News Editor
GlaxoSmithKline (NYSE:GSK) announces that the EMA's Committee for Medicinal Products for Human Use (CHMP) has issued positive opinions recommending Nucala (mepolizumab) for use in three eosinophil-driven diseases; hypereosinophilic syndrome (HES), eosinophilic granulomatosis with polyangiitis (EGPA) and chronic rhinosinusitis with nasal polyps (CRSwNP).
- https://seekingalpha.com/news/3740811-european-advisory-group-backs-glaxosmithklines-nucala-in-three-eosinophil-driven-diseases
- https://seekingalpha.com/symbol/GSK
- https://www.nucala.com/
JYSELECA® (FILGOTINIB)
GALAPAGOS ANNOUNCES POSITIVE CHMP OPINION FOR JYSELECA® (FILGOTINIB) FOR THE TREATMENT OF ADULTS WITH MODERATELY TO SEVERELY ACTIVE ULCERATIVE COLITIS
Mechelen, Belgium; 17 September 2021; 13.15 CET; Galapagos NV (Euronext & Nasdaq: GLPG) announced today that the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Jyseleca® (filgotinib), a once-daily, oral, JAK1 preferential inhibitor for the treatment of adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent. Following this positive opinion, a final decision from the European Commission is expected later this year.
The CHMP positive opinion is based on data from the pivotal Phase 2b/3 SELECTION program, which evaluated filgotinib as an induction and maintenance therapy in adult patients with moderately to severely active UC who have failed conventional therapy or biologics. SELECTION comprised two placebo-controlled induction studies, one in biologic-naive patients and the other in biologic-experienced patients, followed by a 47-week maintenance study for those who responded to filgotinib after 10 weeks. Responders to placebo continued on blinded placebo during the maintenance phase. The trial was recently published in The Lancet1.
Dr Walid Abi-Saab, Chief Medical Officer at Galapagos, said: “Ulcerative colitis can have significant and profound effects on the people who suffer with the condition. Persistent inflammation and uncontrolled disease mean patients may experience debilitating relapses, may need increasing doses of steroids and in some instances may require surgery, which impacts them not only physically, but also psychologically. Today’s decision brings us one step closer to providing a new treatment option for people living with this chronic disease.”
The CHMP positive opinion will now be reviewed by the European Commission and a decision is expected before year end 2021. This positive opinion follows the previous approval of filgotinib for the treatment of patients with moderate to severe active rheumatoid arthritis. The use of filgotinib for UC is investigational and is not approved anywhere globally.
About filgotinib
Filgotinib is approved and marketed as Jyseleca (200mg and 100mg tablets) in the European Union, Great Britain, and Japan for the treatment of adults with moderate to severe active rheumatoid arthritis (RA) who have responded inadequately or are intolerant to one or more disease modifying anti-rheumatic drugs (DMARDs). Filgotinib may be used as monotherapy or in combination with methotrexate (MTX). The European Summary of Product Characteristics for filgotinib, which includes contraindications and special warnings and precautions, is available at www.ema.europa.eu. The interview form from the Japanese Ministry of Health, Labour and Welfare is available at www.info.pmda.go.jp. The individual Great Britain and Northern Ireland Summary of Product Characteristics can be found at www.medicines.org.uk/emc and www.emcmedicines.com/en-GB/northernireland, respectively. Applications have been submitted to the European Medicines Agency (EMA), the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), and Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) for the treatment of adults with moderately to severely active ulcerative colitis (UC) who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a biologic agent and are currently under review. Filgotinib is not approved in any other countries.
Jyseleca® is a trademark of Galapagos NV and Gilead Sciences, Inc. or its related companies.
About the filgotinib collaboration
Gilead and Galapagos NV are collaborative partners in the global development and commercialization of filgotinib. Galapagos will be responsible for the commercialization of filgotinib in Europe (transition anticipated to be completed by end of 2021), while Gilead will remain responsible for filgotinib outside of Europe, including in Japan, where filgotinib is co-marketed with Eisai. Filgotinib in UC has been filed in Europe, Great-Britain and Japan, and a global Phase 3 program is ongoing in Crohn’s Disease. More information about clinical trials can be accessed at https://www.clinicaltrials.gov.
More information at www.glpg.com.
Galapagos' filgotinib wins positive CHMP opinion for treatment of ulcerative colitis
Sep. 17, 2021 8:41 AM ET
By: Mamta Mayani, SA News Editor
- Galapagos (NASDAQ:GLPG) announces that the EMA's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Jyseleca (filgotinib), a once-daily, oral, JAK1 preferential inhibitor for the treatment of adult patients with moderately to severely active ulcerative colitis (UC).
- https://seekingalpha.com/news/3740800-galapagos-filgotinib-wins-positive-chmp-opinion-for-treatment-of-ulcerative-colitis
- https://seekingalpha.com/symbol/GLPG
- https://seekingalpha.com/symbol/GILD
- https://www.glpg.com/
IMFINZI® (durvalumab) oleclumab, an anti-CD73 monoclonal antibody, or monalizumab, an anti-NKG2A monoclonal antibody, in combination with IMFINZI® (durvalumab)
Imfinzi combined with novel immunotherapies improved clinical outcomes for patients with unresectable, Stage III non-small cell lung cancer
PUBLISHED17 September 2021
17 September 2021 12:30 BST
COAST Phase II trial showed oleclumab or monalizumab in combination with Imfinzi significantly delayed disease progression and increased objective response rate
First effectiveness data from PACIFIC-R reinforced long-term benefit of Imfinzi in the real-world setting
Results from the large, randomised COAST Phase II trial showed oleclumab, an anti-CD73 monoclonal antibody, or monalizumab, an anti-NKG2A monoclonal antibody, in combination with Imfinzi (durvalumab) improved progression-free survival (PFS) and objective response rate (ORR) compared to Imfinzi alone in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who had not progressed after concurrent chemoradiation therapy (CRT).
After a median follow-up of 11.5 months, the results of an interim analysis showed Imfinzi in combination with oleclumab reduced the risk of disease progression or death by 56% (hazard ratio [HR] of 0.44; 95% confidence interval [CI] 0.26-0.75), and in combination with monalizumab by 35% (HR of 0.65; 95% CI 0.49-0.85), when compared to Imfinzi alone in Stage III NSCLC patients following CRT. The 10-month PFS rate was 64.8% for the durvalumab plus oleclumab combination and 72.7% for durvalumab plus monalizumab, versus 39.2% with durvalumab alone.
The results, presented during the European Society for Medical Oncology (ESMO) Congress 2021 today, also showed an increase in the primary endpoint of confirmed ORR for Imfinzi plus oleclumab over Imfinzi alone (30% vs. 18%) and for Imfinzi plus monalizumab over Imfinzi alone (36% vs. 18%).
One in four patients with NSCLC are diagnosed at Stage III, where the majority of tumours are unresectable (cannot be removed with surgery).1,2 Imfinzi after CRT is the global standard of care for patients in this setting, based on the PACIFIC Phase III trial.3-5
PACIFIC-real world observational study (PACIFIC-R) demonstrated benefit of Imfinzi in real-world setting at the ESMO Congress 2021
Data from a planned analysis of real-world PFS (rwPFS) from the PACIFIC-R observational study was also presented during ESMO, showing the first effectiveness data from over a thousand patients with unresectable, Stage III NSCLC who were treated with Imfinzi in the real-world setting as part of AstraZeneca’s global PACIFIC Early Access Programme. The analysis showed a median rwPFS of 21.7 months in the real-world setting.
In comparison, a median PFS of 16.9 months was observed among patients treated with Imfinzi in the randomised, double-blinded, placebo-controlled PACIFIC Phase III trial. These results demonstrate the long-term effectiveness of Imfinzi in this real-world patient population and reinforce the PACIFIC regimen as the established standard of care today following platinum-based CRT.
Imfinzi is approved in the curative-intent setting of unresectable, Stage III NSCLC after CRT in the US, Japan, China, across the EU and in many other countries with more than 80,000 patients treated with Imfinzi in this setting since its first approval in February 2018. Imfinzi is also approved for the 1st-line treatment of extensive-stage small cell lung cancer (ES-SCLC) in combination with etoposide and either carboplatin or cisplatin in more than 55 countries, including the US, Japan, China and across the EU, based on the CASPIAN Phase III trial.
AstraZeneca has several ongoing registrational trials focused on evaluating Imfinzi in earlier stages of lung cancer, including in potentially curative settings (PACIFIC-2, 4 and 5, MERMAID-1 and 2, AEGEAN, ADJUVANT BR.31, and ADRIATIC Phase III trials). The Company is also testing novel combinations with Imfinzi in the Phase II NeoCOAST trial in the neoadjuvant early-stage setting.
Imfinzi
Imfinzi is a human monoclonal antibody that binds to PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.
In addition to approvals in unresectable, Stage III NSCLC and ES-SCLC, Imfinzi is also approved for previously treated patients with advanced bladder cancer in several countries.
As part of a broad development programme, Imfinzi is being tested as a single treatment and in combinations with other anti-cancer treatments for patients with NSCLC, SCLC, bladder cancer, hepatocellular carcinoma, biliary tract cancer (a form of liver cancer), oesophageal cancer, gastric and gastroesophageal cancer, cervical cancer, ovarian cancer, endometrial cancer, and other solid tumours.
Oleclumab
Oleclumab is a potentially first-in-class, anti-CD73 monoclonal antibody that selectively binds to and inhibits the activity of CD73. CD73 is a cell surface enzyme which is overexpressed in the tumour microenvironment and promotes tumour growth by limiting anti-tumour immunity via the adenosine receptor pathway.
Preclinical studies have demonstrated that CD73 blockade improved anti-tumour activity in combination with radiotherapy, chemotherapy and immunotherapy. Oleclumab is also being examined in various Phase II trials for solid tumour malignancies.
Monalizumab
Monalizumab is a potentially first-in-class, anti-NKG2A antibody. NKG2A is a checkpoint receptor expressed on tumour-infiltrating cytotoxic T-cells and natural killer cells that inhibits their anti-cancer functions.
Monalizumab is also being studied in the ongoing INTERLINK-1 Phase III trial, evaluating monalizumab in combination with cetuximab for the treatment of patients with previously-treated recurrent or metastatic head and neck squamous cell carcinoma.
Monalizumab is being developed in collaboration with Innate Pharma. AstraZeneca obtained full oncology rights to monalizumab in October 2018 through a co-development and commercialisation agreement initiated in 2015.
Please visit astrazeneca.com
AstraZeneca's Imfinzi combo improves clinical outcomes in lung cancer patients
Sep. 17, 2021 8:17 AM ET AstraZeneca PLC (AZN)Innate Pharma S.A. (IPHA) By: Mamta Mayani, SA News Editor
- AstraZeneca (NASDAQ:AZN) announces that Imfinzi (durvalumab) in combination with oleclumab or monalizumab in Phase II COAST trial improved progression-free survival (PFS) and objective response rate (ORR) compared to Imfinzi alone in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who had not progressed after concurrent chemoradiation therapy (CRT).
- https://seekingalpha.com/news/3740782-astrazenecas-imfinzi-combo-improved-clinical-outcomes-in-lung-cancer-patients
- https://seekingalpha.com/symbol/AZN
- https://www.imfinzi.com/
- https://seekingalpha.com/symbol/IPHA
MONALIZUMAB DATA FROM COAST TRIAL PRESENTED AT ESMO CONGRESS 2021
Fri, 09/17/2021 - 13:45
Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced that AstraZeneca (LSE/STO/Nasdaq: AZN) presented results from the randomized COAST Phase 2 trial during the European Society for Medical Oncology (ESMO) Congress 2021 on September 17, 2021.
In particular, the results of the interim analysis showed monalizumab in combination with durvalumab improved progression-free survival (PFS) and objective response rate (ORR) compared to durvalumab alone in patients with unresectable, Stage III non-small cell lung cancer (NSCLC) who had not progressed after concurrent chemoradiation therapy (CRT). Monalizumab, Innate’s lead partnered asset, is a potentially first-in-class immune checkpoint inhibitor targeting NKG2A receptors expressed on tumor infiltrating cytotoxic CD8+ T cells and NK cells.
To read more about the Phase 2 COAST results, please see AstraZeneca’s press release here.
https://www.innate-pharma.com/products/monalizumab
https://www.innate-pharma.com/products/lacutamab
https://www.innate-pharma.com/
LUMAKRAS™ (Sotorasib) Combined With Vectibix® (Panitumumab)
LUMAKRAS™ (Sotorasib) Combined With Vectibix® (Panitumumab) Showed Encouraging Efficacy And Safety In Patients With KRAS G12C-Mutated Colorectal Cancer
New Data at ESMO 2021 Support Initiation of Phase 3 Trial of LUMAKRAS Plus Vectibix in Patients With 3L+ Colorectal Cancer
Amgen is Leading the Largest and Most Comprehensive Development Program in Patients with the KRAS G12C Mutation
THOUSAND OAKS, Calif., Sept. 16, 2021 /PRNewswire/ -- Amgen (NASDAQ: AMGN) today announced the first combination study results from the Phase 1b/2 CodeBreaK 101 study, the most comprehensive global clinical development program in patients with KRAS G12C-mutated advanced colorectal cancer (CRC). These new data show that combining LUMAKRAS™ (sotorasib) with Vectibix® (panitumumab), Amgen's monoclonal antibody epidermal growth factor receptor (EGFR) inhibitor, demonstrated encouraging efficacy and safety. Overall, the objective response rate (ORR) was 27% (confirmed and unconfirmed) among 26 patients in the efficacy analysis set (which included 5 patients who had progressed with prior sotorasib monotherapy). The disease control rate (DCR) was 81%. ORR and DCR were secondary endpoints. In the expansion cohort of sotorasib-naïve patients with refractory CRC (n=18), 33% of patients experienced a response (confirmed and unconfirmed). These data are being featured during the European Society of Medical Oncology 2021 (ESMO21) Virtual Congress.
Advancing Tarlatamab (formerly AMG 757) and AMG 404 in Small Cell Lung Cancer
In addition to the LUMAKRAS combination research, a presentation will detail the design of an ongoing study of half-life extended (HLE) bispecific T cell engager (BiTE®) molecule tarlatamab with anti-PD-1 antibody AMG 404 in patients with small cell lung cancer. The multicenter, open-label, Phase 1b study will evaluate the safety and tolerability of the combination and determine dosing as primary objectives, as well as examine preliminary antitumor activity and pharmacokinetics as secondary objectives.
About LUMAKRASTM (sotorasib)
Amgen took on one of the toughest challenges of the last 40 years in cancer research by developing LUMAKRAS, a KRASG12C inhibitor.1 LUMAKRAS has demonstrated a positive benefit-risk profile with rapid, deep and durable anticancer activity in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) harboring the KRAS G12C mutation with a once daily oral formulation.2
In May 2021, LUMAKRAS was the first KRASG12C inhibitor to receive regulatory approval anywhere in the world with its approval in the U.S., under accelerated approval. LUMAKRAS is also approved in the United Arab Emirates, and in Great Britain and Canada under Project Orbis.
Amgen is progressing the largest and broadest global KRASG12C inhibitor development program with unparalleled speed and exploring more than 10 sotorasib combination regimens, including triplets, with clinical trial sites spanning five continents. To date, LUMAKRAS has treated almost 3,000 patients around the world through the clinical development program and commercial use.
In the U.S., LUMAKRAS was reviewed by the FDA under its Real-Time Oncology Review (RTOR), a pilot program that aims to explore a more efficient review process that ensures safe and effective treatments are made available to patients as early as possible. Amgen is participating in the FDA's Project Orbis initiative and through the initiative, has Marketing Authorization Applications (MAAs) for sotorasib in review in Australia and Brazil. Additionally, Amgen has submitted an MAA in the European Union, Japan, Switzerland, South Korea, Singapore, Israel, Turkey, Taiwan, Colombia, Thailand, Mexico and Hong Kong.
LUMAKRAS is also being studied in multiple other solid tumors.1
About CodeBreaK
The CodeBreaK clinical development program for Amgen's drug sotorasib is designed to treat patients with an advanced solid tumor with the KRAS G12C mutation and address the longstanding unmet medical need for these cancers. As the most advanced KRASG12C inhibitor clinical development program, CodeBreaK has enrolled more than 800 patients across 13 tumor types since its inception.
CodeBreaK 100, the Phase 1 and 2, first-in-human, open-label multicenter study, enrolled patients with KRAS G12C-mutant solid tumors. Eligible patients must have received a prior line of systemic anticancer therapy, consistent with their tumor type and stage of disease. The primary endpoint for the Phase 2 study was centrally assessed objective response rate. The Phase 2 trial in NSCLC enrolled 126 patients, 124 of whom had centrally evaluable lesions by RECIST at baseline. The Phase 2 trial in colorectal cancer (CRC) is fully enrolled and results have been submitted for publication.
A global Phase 3 randomized active-controlled study comparing sotorasib to docetaxel in patients with KRAS G12C-mutated NSCLC (CodeBreaK 200) has completed enrollment. Amgen also has several Phase 1b studies investigating sotorasib monotherapy and sotorasib combination therapy across various advanced solid tumors (CodeBreaK 101) open for enrollment. A Phase 2 randomized study will evaluate sotorasib in patients with stage IV KRAS G12C-mutated NSCLC in need of first-line treatment (CodeBreaK 201).
For information, please visit www.hcp.codebreaktrials.com.
LUMAKRASTM (sotorasib) U.S. Indication
LUMAKRASTM is indicated for the treatment of adult patients with KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC), as determined by an FDA-approved test, who have received at least one prior systemic therapy.
This indication is approved under accelerated approval based on overall response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial(s).
Please see LUMAKRASTM full Prescribing Information.
About Vectibix® (panitumumab)
Vectibix is the first fully human monoclonal anti-EGFR antibody approved by the FDA for the treatment of mCRC. Vectibix was approved in the U.S. in September 2006 as a monotherapy for the treatment of patients with EGFR-expressing mCRC after disease progression after prior treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-containing chemotherapy.
In May 2014, the FDA approved Vectibix for use in combination with FOLFOX, as first-line treatment in patients with wild-type KRAS (exon 2) mCRC. With this approval, Vectibix became the first-and-only biologic therapy indicated for use with FOLFOX, one of the most commonly used chemotherapy regimens, in the first-line treatment of mCRC for patients with wild-type KRAS mCRC.
In June 2017, the FDA approved a refined indication for Vectibix for use in in patients with wild-type RAS (defined as wild-type in both KRAS and NRAS as determined by an FDA-approved test for this use) mCRC.
INDICATION AND LIMITATION OF USE
Vectibix® is indicated for the treatment of patients with wild-type RAS (defined as wild-type in both KRAS and NRAS as determined by an FDA-approved test for this use) metastatic colorectal cancer (mCRC): as first-line therapy in combination with FOLFOX, and as monotherapy following disease progression after prior treatment with fluoropyrimidine-, oxaliplatin-, and irinotecan-containing chemotherapy.
Limitation of Use: Vectibix® is not indicated for the treatment of patients with RAS mutant mCRC or for whom RAS mutation status is unknown.
To see the Vectibix® Prescribing Information, including Boxed Warning visit www.vectibix.com.
For more information, visit www.amgen.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/lumakras-sotorasib-combined-with-vectibix-panitumumab-showed-encouraging-efficacy-and-safety-in-patients-with-kras-g12c-mutated-colorectal-cancer-301378188.html
SOURCE Amgen
Amgen's Lumakras + Vectibix shows encouraging efficacy and safety in colorectal cancer
Sep. 16, 2021 5:11 AM ETAmgen Inc. (AMGN)By: Mamta Mayani, SA News Editor
- Amgen (NASDAQ:AMGN) announces the first combination study results from Phase 1b/2 CodeBreaK 101 study in patients with KRAS G12C-mutated advanced colorectal cancer (CRC).
- https://seekingalpha.com/news/3740278-amgens-lumakras-vectibix-shows-encouraging-efficacy-and-safety-in-colorectal-cancer
- https://seekingalpha.com/symbol/AMGN
- https://www.vectibix.com/
- https://www.lumakras.com/
Bemarituzumab (FPA144)
Zai Lab Announces Breakthrough Therapy Designation Granted for Bemarituzumab (FPA144) in China
September 14, 2021 at 7:30 AM EDTPDF VersionPotential first-in-class therapy for a subset of gastric and gastroesophageal (GEJ) cancers that overexpress fibroblast growth factor receptor 2 (FGFR2b)
SHANGHAI and SAN FRANCISCO, Sept. 14, 2021 (GLOBE NEWSWIRE) -- Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688), an innovative commercial-stage biopharmaceutical company, today announced that the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted Breakthrough Therapy Designation for investigational bemarituzumab (FPA144), for first-line treatment for patients with FGFR2b overexpressing and human epidermal growth factor receptor (HER2)-negative metastatic and locally advanced gastric and GEJ cancers in combination with modified FOLFOX6 (fluoropyrimidine, leucovorin, and oxaliplatin).
“In granting Breakthrough Therapy Designation, we are pleased to see that the CDE recognizes the promise of bemarituzumab. In combination with chemotherapy, bemarituzumab demonstrated clinically meaningful outcomes in key endpoints for patients with advanced gastric or GEJ cancer as a frontline therapy,” said Alan Sandler, M.D., president and head of global development, oncology, at Zai Lab. “We look forward to working with regulatory authorities in China as we advance bemarituzumab into global, registrational studies.”
The designation is supported by results from the Phase 2 FIGHT study, which evaluated bemarituzumab plus chemotherapy (modified FOLFOX6) versus chemotherapy alone in patients with FGFR2b overexpression, HER2-negative frontline advanced gastric or GEJ cancer. All three efficacy endpoints in the FIGHT trial – PFS, OS and ORR – achieved pre-specified statistical significance in the bemarituzumab arm compared to the placebo arm. Additional analysis showed a positive correlation between benefit and the prevalence of FGFR2b overexpression tumor cells, affirming both the importance of the FGFR2b target and the activity of bemarituzumab against this target. The Breakthrough Therapy Designation was granted based upon this subset of patients, based on IHC testing, showing at least 10% of tumor cells overexpressing FGFR2b.
More than one million new gastric cancer cases are diagnosed annually, and approximately half of all gastric cancer cases occur in China1. Nearly 88% of patients with advanced gastric and GEJ cancers are HER2-negative, and approximately 30% of these patients present with FGFR2b overexpression.
The Breakthrough Therapy Designation review policy is designed to promote the research and creation of drugs with apparent clinical advantages, which are intended for the prevention or treatment of serious life-threatening diseases or diseases which severely impact the quality of life for which there is no existing treatment or where sufficient evidence indicates advantages of the novel drug over currently available treatment options. Drugs that have been granted the Breakthrough Therapy Designation are prioritized by the CDE in communications, and in receiving guidance to promote the drug development progress.
Source: (1) Globocan 2020.
About Bemarituzumab
Bemarituzumab (anti-FGFR2b), also called FPA144, is a potential first-in-class investigational targeted antibody that is designed to block fibroblast growth factors (FGFs) from binding and activating FGFR2b, inhibiting several downstream pro-tumor signaling pathways and potentially slowing cancer progression. Bemarituzumab is being developed in gastric and GEJ cancers as a targeted therapy for tumors that overexpress FGFR2b.
Zai Lab has an exclusive license to develop and commercialize bemarituzumab in Greater China. Zai Lab collaborated with Five Prime (later acquired by Amgen) on the Phase 2 FIGHT trial in Greater China.
Initiation of the registrational program of bemarituzumab by Amgen is planned for the fourth quarter of 2021. Planning is underway for bemarituzumab clinical studies in other solid tumors, including squamous NSCLC.
Bemarituzumab (FGFR2b)
Overview
Bemarituzumab is a humanized monoclonal antibody specific to the human fibroblast growth factor receptor 2b (FGFR2b) in clinical development as a targeted therapy for tumors that overexpress FGFR2b, including gastric and gastroesophageal junction cancers. We have an exclusive license for bemarituzumab in mainland China, Hong Kong (China), Macau (China) and Taiwan region from Five Prime Therapeutics, Inc. (Five Prime). We are collaborating with Five Prime to conduct the randomized, controlled Phase III clinical trial FIGHT to evaluate bemarituzumab in combination with modified FOLFOX6 chemotherapy as a front-line therapy for the treatment of gastric and gastro-esophageal junction cancers. In a Phase I trial, bemarituzumab demonstrated monotherapy activity in heavily pre-treated patients with FGFR2b-positive gastric cancer and did not cause some of the toxicities that had been seen with less selective small molecule FGFR2 inhibitors. FGFR2b is a splice variant of a receptor for some members of the fibroblast growth factor (FGF) family. An estimated 10% of patients with gastric cancer have tumors that overexpress FGFR2b or have FGFR2 gene amplification, which is associated with poor prognosis.
http://www.zailaboratory.com/pipeline/info.aspx?lcid=5#d
For additional information about the company, please visit www.zailaboratory.com
http://www.zailaboratory.com/pipeline/list.aspx
Zai Lab nabs Breakthrough Therapy tag in China for GIT cancer therapy
Sep. 14, 2021 8:56 AM ET Zai Lab Limited (ZLAB) By: Dulan Lokuwithana, SA News Editor
- Zai Lab (NASDAQ:ZLAB) has been granted the Breakthrough Therapy Designation in China for experimental therapy bemarituzumab (FPA144) and modified FOLFOX6 combo as a first-line treatment for patients with certain forms of gastric and GEJ cancers.
- https://seekingalpha.com/news/3739480-zai-lab-nabs-breakthrough-therapy-tag-in-china-for-git-cancer-therapy
- https://seekingalpha.com/symbol/ZLAB
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab)
Five-Year Data from CheckMate -214 Show Opdivo (nivolumab) Plus Yervoy (ipilimumab) Demonstrates Longest Median Overall Survival Currently Reported in Phase 3 Trial of Patients with Previously Untreated Advanced or Metastatic Renal Cell Carcinoma
09/16/2021CATEGORY:
Nearly half of patients treated with the dual immunotherapy combination were alive at five years from the start of therapy in CheckMate -214 trial
Opdivo plus Yervoy continued to show durable responses, with the median duration of response not reached among all randomized patients after five years of follow-up
Data to be presented at the European Society for Medical Oncology Virtual Congress 2021
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that Opdivo (nivolumab) plus Yervoy (ipilimumab) continued to demonstrate durable, long-term survival in the Phase 3 CheckMate -214 trial, with a five-year survival rate of 48% in patients with previously untreated advanced or metastatic renal cell carcinoma (RCC). After a median follow-up of 67.7 months, Opdivo plus Yervoy maintained superior overall survival (OS) and response benefits versus sunitinib in both patients with intermediate- and poor-risk prognostic factors, the primary endpoint population, and across all randomized patients.
In intermediate- and poor-risk patients (n=847), Opdivo plus Yervoy (n=425) maintained treatment effects over five years, with ongoing improvements in OS and overall response rate (ORR), two of the trial’s primary endpoints, as well as in supportive endpoints:
- OS: The median OS was 47.0 months for intermediate- and poor-risk patients treated withOpdivo plus Yervoyversus 26.6 months with sunitinib (Hazard Ratio [HR] 0.68; 95% Confidence Interval [CI]: 0.58 to 0.81), and five-year survival rates were 43% and 31%, respectively.
- ORR: ORR benefits were maintained with Opdivoplus Yervoy compared to sunitinib (42% vs. 27%). In addition, more patients treated with the combinationachieved complete responses (11% vs. 2%).
- Duration of response (DOR): For patients treated with Opdivoplus Yervoy, median DOR was not reached, compared to 19.7 months with sunitinib.
An analysis of all randomized patients, or the intent-to-treat (ITT) population (n=1,096), similarly showed long-term benefits with Opdivo plus Yervoy (n=550):
- OS: Opdivoplus Yervoydemonstrated a median OS of 55.7 months in the ITT population, representing the longest survival outcome reported to date in a Phase 3 trial in advanced RCC, compared to 38.4 months with sunitinib (HR 0.72; 95% CI: 0.62 to 0.85). Five-year OS rates were 48% for patients treated with the dual immunotherapy combination and 37% for those receiving sunitinib.
- ORR: Opdivoplus Yervoy continued to show a higher ORR (39% vs. 32%) and complete response rate than sunitinib (12% vs. 3%). Also, more patients treated with the combinationachieved complete responses and subsequently did not progress (9.6% vs. 2.4%).
- DOR: Median DOR was not reached with Opdivoplus Yervoy and was 24.8 months with sunitinib.
Opdivo plus Yervoy-based combinations have shown significant improvements in OS in six Phase 3 clinical trials in five tumors to date: advanced renal cell carcinoma, non-small cell lung cancer, metastatic melanoma, malignant pleural mesothelioma and esophageal squamous cell carcinoma.
About CheckMate -214
CheckMate -214 is a Phase 3, randomized, open-label study evaluating the combination of Opdivo plus Yervoy versus sunitinib in patients with previously untreated advanced or metastatic renal cell carcinoma (RCC). Patients in the combination group (n=550) received Opdivo 3 mg/kg plus Yervoy 1 mg/kg every three weeks for four doses followed by Opdivo 3 mg/kg every two weeks. Patients in the comparator group (n=546) received sunitinib 50 mg once daily for four weeks, followed by two weeks off before continuation of treatment. Patients were treated until progression or unacceptable toxic effects for up to two years. The primary endpoints of the trial are overall survival (OS), progression-free survival (PFS) and objective response rate (ORR) in an intermediate- to poor-risk patient population (approximately 75% of patients). ORR was assessed by independent radiology review committee (IRRC).
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
About Yervoy
Yervoy is a recombinant, human monoclonal antibody that binds to the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activity. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation, including the activation and proliferation of tumor infiltrating T-effector cells. Inhibition of CTLA-4 signaling can also reduce T-regulatory cell function, which may contribute to a general increase in T-cell responsiveness, including the anti-tumor immune response. On March 25, 2011, the U.S. Food and Drug Administration (FDA) approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is approved for unresectable or metastatic melanoma in more than 50 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types.
INDICATIONS
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY.
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers' Opdivo + Yervoy shows longest overall survival in late-stage kidney cancer study
Sep. 16, 2021 8:18 AM ETBristol-Myers Squibb Company (BMY)By: Mamta Mayani, SA News Editor4 Comments
- Bristol Myers Squibb (NYSE:BMY) announces that Opdivo (nivolumab) plus Yervoy (ipilimumab) continued to demonstrate durable, long-term survival in Phase 3 CheckMate -214 trial, with a five-year survival rate of 48% in patients with previously untreated advanced or metastatic renal cell carcinoma (RCC).
- https://seekingalpha.com/news/3740391-bristol-myers-opdivo-yervoy-shows-longest-median-overall-survival-in-late-stage-kidney-cancer-study
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy
BRUKINSA® (Zanubrutinib)
U.S. FDA Grants BRUKINSA® (Zanubrutinib) Accelerated Approval in Relapsed or Refractory Marginal Zone Lymphoma
Sep 15, 2021 7:00 AM
This marks the third FDA approval for BRUKINSA and first approval in marginal zone lymphoma
Twenty percent of patients achieved complete remission with single-agent BRUKINSA
BRUKINSA was generally well-tolerated, consistent with its known safety profile
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that BRUKINSA® (zanubrutinib) has received accelerated approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with relapsed or refractory (R/R) marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen.
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This accelerated approval is based on overall response rate (ORR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
“We are excited about the FDA’s approval for BRUKINSA in patients with previously treated marginal zone lymphoma, a significant milestone that was made possible by the diligent BeiGene team, the dedicated investigators, and the participating patients and their families. The MAGNOLIA trial results provided additional evidence that the selective design of BRUKINSA can be translated to improved treatment outcomes for these patients,” said Jane Huang, M.D., Chief Medical Officer, Hematology at BeiGene. “The ongoing evaluation of BRUKINSA in its broad global clinical program will enable us to further understand this potentially best-in-class BTK inhibitor and its impact on patients. Since the initial FDA approval in November 2019, BRUKINSA has been granted 12 approvals in four indications globally. We will continue to execute on our mission to improve access to innovative and quality treatments for cancer patients worldwide.”
“BTK plays a critical role in B-cell receptor signaling, a driver in the development of marginal zone lymphoma. In the MAGNOLIA trial, BRUKINSA demonstrated impressive overall response and complete remission rates, with responses observed in all MZL subtypes. In addition, this next-generation BTK inhibitor was well-tolerated in these patients, with low rate of discontinuation due to adverse reactions. We are optimistic that BRUKINSA will bring clinically meaningful benefit to patients with relapsed or refractory marginal zone lymphoma,” said Stephen Opat, FRACP, FRCPA, MBBS, Director of Clinical Hematology at Monash Health, Head of Department of Hematology at Monash University, and lead principal investigator of the MAGNOLIA trial.
“The approval of BRUKINSA offers patients with relapsed and refractory marginal zone lymphoma a new treatment option and new hope for improving patient outcomes,” commented Meghan Gutierrez, Chief Executive Officer of the Lymphoma Research Foundation.
The FDA approval of BRUKINSA is based on efficacy results from two single-arm clinical trials, with ORR as assessed by independent review committee (IRC) per 2014 Lugano Classification as the primary endpoint.
About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA is approved in the following indications and regions:
- For the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (United States, November 2019)*;
- For the treatment of MCL in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of relapsed or refractory MCL (United Arab Emirates, February 2021);
- For the treatment of Waldenström’s macroglobulinemia (WM) in adult patients (Canada, March 2021);
- Registered and reimbursed for the treatment of MCL in patients who have received at least one prior therapy (Israel, April 2021);
- For the treatment of adult patients with WM who have received at least one prior therapy (China, June 2021)**;
- For the treatment of MCL in adult patients who have received at least one prior therapy (Canada, July 2021);
- For the treatment of MCL in adult patients who have received at least one prior therapy (Chile, July 2021);
- For the treatment of adult patients with MCL who have received at least one previous therapy (Brazil, August 2021);
- For the treatment of adult patients with WM (United States, August 2021); and
- For the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen (United States, September 2021)*.
To date, more than 30 marketing authorization applications in multiple indications have been submitted in the United States, China, the European Union, and more than 20 other countries or regions.
* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
** This indication was approved under conditional approval. Complete approval for this indication may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
Please see full U.S. Prescribing Information at www.beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at www.beigene.com/PDF/BRUKINSAUSPPI.pdf.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210915005310/en/
Source: BeiGene, Ltd.
FDA grants BeiGene's Brukinsa accelerated approval in marginal zone lymphoma
Sep. 15, 2021 7:44 AM ET BeiGene, Ltd. (BGNE)
By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) announces that Brukinsa (zanubrutinib) has received accelerated approval from the FDA for the treatment of adult patients with relapsed or refractory (R/R) marginal zone lymphoma (MZL) who have received at least one anti-CD20-based regimen.
- https://seekingalpha.com/news/3739857-fda-grants-beigenes-brukinsa-accelerated-approval-in-marginal-zone-lymphoma
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/
Tislelizumab (BGB-A317)
BeiGene Announces U.S. FDA Acceptance of Biologics License Application for Tislelizumab in Esophageal Squamous Cell Carcinoma
Sep 13, 2021 12:00 AM
This marks the first ex-China regulatory filing for tislelizumab, following approval in five indications in China
The accepted BLA, filed in collaboration with Novartis, is supported by the positive global Phase 3 RATIONALE 302 trial in patients with previously treated, advanced or metastatic ESCC and safety data from tislelizumab’s broad clinical program
With its second internally developed medicine filed outside China, BeiGene furthers its commitment to expanding access to innovative treatments for cancer patients worldwide
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global, science-driven biotechnology company focused on developing innovative and affordable medicines to improve treatment outcomes and access for patients worldwide, today announced that the U.S. Food and Drug Administration (FDA) accepted for review a Biologics License Application (BLA) for its anti-PD-1 antibody tislelizumab as a treatment for patients with unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) after prior systemic therapy. The Prescription Drug User Fee Act (PDUFA) target action date is July 12, 2022.
“Our uniquely designed anti-PD-1 antibody tislelizumab has been shown to significantly improve survival compared to chemotherapy for people with a variety of solid tumors and hematologic malignancies. We previously shared the compelling results at ASCO 2021 with tislelizumab significantly prolonging survival and demonstrating a favorable safety profile over chemotherapy in patients with locally advanced or metastatic ESCC, a devastating disease with an average five-year survival rate of just five percent. This BLA acceptance brings us closer to potentially providing tislelizumab as a treatment for these patients in the United States,” said Yong (Ben) Ben, M.D., Chief Medical Officer, Immuno-Oncology at BeiGene. “Tislelizumab is already approved in five indications in China and has the potential to become a preferred immunotherapy option there. We look forward to continued collaboration with Novartis to work to bring access to tislelizumab to patients around the world.”
The BLA submission is based on results from RATIONALE 302, a randomized, open-label, multicenter global Phase 3 trial (NCT03430843) designed to evaluate the efficacy and safety of tislelizumab when compared to investigator’s choice chemotherapy as a second-line treatment for patients with advanced or metastatic ESCC. Results of this trial were presented at the 2021 American Society of Clinical Oncology Annual Meeting (ASCO 2021). The submission also included safety data on 1,972 patients who received tislelizumab as a monotherapy from seven clinical trials.
In addition to the United States, tislelizumab is also under regulatory review in China as a treatment for patients with locally advanced or metastatic ESCC who have disease progression following or are intolerant to first-line standard chemotherapy.
About Tislelizumab
Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
The China National Medical Products Administration (NMPA) has approved tislelizumab in five indications, including full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy and for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy. NMPA also granted conditional approval for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy, and for the treatment of patients with hepatocellular carcinoma (HCC) who have received at least one systemic therapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
In addition, three supplemental Biologics License Applications for tislelizumab are under review by the Center for Drug Evaluation (CDE) of the NMPA, including as second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy, for patients with previously treated, locally advanced unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR) solid tumors and for the treatment of patients with locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression following or are intolerant to first-line standard chemotherapy.
In the U.S., a Biologics License Application for tislelizumab as a treatment for patients with unresectable recurrent locally advanced or metastatic ESCC after prior systemic therapy is currently under review by the U.S. Food and Drug Administration with a PDUFA target action date of July 12, 2022.
BeiGene has initiated or completed 17 potentially registration-enabling clinical trials in China and globally, including 13 Phase 3 trials and four pivotal Phase 2 trials.
In January 2021, BeiGene and Novartis entered into a collaboration and license agreement granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.
About the Tislelizumab Clinical Program
Clinical trials of tislelizumab include:
- Phase 3 trial comparing tislelizumab with docetaxel in the second- or third-line setting in patients with NSCLC (NCT03358875);
- Phase 3 trial comparing tislelizumab to salvage chemotherapy in patients with relapsed or refractory classical Hodgkin Lymphoma (cHL; NCT04486391);
- Phase 3 trial in patients with locally advanced or metastatic urothelial carcinoma (NCT03967977);
- Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced squamous NSCLC (NCT03594747);
- Phase 3 trial of tislelizumab in combination with chemotherapy versus chemotherapy as first-line treatment for patients with advanced non-squamous NSCLC (NCT03663205);
- Phase 3 trial of tislelizumab in combination with platinum-based doublet chemotherapy as neoadjuvant treatment for patients with NSCLC (NCT04379635);
- Phase 3 trial of tislelizumab combined with platinum and etoposide versus placebo combined with platinum and etoposide in patients with extensive-stage small cell lung cancer (NCT04005716);
- Phase 3 trial comparing tislelizumab with sorafenib as first-line treatment for patients with hepatocellular carcinoma (HCC; NCT03412773);
- Phase 2 trial in patients with previously treated unresectable HCC (NCT03419897);
- Phase 2 trial in patients with locally advanced or metastatic urothelial bladder cancer (NCT04004221);
- Phase 3 trial comparing tislelizumab with chemotherapy as second-line treatment for patients with advanced esophageal squamous cell carcinoma (ESCC; NCT03430843);
- Phase 3 trial of tislelizumab in combination with chemotherapy as first-line treatment for patients with ESCC (NCT03783442);
- Phase 3 trial of tislelizumab versus placebo in combination with chemoradiotherapy in patients with localized ESCC (NCT03957590);
- Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment for patients with gastric cancer (NCT03777657);
- Phase 2 trial of tislelizumab in patients with relapsed or refractory cHL (NCT03209973);
- Phase 2 trial in patients with MSI-H/dMMR solid tumors (NCT03736889); and
- Phase 3 trial of tislelizumab combined with chemotherapy versus placebo combined with chemotherapy as first-line treatment in patients with nasopharyngeal cancer (NCT03924986).
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210912005031/en/
Source: BeiGene, Ltd.
FDA accepts BLA from BeiGene/Novartis for tislelizumab for esophageal cancer
Sep. 14, 2021 12:10 PM ET BeiGene, Ltd. (BGNE), NVS By: Jonathan M Block, SA News Editor
- The FDA has accepted a BLA filing from BeiGene (BGNE +0.5%) for its anti-PD-1 immune checkpoint inhibitor tislelizumab for the treatment of unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma as a second-line therapy.
- https://seekingalpha.com/news/3739572-fda-accepts-bla-from-beigene-novartis-for-tislelizumab-for-esophageal-cancer
- https://seekingalpha.com/symbol/BGNE
- https://seekingalpha.com/symbol/NVS
biotecMAX™ April/May/June/July/Aug 2021 Insight
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab)
Opdivo (nivolumab) Plus Yervoy (ipilimumab) Demonstrates Durable Overall Survival at Three YearsCompared to Chemotherapy in First-Line Unresectable Malignant Pleural Mesothelioma in Phase 3 CheckMate -743 Trial
09/13/2021CATEGORY:
CheckMate -743 remains the only Phase 3 trial to show a survival improvement – now sustained over three years – with first-line immunotherapy treatment in patients with unresectable malignant pleural mesotheliomaData to be featured in a Proffered Paper session during the 2021 European Society for Medical Oncology Virtual CongressMesothelioma is the fourth tumor type in which Opdivo plus Yervoy has demonstrated durable, superior survival with three years or more of follow-upPRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced three-year data from the CheckMate -743 trial that demonstrated a durable survival benefit with first-line treatment with Opdivo (nivolumab) plus Yervoy (ipilimumab) compared to platinum-based standard-of-care chemotherapy in patients with unresectable malignant pleural mesothelioma (MPM), regardless of histology.With a minimum follow-up of three years (35.5 months):
- Among patients treated with Opdivo plus Yervoy, 23% were alive at three years, compared to 15% of patients treated with chemotherapy.
- Treatment with the dual immunotherapy combination continued to show a reduction in the risk of death (Hazard Ratio [HR] 0.73; 95% Confidence Interval [CI]: 0.61 to 0.87), and improvement in median overall survival (OS), the trial’s primary endpoint, vs. chemotherapy (18.1 months vs. 14.1 months, respectively).
The safety profile for Opdivo plus Yervoy remained consistent with previously reported data in first-line MPM, with no new safety signals identified. These data will be presented on September 17, 2021 at 13:40 CEST/7:40 a.m. EDT (Abstract #LBA65) during the Proffered Paper session at the 2021 European Society for Medical Oncology (ESMO) Virtual Congress.“For patients with malignant pleural mesothelioma, the prognosis is generally poor, with a five-year survival rate of approximately 10%,” said Solange Peters, M.D., Ph.D., Medical Oncology Service, Chair, Thoracic Oncology, Lausanne University Hospital, Lausanne, Switzerland. “In this aggressive cancer that historically has had limited treatment options, we’ve now not only seen the potential for patients to live longer with nivolumab plus ipilimumab, but that this benefit is sustained at three years compared to treatment with chemotherapy. These results give us further proof of the durability of the outcomes achieved with this combination.”At three years of follow-up and approximately one year of being off treatment per protocol, more patients who responded to Opdivo plus Yervoy remained in response than those treated with chemotherapy, and duration of response (DOR) was longer with the dual immunotherapy combination, regardless of histology:
- 28% of patients who responded to Opdivo plus Yervoy remained in response at three years compared to no patients (0%) in the chemotherapy arm.
- Those treated with the dual immunotherapy combinationhad a median DOR of 11.6 months, compared to 6.7 months with chemotherapy.
- Objective response rate (ORR) in patients treated with the combination was comparable to chemotherapy (39.6% vs. 44.0%, respectively).
“The results from the CheckMate -743 trial have changed the way physicians treat malignant pleural mesothelioma, a disease that had no new systemic treatment options for nearly 15 years before the approval of Opdivo plus Yervoy,” said Abderrahim Oukessou, M.D., vice president, thoracic cancers development lead, Bristol Myers Squibb. “We continue to see more evidence for the sustained survival benefits of dual immunotherapy across several tumors, including durable overall survival at four years in non-small cell lung cancer. Now, the combination has demonstrated long-term improvements in overall survival in mesothelioma, another thoracic cancer, extending the lives of patients with a devastating disease.”Opdivo plus Yervoy has received approval for use in first-line unresectable MPM from 14 health authorities around the world, including in the U.S., European Union, Japan and China. Additional regulatory applications are under review globally.Opdivo plus Yervoy-based combinations have shown significant improvements in OS in six Phase 3 clinical trials in five tumors to date: malignant pleural mesothelioma, non-small cell lung cancer, metastatic melanoma, advanced renal cell carcinoma and esophageal squamous cell carcinoma.
About CheckMate -743CheckMate -743 is an open-label, multi-center, randomized Phase 3 trial evaluating Opdivo plus Yervoy compared to chemotherapy (pemetrexed and cisplatin or carboplatin) in patients with previously untreated unresectable malignant pleural mesothelioma (n=605). In the trial, 303 patients were randomized to receive Opdivo at 3 mg/kg every two weeks and Yervoy at 1 mg/kg every six weeks; 302 patients were randomized to receive cisplatin 75 mg/m2 or carboplatin AUC 5 plus pemetrexed 500 mg/m2 in 21-day cycles for six cycles. Treatment in both arms continued until disease progression or unacceptable toxicity or, in the Opdivo plus Yervoy arm, up to 24 months. The primary endpoint of the trial was overall survival (OS) in all randomized patients. Additional efficacy outcome measures included progression-free survival (PFS), objective response rate (ORR) and duration of response (DOR), as assessed by blinded independent central review (BICR) utilizing modified RECIST criteria. Exploratory endpoints included safety, pharmacokinetics, immunogenicity and patient reported outcomes.
About OpdivoOpdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology, and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
About YervoyYervoy is a recombinant, human monoclonal antibody that binds to the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activity. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation, including the activation and proliferation of tumor infiltrating T-effector cells. Inhibition of CTLA-4 signaling can also reduce T-regulatory cell function, which may contribute to a general increase in T-cell responsiveness, including the anti-tumor immune response. On March 25, 2011, the U.S. Food and Drug Administration (FDA) approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is approved for unresectable or metastatic melanoma in more than 50 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types.
INDICATIONSOPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable or metastatic melanoma.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
Please see US Full Prescribing Information for OPDIVO and YERVOY.
For more information about Bristol Myers Squibb, visit us at BMS.com
Opdivo/Yervoy combo shows long-term survival benefit as first-line mesothelioma treatment
Sep. 13, 2021 11:56 AM ET Bristol-Myers Squibb Company (BMY) By: Jonathan M Block, SA News Editor4 Comments
- A combination of Bristol-Myers Squibb's (BMY +0.1%) Opdivo (nivolumab) and Yervoy (ipilimumab) demonstrated a superior survival benefit over at least three years compared to chemotherapy as a first-line therapy in unresectable malignant pleural mesothelioma.
- https://seekingalpha.com/news/3739158-opdivoyervoy-combo-shows-long-term-survival-benefit-as-first-line-mesothelioma-treatment
- https://www.opdivo.com/about-opdivo/how-the-combination-works-combinationtherapy
- https://www.bms.com/
- https://seekingalpha.com/symbol/BMY
Zabdeno® (Ad26.ZEBOV) and Mvabea® (MVA-BN-Filo)
Johnson & Johnson Ebola Vaccine Regimen Demonstrated Robust and Durable Immune Response in Adults and Children in Data Published in The Lancet Infectious Diseases
Sep 13, 2021United States
Data show the vaccine regimen induced neutralizing antibody responses in nearly all participating adults and children 21 days after the second dose
Adults receiving booster shots two years after initial vaccination regimen showed strong immune responses
The data support the potential prophylactic use of the Johnson & Johnson Ebola vaccine regimen to protect adults and children
NEW BRUNSWICK, N.J., September 13, 2021 – Data from two papers published in The Lancet Infectious Diseases demonstrated that the Johnson & Johnson (the Company) Ebola vaccine regimen, Zabdeno® (Ad26.ZEBOV) and Mvabea® (MVA-BN-Filo), generated robust humoral (antibody) immune responses in adults and children (ages 1-17) with the immune responses persisting in adults for at least two years. The data also showed that booster vaccination with Ad26.ZEBOV, administered to adults two years after the initial vaccination, induced a strong anamnestic (immune) response within seven days. These findings support the potential prophylactic use of the vaccine regimen, which was developed by the Janssen Pharmaceutical Companies of Johnson & Johnson (Janssen) in collaboration with Bavarian Nordic A/S, and was granted Marketing Authorisation by the European Commission in July 2020 and Prequalification from the World Health Organization (WHO) in April 2021.
The data is from the Phase 3 EBOVAC-Salone clinical study and showed that the vaccine regimen was well-tolerated and induced antibody responses to the Zaire ebolavirus species 21 days after the second dose in 98 percent of all participants. There were no safety signals of concern.
“These peer-reviewed data support the prophylactic use of the Johnson & Johnson Ebola vaccine regimen to protect people at risk of Ebola, which is essential to our vision of preventing Ebola outbreaks before they can begin,” said Paul Stoffels, M.D., Vice Chairman of the Executive Committee and Chief Scientific Officer of Johnson & Johnson. “Recent and ongoing outbreaks in Africa underscore that the threat of Ebola is not going away, which is why we collaborated to develop a vaccine regimen capable of inducing long-term immunity against Ebola and are now working to ensure that it is accessible to people in need.”
The EBOVAC-Salone study was conducted in Sierra Leone and is the first to assess the safety and tolerability of the Johnson & Johnson Ebola vaccine regimen in adults in a region affected by the 2014-2016 West African Ebola outbreak, which was the worst on record. It is also the first study evaluating the Johnson & Johnson Ebola vaccine regimen in a randomized, double-blind, controlled trial in a pediatric population.
Phase 3 Study Design (NCT02509494)
This Phase 3 study was designed to gather information on the safety and immunogenicity of the two-dose, heterologous (containing different vaccine components administered at different timepoints) Johnson & Johnson Ebola vaccine regimen. In this regimen, Ad26.ZEBOV was administered intramuscularly as the first dose vaccination followed 56 days later by MVA-BN-Filo as the second dose vaccination.
The study was divided into two stages. In stage one, 43 adults aged 18 years or older were vaccinated to gain information about the safety and immunogenicity of the two-dose vaccine regimen. In stage two, 400 adults and 576 children or adolescents (including 192 in each of the three age cohorts of 1-3, 4-11 and 12-17 years of age) were vaccinated. Consenting adults participating in stage one of the study were administered a booster dose of A26.ZEBOV two years after the first dose.
The study was conducted at three clinics in Kambia District, Sierra Leone. Long term follow-up of the study participants is underway.
Johnson & Johnson’s Ebola vaccine regimen originates from a collaborative research program with the NIH and received direct funding and preclinical services from the National Institute of Allergy and Infectious Diseases, part of NIH, under Contract Number HHSN272200800056C. Further funding for the Ebola vaccine regimen has been provided in part with federal funds from the Office of the Assistant Secretary for Preparedness and Response, BARDA under Contract Numbers HHSO100201700013C and HHSO100201500008C.
Learn more at www.JNJ.com/Ebola.
Learn more at www.jnj.com.
Learn more at www.jnj.com.
Learn more at www.janssen.com.
Johnson & Johnson Ebola vaccine yields strong immune response, peer reviewed data show
Sep. 14, 2021 6:39 AM ET Johnson & Johnson (JNJ)
By: Dulan Lokuwithana, SA News Editor2 Comments
- The Ebola vaccine, developed by Johnson & Johnson (NYSE:JNJ) in partnership with Bavarian Nordic A/S, generated a strong immune reaction in adults and children and the response last at least two years, according to two papers published in the medical journal The Lancet Infectious Diseases.
- In Phase 3 EBOVAC-Salone trial, antibody responses to the Zaire ebolavirus species were seen 21 days after the second dose in 98% of the participants who received the Zabdeno (Ad26.ZEBOV) and Mvabea (MVA-BN-Filo) vaccine regimen, J&J (JNJ) said.
- https://seekingalpha.com/news/3739398-johnson-johnson-ebola-vaccine-yields-strong-immune-response-peer-reviewed-data-show
- https://seekingalpha.com/symbol/JNJ
- https://www.jnj.com/
- https://www.janssen.com/
- https://www.jnj.com/ebola
Zanidatamab
Zymeworks Announces Abstract For Zanidatamab In First-Line HER2-Expressing Gastroesophageal Cancers (GEA) At The European Society For Medical Oncology (ESMO) Annual Congress
09/12/2021 at 6:49 PM EDTPDF Version
- Abstract data demonstrate encouraging antitumor activity in first-line GEA
- Full ESMO presentation available September 16 at 8:30 am CEST, 2:30 am ET
- Conference call and webcast with principal investigator, Dr. Geoffrey Ku, on September 16 at 7:30 am ET
VANCOUVER, British Columbia--(BUSINESS WIRE)--Sep. 12, 2021-- Zymeworks Inc. (NYSE: ZYME), a clinical-stage biopharmaceutical company developing multifunctional biotherapeutics, today announced the publication of an abstract highlighting new clinical data for zanidatamab, a HER2-targeted bispecific antibody, in first-line HER2-expressing GEA. An updated and expanded data set will be presented at the ESMO Annual Congress taking place virtually on September 16-21, 2021.
Abstract highlights from March 18, 2021 data cut:
- Thirty patients had been treated with zanidatamab in combination with standard of care chemotherapy (either mFOLFOX6, CAPOX, or FP), and 14 patients remained on treatment.
- The confirmed objective response rate was 68.2% and the disease control rate was 90.9% in 22 HER2-positive response-evaluable patients.
- Treatment related adverse events were generally consistent with previous reports of zanidatamab and/or the chemotherapy regimens, with the majority reported as Grade 1 or 2 in severity.
“The initial data from the abstract highlight an encouraging objective response rate for zanidatamab combined with standard of care chemotherapy in patients with metastatic HER2-positive GEA,” said Neil Josephson, M.D., Zymeworks’ Interim Chief Medical Officer. “We’re looking forward to presenting at the Congress the full updated data, which further support zanidatamab’s potential as the new foundational HER2-targeted therapy.”
ESMO Presentation
The abstract is available on the ESMO conference website. The presentation will be available on Thursday, September 16 at 8:30 am CEST, 2:30 am ET, to conference registrants on the ESMO conference website as well as to the general public on the Zymeworks website at https://ir.zymeworks.com/events-and-presentations.
Title: Phase (Ph) 2 Study of Zanidatamab + Chemotherapy (chemo) in First Line (1L) HER2-expressing Gastroesophageal Adenocarcinoma (GEA)
Lead Author: Geoffrey Ku, M.D., Memorial Sloan Kettering Cancer Center, New York, NY, US
Abstract: 3678
E-poster: 1380P
About Zanidatamab
Zanidatamab is a bispecific antibody, based on Zymeworks’ Azymetric™ platform, that can simultaneously bind two non-overlapping epitopes of HER2, known as biparatopic binding. This unique design results in multiple mechanisms of action including dual HER2 signal blockade, increased binding and removal of HER2 protein from the cell surface, and potent effector function leading to encouraging antitumor activity in patients. Zymeworks is developing zanidatamab in multiple Phase 1, Phase 2, and pivotal clinical trials globally as a targeted treatment option for patients with solid tumors that express HER2. The FDA has granted Breakthrough Therapy designation for zanidatamab in patients with previously treated HER2 gene-amplified biliary tract cancer (BTC), and two Fast Track designations to zanidatamab, one as a single agent for refractory BTC and one in combination with standard of care chemotherapy for first-line gastroesophageal adenocarcinoma (GEA). These designations mean zanidatamab is eligible for Accelerated Approval, Priority Review and Rolling Review, as well as intensive FDA guidance on an efficient drug development program. Zanidatamab has also received Orphan Drug designations for the treatment of biliary tract, gastric and ovarian cancers from the FDA, as well as Orphan Drug designation for the treatment of biliary tract and gastric cancer from the European Medicines Agency.
For additional information about Zymeworks, visit www.zymeworks.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210912005060/en/
Source: Zymeworks Inc.
Zymeworks' zanidatamab shows encouraging antitumor activity in esophageal cancer
Sep. 12, 2021 11:52 PM ET Zymeworks Inc. (ZYME) By: Mamta Mayani, SA News Editor1 Comment
- Zymeworks (NYSE:ZYME) announces the publication of an abstract highlighting new clinical data for zanidatamab, a HER2-targeted bispecific antibody, in first-line HER2-expressing Gastroesophageal Adenocarcinoma (GEA).
- https://seekingalpha.com/news/3738906-zymeworks-zanidatamab-shows-encouraging-antitumor-activity-in-esophageal-cancer
- https://seekingalpha.com/symbol/ZYME
- https://www.zymeworks.com/pipeline
Jardiance® (empagliflozin)
FDA grants Jardiance® Breakthrough Therapy designation for heart failure with preserved ejection fraction
September 9, 2021Download PDF- Designation is based on results from the EMPEROR-Preserved phase III trial, which established Jardiance as the first therapy to show statistically significant improvement in heart failure outcomes in adults with heart failure with preserved ejection fraction
RIDGEFIELD, Conn. and INDIANAPOLIS, Sept. 9, 2021 /PRNewswire/ -- The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation for Jardiance® (empagliflozin) as an investigational treatment for adults with heart failure with preserved ejection fraction (HFpEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced today.The decision is based on results from the landmark EMPEROR-Preserved phase III trial, in which Jardiance demonstrated a 21% relative risk reduction for the composite primary endpoint of cardiovascular death or hospitalization for heart failure in adults with heart failure with preserved ejection fraction compared with placebo. The benefit was independent of ejection fraction or diabetes status. Results from EMPEROR-Preserved were presented at the European Society of Cardiology Congress 2021 on August 27 and published in The New England Journal of Medicine.HFpEF accounts for approximately half of the more than 6 million heart failure cases in the U.S. No currently approved treatments have been clinically proven to significantly improve outcomes specifically for people with HFpEF."This Breakthrough Therapy designation underscores the potential of Jardiance to help fill a critical need for a clinically proven treatment for people with this highly prevalent, difficult-to-treat condition," said Mohamed Eid, M.D., M.P.H., M.H.A., vice president, Clinical Development & Medical Affairs, Cardio-Metabolism & Respiratory Medicine, Boehringer Ingelheim Pharmaceuticals, Inc. "Following the recent FDA approval of Jardiance for heart failure with reduced ejection fraction, this is another important milestone supporting the potential of Jardiance as the first therapy clinically proven to improve outcomes across the full heart failure spectrum."According to the FDA, Breakthrough Therapy designation is designed to expedite the development and review of therapies that are intended to treat a serious condition where preliminary clinical evidence indicates potentially substantial improvement over available therapies on a clinically significant endpoint. "Given the scarcity of treatment options for this debilitating condition, the benefits demonstrated in the EMPEROR-Preserved trial constituted a major clinical breakthrough," said Jeff Emmick, M.D., Ph.D., vice president, Product Development, Lilly. "Together with our Boehringer Ingelheim partners, we look forward to working closely with the FDA through this accelerated process to potentially bring Jardiance to adults with heart failure with preserved ejection fraction as soon as possible."The FDA previously granted Fast Track designation for the development of Jardiance to reduce the risk of cardiovascular death and hospitalization for heart failure. This designation is for the EMPEROR program, which consists of the EMPEROR-Reduced and EMPEROR-Preserved trials. The EMPEROR-Reduced results formed the basis of the recent FDA approval for heart failure with reduced ejection fraction. Jardiance is not indicated for the treatment of HFpEF.
About EMPEROR-PreservedEMPEROR-Preserved (NCT03057951) was a phase III international, randomized, double–blind trial that enrolled 5,988 adults with and without type 2 diabetes. All participants had heart failure (New York Heart Association [NYHA] functional class II, III or IV) and preserved ejection fraction (left ventricular ejection fraction > 40%).Participants were randomized to once-daily Jardiance 10 mg (n=2997) or placebo (n=2991), on top of treatment with guideline-directed heart failure therapy. Median follow-up time was 26.2 months. The composite primary endpoint was defined as time to first event of cardiovascular death or hospitalization for heart failure.
What is JARDIANCE? (www.jardiance.com)JARDIANCE is a prescription medicine used to:
- lower blood sugar along with diet and exercise in adults with type 2 diabetes
- reduce the risk of cardiovascular death in adults with type 2 diabetes who also have known cardiovascular disease
- reduce the risk of cardiovascular death and hospitalization for heart failure (when the heart is weak and cannot pump enough blood to the rest of your body) in adults with heart failure
JARDIANCE is not for people with type 1 diabetes. It may increase their risk of diabetic ketoacidosis (increased ketones in the blood or urine).JARDIANCE is not for use to lower blood sugar in adults with type 2 diabetes who have severe kidney problems, because it may not work.
Boehringer Ingelheim and Eli Lilly and CompanyIn January 2011, Boehringer Ingelheim and Eli Lilly and Company announced an alliance that centers on compounds representing several of the largest diabetes treatment classes. Depending on geographies, the companies either co-promote or separately promote the respective molecules each contributing to the alliance. The alliance leverages the strengths of two of the world's leading pharmaceutical companies to focus on patient needs. By joining forces, the companies demonstrate their commitment, not only to the care of people with diabetes, but also to investigating the potential to address areas of unmet medical need. Clinical trials have been initiated to evaluate the impact of Jardiance on people living with heart failure or chronic kidney disease.
For more information, please see Prescribing Information and Medication Guide.
For more information, please visit www.boehringer-ingelheim.us,
To learn more about Lilly, please visit us at lilly.com
View original content to download multimedia:https://www.prnewswire.com/news-releases/fda-grants-jardiance-breakthrough-therapy-designation-for-heart-failure-with-preserved-ejection-fraction-301372022.htmlSOURCE Eli Lilly and Company
Eli Lilly's Jardiance wins FDA's Breakthrough Therapy status for heart failure indication
Sep. 09, 2021 8:35 AM ETEli Lilly and Company (LLY)By: Aakash Babu, SA News Editor
- The U.S. FDA has granted Eli Lilly (NYSE:LLY) and Boehringer Ingelheim's Jardiance Breakthrough Therapy designation as an investigational treatment for adults with heart failure with preserved ejection fraction ((HFpEF)).
- https://seekingalpha.com/news/3738291-eli-lillys-jardiance-wins-fdas-breakthrough-therapy-status-for-heart-failure-indication
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
Crysvita™ (Burosumab Injection)
Ultragenyx Canada Announces Health Canada Approval of a Second Indication for Crysvita™ (Burosumab Injection) for the Treatment of Tumour Induced Osteomalacia in Adults
Sep. 07, 2021 8:00 AM ETUltragenyx Pharmaceutical Inc. (RARE)
OTTAWA, Sept. 07, 2021 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (RARE), a biopharmaceutical company focused on the development and commercialization of novel products for rare and ultra-rare diseases, today announced that Crysvita™ (Burosumab Injection) has been approved by Health Canada for the treatment of Tumour Induced Osteomalacia (TIO) in adults. Crysvita is also approved for the treatment of X-Linked Hypophosphatemia.
TIO is a rare disease that is characterized by the development of tumours that cause weakened and softened bones. The tumours associated with TIO release a protein known as fibroblast growth factor 23 (FGF23) that lowers phosphate levels.
INDICATION FOR TUMOUR INDUCED OSTEOMALACIA (TIO)
CRYSVITA (Burosumab Injection) is indicated for the treatment of fibroblast growth factor 23 (FGF23)–related hypophosphatemia in tumour-induced osteomalacia (TIO) associated with tumours that cannot be curatively resected or localized in adult patients.
For more information on Ultragenyx, please visit the company's website at: www.ultragenyx.com.
https://seekingalpha.com/symbol/RARE
Tezepelumab
New Tezepelumab Data Show 86% Reduction In Exacerbations In Patients With Severe Asthma And Comorbid Nasal Polyps
Improvements Also Demonstrated in Lung Function and Nasal Polyp Symptoms
Exploratory Analysis From NAVIGATOR Phase 3 Trial Presented at European Respiratory Society (ERS) International Congress 2021
NEWS PROVIDED BY
Sep 05, 2021, 07:15 ET
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THOUSAND OAKS, Calif., Sept. 5, 2021 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced new data from the pivotal NAVIGATOR Phase 3 trial demonstrating that tezepelumab reduced exacerbations and improved lung function and nasal symptoms in patients with severe, uncontrolled asthma and comorbid nasal polyps.1 Tezepelumab is a potential first-in-class treatment that acts at the top of the inflammatory cascade by targeting thymic stromal lymphopoietin (TSLP), an epithelial cytokine, and has the potential to treat a broad population of patients with severe asthma.2,3 Tezepelumab is being developed by Amgen in collaboration with AstraZeneca.
The pre-specified exploratory analysis evaluated the effect of tezepelumab in NAVIGATOR patients with or without reported nasal polyps (NP+ or NP−) in the past two years. The analysis showed tezepelumab achieved an 86% reduction in the annualized asthma exacerbation rate (AAER) in NP+ patients (95% CI: 70, 93) and 52% (95% CI: 42, 61) in NP− patients over 52 weeks, compared to placebo when added to standard of care (SoC).1
Tezepelumab improved lung function at week 52 in both groups of patients with an increase in pre-bronchodilator forced expiratory volume in one second (FEV1) of 0.20 L (95% CI: 0.02, 0.37) and 0.13 L (95% CI: 0.08, 0.18) versus placebo in NP+ and NP− patients, respectively.1 Tezepelumab also achieved a clinically relevant improvement in nasal polyp symptoms at week 52, as measured by the Sinonasal Outcome Test (SNOT-22), reducing the SNOT-22 score in NP+ patients by 9.6 points (95% CI: 0.9, 18.2) versus placebo.1,4 The adjusted mean score reductions from baseline for tezepelumab and placebo were 20.10 points (SE: 3.07) and 10.55 points (SE: 2.94). Baseline mean (sd) SNOT-22 score was 49.4 (21.5) and 47.8 (19.0) for tezepelumab and placebo, respectively.1
"This new analysis from NAVIGATOR is exciting for the up to one in five severe asthma patients who have comorbid nasal polyps," said Professor Andrew Menzies-Gow, director of the Lung Division, Royal Brompton Hospital, London, UK, the principal investigator of the NAVIGATOR trial. "The analysis shows tezepelumab's ability to reduce exacerbations, improve lung function and reduce the symptoms of nasal polyps in this comorbid population who are typically more prone to asthma attacks, have an increased likelihood of airway obstruction, and may have a worse quality of life."
Results from the NAVIGATOR Phase 3 trial were published in the New England Journal of Medicine in May 2021.5 A Phase 3 clinical trial, WAYPOINT, has been initiated to explore the efficacy and safety of tezepelumab in adults with severe, chronic rhinosinusitis with nasal polyps.6
About the NAVIGATOR and the PATHFINDER Clinical Trial Program
Building on the Phase 2b PATHWAY trial, the Phase 3 PATHFINDER program included two trials, NAVIGATOR5,23 and SOURCE.24,25 The program includes additional planned mechanistic and long-term safety trials.26 In addition, a Phase 3 clinical trial, WAYPOINT, has been initiated to explore the efficacy and safety of tezepelumab in adults with severe, chronic rhinosinusitis with nasal polyps.6
NAVIGATOR is a Phase 3, randomized, double-blinded, placebo-controlled trial in 1,061 adults (18–80 years old) and adolescents (12–17 years old) with severe, uncontrolled asthma, who were receiving treatment with medium- or high-dose inhaled corticosteroids (ICS) plus at least one additional controller medication with or without OCS. Of the 1,061 randomized patients, 1,059 received either tezepelumab 210 mg (n=528) or placebo (n=531). In total for this pre-specified exploratory analysis, 83 patients had NP in the past 2 years (tezepelumab 210 mg, n=42; placebo, n=41) and 976 did not (tezepelumab 210 mg, n=486; placebo, n=490). NAVIGATOR met the primary endpoint with tezepelumab added to SoC demonstrating a statistically significant and clinically meaningful reduction in the AAER over 52 weeks in the overall patient population, compared to placebo added to SoC. The trial also met the primary endpoint in the subgroup of patients with baseline eosinophil counts less than 300 cells per microliter, with tezepelumab demonstrating a statistically significant and clinically meaningful reduction in AAER in that patient population. Similar reductions in AAER were observed in the subgroup of patients with baseline eosinophil counts less than 150 cells per microliter.26,27
About Tezepelumab
Tezepelumab is being developed by AstraZeneca in collaboration with Amgen (see AstraZeneca and Amgen collaboration below) as an investigational, potential first-in-class human monoclonal antibody that works on the primary source of inflammation: the airway epithelium, which is the first point of contact for viruses, allergens, pollutants and other environmental insults. Specifically, tezepelumab targets and blocks thymic stromal lymphopoietin (TSLP), a key epithelial cytokine that sits at the top of multiple inflammatory cascades and initiates an overreactive immune response to allergic, eosinophilic and other types of airway inflammation associated with severe asthma.2,29
TSLP is released in response to multiple triggers associated with asthma exacerbations, including allergens, viruses and other airborne particles.2,29 Expression of TSLP is increased in the airways of patients with asthma and has been correlated with disease severity.2,3 Blocking TSLP may prevent the release of pro-inflammatory cytokines by immune cells, resulting in the prevention of asthma exacerbations and improved asthma control.2,3 By working at the top of the cascade, tezepelumab helps stop inflammation at the source and has the potential to treat a broad population of severe asthma patients.2,3
About the Amgen and AstraZeneca Collaboration
In 2020, Amgen and AstraZeneca updated the 2012 collaboration agreement for tezepelumab. Both companies will continue to share costs and profits equally after payment by AstraZeneca of a mid-single-digit royalty to Amgen. AstraZeneca continues to lead development and Amgen continues to lead manufacturing. All aspects of the collaboration are under the oversight of joint governing bodies. Under the amended agreement in North America, Amgen and AstraZeneca will jointly commercialize tezepelumab; Amgen will record sales in the U.S. and AstraZeneca will record sales in Canada. Outside the U.S., Amgen will record sales as collaboration revenue.
For more information, visit www.amgen.com
Amgen/AstraZeneca's tezepelumab reduces exacerbations in severe asthma patients
Sep. 07, 2021 5:10 AM ET Amgen Inc. (AMGN), AZN
By: Mamta Mayani, SA News Editor
- Amgen (NASDAQ:AMGN) announces new data from Phase 3 NAVIGATOR trial demonstrating that tezepelumab reduced exacerbations and improved lung function and nasal symptoms in patients with severe, uncontrolled asthma and comorbid nasal polyps. Tezepelumab is being developed in collaboration with AstraZeneca (NASDAQ:AZN).
- https://seekingalpha.com/news/3737395-amgenastrazenecas-tezepelumab-reduces-exacerbations-in-severe-asthma-patients
- https://seekingalpha.com/symbol/AMGN
- https://www.amgen.com/
- https://seekingalpha.com/symbol/AZN
- https://www.astrazeneca.com/
KEYTRUDA® (pembrolizumab)
Merck’s KEYTRUDA® (pembrolizumab) Approved in China in Combination With Chemotherapy for First-Line Treatment of Patients With Locally Advanced Unresectable or Metastatic Esophageal or Gastroesophageal Junction (GEJ) Carcinoma
September 7, 2021 6:45 am ET
First Anti-PD-1 Regimen Approved in China for First-Line Treatment of Advanced Esophageal or GEJ Cancer, Regardless of Histology or PD-L1 Expression
KEYTRUDA Is Now Approved for Eight Indications Across Five Different Types of Cancer in ChinaKENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that KEYTRUDA, Merck’s anti-PD-1 therapy, has been approved by the National Medical Products Administration (NMPA) in China in combination with platinum- and fluoropyrimidine-based chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic carcinoma of the esophagus or gastroesophageal junction (GEJ). This new indication was granted approval based on overall survival (OS) findings from the pivotal Phase 3 KEYNOTE-590 trial. KEYTRUDA is now approved for eight indications across five different types of cancer in China.“In China, esophageal and gastroesophageal junction cancers are leading causes of death, and there have been few treatment advances for patients over the past several decades,” said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. “With this approval of KEYTRUDA plus chemotherapy – the first for an anti-PD-1 regimen in the first-line setting – we can now provide patients with an immunotherapy treatment option earlier in the course of treatment that has been shown to significantly improve survival.”In the global KEYNOTE-590 study, KEYTRUDA in combination with chemotherapy (5-fluorouracil [5-FU] plus cisplatin) demonstrated statistically significant improvements in OS and progression-free survival (PFS) compared with 5-FU and cisplatin alone. KEYTRUDA plus 5-FU and cisplatin reduced the risk of death by 27% (HR=0.73 [95% CI, 0.62-0.86]; p<0.0001). The median OS was 12.4 months (95% CI, 10.5-14.0) for patients treated with KEYTRUDA plus 5-FU and cisplatin and 9.8 months (95% CI, 8.8-10.8) for patients treated with 5-FU and cisplatin alone. KEYTRUDA plus 5-FU and cisplatin reduced the risk of disease progression or death by 35% (HR=0.65 [95% CI, 0.55-0.76]; p<0.0001). The median PFS was 6.3 months (95% CI, 6.2-6.9) for patients treated with KEYTRUDA plus 5-FU and cisplatin and 5.8 months (95% CI, 5.0-6.0) for patients treated with 5-FU and cisplatin alone in all randomized patients. The Chinese package insert notes that the safety of KEYTRUDA plus chemotherapy was investigated in 51 Chinese patients with esophageal carcinoma in KEYNOTE-590, and the safety profile of the Chinese patients was generally consistent with the known safety profiles of KEYTRUDA monotherapy and chemotherapy.“Compared to the rest of the world, China has a disproportionate number of patients who are diagnosed with and who die from esophageal cancer, the fifth most commonly diagnosed cancer and the fourth leading cause of cancer-related death in the country,” said Prof. Shen Lin, vice president, Clinical Oncology, Beijing Cancer Hospital and Peking University, and deputy director, Beijing Institute for Cancer Research. “This approval for KEYTRUDA provides an important new option to patients with advanced esophageal carcinoma in China who currently have limited available treatment options in the first-line setting.”
About KEYNOTE-590KEYNOTE-590 (ClinicalTrials.gov, NCT03189719) is a multicenter, randomized, double-blind, placebo-controlled trial that enrolled 749 patients with locally advanced unresectable or metastatic esophageal or GEJ carcinoma (Siewert type I) who were not candidates for surgical resection or definitive chemoradiation. Patients were randomized (1:1) to receive either KEYTRUDA (200 mg on Day 1 every three weeks) or placebo (on Day 1 every three weeks) in combination with cisplatin (80 mg/m2 on Day 1 every three weeks for up to six cycles) plus 5-FU (800 mg/m2 per day on Days 1 to 5 every three weeks, or per local standard for 5-FU administration, for up to 24 months); all study medications were administered via intravenous infusion.The primary endpoints were OS and PFS, as assessed by the investigator according to RECIST v1.1, in squamous cell histology, PD-L1 expression (CPS ≥10) and in all patients. The secondary endpoints include objective response rate and duration of response, as assessed by the investigator according to RECIST v1.1.
About KEYTRUDA® (pembrolizumab) Injection, 100 mgKEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,500 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.MelanomaKEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.Non-Small Cell Lung CancerKEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is:
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.Head and Neck Squamous Cell CancerKEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS ≥1)] as determined by an FDA-approved test.KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.Classical Hodgkin LymphomaKEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.Primary Mediastinal Large B-Cell LymphomaKEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.Urothelial CarcinomaKEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC):
- who are not eligible for any platinum-containing chemotherapy, or
- who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.Microsatellite Instability-High or Mismatch Repair Deficient CancerKEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.Microsatellite Instability-High or Mismatch Repair Deficient Colorectal CancerKEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).Gastric CancerKEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.Esophageal CancerKEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical CancerKEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.Hepatocellular CarcinomaKEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.Merkel Cell CarcinomaKEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.Renal Cell CarcinomaKEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma.Tumor Mutational Burden-High CancerKEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.Cutaneous Squamous Cell CarcinomaKEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.Triple-Negative Breast CancerKEYTRUDA is indicated for the treatment of patients with high-risk early-stage triple-negative breast cancer (TNBC) in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.https://www.keytruda.com/
For more information, visit www.merck.com
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.View source version on businesswire.com: https://www.businesswire.com/news/home/20210907005361/en/ Source: Merck & Co., Inc.
Merck’s Keytruda combo OK'd in China for first-line treatment of esophageal cancer
Sep. 07, 2021 7:14 AM ETMerck & Co., Inc. (MRK)By: Mamta Mayani, SA News Editor
- Merck's (NYSE:MRK) Keytruda has been approved by the National Medical Products Administration in China in combination with chemotherapy for the first-line treatment of patients with locally advanced unresectable or metastatic carcinoma of the esophagus or gastroesophageal junction (GEJ).
- https://seekingalpha.com/news/3737429-mercks-keytruda-combo-okd-in-china-for-first-line-treatment-of-esophageal-cancer
- https://seekingalpha.com/symbol/MRK
- https://www.keytruda.com/
- https://www.merck.com/
ULTOMIRIS® (ravulizumab)
Ultomiris approved in the EU for children and adolescents with paroxysmal nocturnal haemoglobinuria
PUBLISHED3 September 2021
3 September 2021 07:00 BST
Approval based on interim results from Phase III trial demonstrating complete terminal complement inhibition with reduced dosing frequency compared to Soliris
Ultomiris (ravulizumab) has been approved in the European Union for expanded use to include children (with a body weight of 10 kg or above) and adolescents with paroxysmal nocturnal haemoglobinuria (PNH), an ultra-rare and severe blood disorder characterised by the destruction of red blood cells that can cause thrombosis (blood clots) and result in organ damage and potentially premature death.1-4
The approval by the European Commission was based on interim results from the Phase III clinical trial in children and adolescents with PNH that demonstrated the safety and efficacy of Ultomiris in these patients.
Ultomiris has an established safety and efficacy profile, offering reduced dosing frequency compared to Soliris (eculizumab), which is the current standard of care in the EU for the treatment of children and adolescents with PNH. Ultomiris is administered every four or eight weeks (depending on body weight), following a loading dose. The approval follows the recommendation of the Committee for Medicinal Products for Human Use of the European Medicines Agency in July 2021.
Austin Kulasekararaj, MD, King’s College Hospital, London, UK, said: “PNH is a devastating disease, and Ultomiris provides an advancement for paediatric patients in the EU with an established safety and efficacy profile. By requiring fewer infusions each year than Soliris, Ultomiris may reduce the need for these young patients to miss school to receive treatment.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “This approval of Ultomiris reflects our ongoing commitment to delivering new treatments that can make a meaningful difference in patients’ lives. Ultomiris has become the standard of care for the treatment of adults with PNH and we will make it available to this younger patient population as soon as possible.”
Interim results from the Phase III trial demonstrated Ultomiris was effective in achieving complete C5 complement inhibition through 26 weeks for the treatment of patients up to 18 years of age with PNH. Additionally, Ultomiris had no reported treatment-related severe adverse events, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough haemolysis, which can lead to disabling or potentially fatal blood clots.1 The efficacy and safety profile of Ultomiris in children and adolescents is consistent with the profile of Ultomiris in clinical trials involving adults with PNH and is representative of the broad PNH patient population seen in the real-world clinical setting.5,6
Ultomiris was first approved in the EU in 2019 for the treatment of adults with PNH and is also approved in the EU for the treatment of adults and children with atypical haemolytic uraemic syndrome (aHUS). In June 2021, the US Food and Drug Administration approved the expanded use of Ultomiris to include children (one month of age and older) and adolescents with PNH, the first and only treatment for this age group in the US.
Ultomiris
Ultomiris (ravulizumab), the first and only long-acting C5 complement inhibitor, offers immediate, complete, and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Ultomiris is administered intravenously every eight weeks or, for paediatric patients less than 20 kg, every four weeks, following a loading dose. Ultomiris is approved in the US for the treatment of adults and children (one month of age and older) with PNH; in the EU for adults, as well as for children (with a body weight of 10 kg or above) and adolescents with PNH who experience haemolysis with clinical symptom(s) indicative of high disease activity, as well as for individuals who are clinically stable after having been treated with Soliris for at least the past six months; and in Japan as a treatment for adults with PNH. It is also approved in the US for aHUS to inhibit complement-mediated thrombotic microangiopathy in adult and paediatric (one month of age and older) patients, in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS, as well as in Japan for adults and children with aHUS.
Please visit astrazeneca.com
AstraZeneca's Ultomiris OK'd in EU for children with rare blood disorder
Sep. 03, 2021 6:07 AM ET AstraZeneca PLC (AZN) By: Mamta Mayani, SA News Editor
- AstraZeneca (NASDAQ:AZN) announces that Ultomiris (ravulizumab) has been approved in the European Union (EU) for expanded use to include children and adolescents with paroxysmal nocturnal haemoglobinuria (PNH).
- https://seekingalpha.com/news/3737086-astrazenecas-ultomiris-okd-in-eu-for-children-with-rare-blood-disorder
- https://seekingalpha.com/symbol/AZN
- https://www.ultomiris.com/
- https://www.astrazeneca.com/
BRUKINSA® (Zanubrutinib)
U.S. FDA Grants BRUKINSA® (Zanubrutinib) Approval in Waldenström’s Macroglobulinemia
Sep 01, 2021 5:33 PM
This marks the second FDA approval for BRUKINSA and its third approval in Waldenström’s macroglobulinemia globally
The approval is based on Phase 3 ASPEN trial comparing BRUKINSA against ibrutinib
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that BRUKINSA® (zanubrutinib) has received approval from the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with Waldenström’s macroglobulinemia (WM).
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210901006049/en/
“We are delighted by today’s FDA approval for BRUKINSA in its second indication, offering a new treatment option with demonstrated efficacy and safety benefits for patients with Waldenström’s macroglobulinemia. As shown in the ASPEN trial, BRUKINSA can improve treatment outcomes for these patients and potentially make a positive impact on their lives,” commented Jane Huang, M.D., Chief Medical Officer, Hematology at BeiGene.
The FDA’s approval of BRUKINSA in WM is primarily based on efficacy results from the multicenter, open-label Phase 3 ASPEN trial (NCT03053440) comparing BRUKINSA to ibrutinib in patients with WM. A total of 201 patients with a MYD88 mutation (MYD88MUT) were enrolled in the randomized Cohort 1.
The primary efficacy endpoint of the ASPEN trial was very good partial response (VGPR) rate in the overall intention-to-treat (ITT) population as assessed by independent review committee (IRC). Based on the modified Sixth International Workshop on Waldenström’s Macroglobulinemia (IWWM-6) response criteria (Treon 2015), the VGPR rate was 28% with BRUKINSA, compared to 19% with ibrutinib; based on the IWWM-6 response criteria (Owen et al 2013), the VGPR rate was 16% with BRUKINSA, compared to 7% with ibrutinib.
In the FDA-approved label, the major efficacy outcome is defined as response rate of partial response (PR) or better as assessed by IRC. Based on either IWWM-6 response criteria, the response rate was 78% with BRUKINSA (95% CI: 68, 85), compared to 78% with ibrutinib (95% CI: 68, 86), and the event-free duration of response (DoR) at 12 months was 94% with BRUKINSA (95% CI: 86, 98), compared to 88% with ibrutinib (95% CI: 77, 94).
The most common (≥20%) adverse reactions based on the pooled safety population of 779 patients were decreased neutrophil count, upper respiratory tract infection, decreased platelet count, rash, hemorrhage, musculoskeletal pain, decreased hemoglobin, bruising, diarrhea, pneumonia, and cough.
The recommended dose of BRUKINSA is either 160 mg twice daily or 320 mg once daily, taken orally with or without food. The dose may be adjusted for adverse reactions and reduced for patients with severe hepatic impairment and certain drug interactions.
About BRUKINSA
BRUKINSA is a small molecule inhibitor of Bruton’s tyrosine kinase (BTK) discovered by BeiGene scientists that is currently being evaluated globally in a broad clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. Because new BTK is continuously synthesized, BRUKINSA was specifically designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared to other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease relevant tissues.
BRUKINSA is approved in the following indications and regions:
- For the treatment of mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy (United States, November 2019)*;
- For the treatment of MCL in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) in adult patients who have received at least one prior therapy (China, June 2020)**;
- For the treatment of relapsed or refractory MCL (United Arab Emirates, February 2021);
- For the treatment of Waldenström’s macroglobulinemia (WM) in adult patients (Canada, March 2021);
- Registered and reimbursed for the treatment of MCL in patients who have received at least one prior therapy (Israel, April 2021);
- For the treatment of adult patients with WM who have received at least one prior therapy (China, June 2021)**;
- For the treatment of MCL in adult patients who have received at least one prior therapy (Canada, July 2021);
- For the treatment of adult patients with MCL who have received at least one previous therapy (Brazil, August 2021); and
- For the treatment of adult patients with WM (United States, August 2021).
To-date, more than 30 marketing authorization applications in multiple indications have been submitted in the United States, China, the European Union, and more than 20 other countries or regions.
* This indication was approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
** This indication was approved under conditional approval. Complete approval for this indication may be contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
Please see full U.S. Prescribing Information at https://www.beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at https://www.beigene.com/PDF/BRUKINSAUSPPI.pdf.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210901006049/en/
Source: BeiGene, Ltd.
FDA approves BeiGene's Brukinsa in rare blood cancer
Sep. 02, 2021 4:14 AM ET BeiGene, Ltd. (BGNE) By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) announces that BRUKINSA (zanubrutinib) has received FDA approval for the treatment of adult patients with Waldenström’s macroglobulinemia (WM).
- https://seekingalpha.com/news/3736525-fda-approves-beigenes-brukinsa-in-rare-blood-cancer
- https://seekingalpha.com/symbol/BGNE
- https://www.brukinsa.com/
- https://www.beigene.com/
INVEGA HAFYERA™ (6-month paliperidone palmitate)
Janssen Announces U.S. FDA Approval of INVEGA HAFYERA™
(6-month paliperidone palmitate), First and Only Twice-Yearly Treatment for Adults with Schizophrenia
INVEGA HAFYERA™ offers patients the fewest doses per year for a life less defined by schizophrenia medication
Phase 3 non-inferiority study results showed over 92% of participants were relapse-free at 12 months
Approval is backed by nearly two decades of proven efficacy and safety of Janssen’s long-acting injectable portfolio of schizophrenia medicines
TITUSVILLE, N.J., Sept. 1, 2021 – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced the U.S. Food and Drug Administration (FDA) has approved long-acting atypical antipsychotic INVEGA HAFYERA™ (6-month paliperidone palmitate), the first-and-only twice-yearly injectable for the treatment of schizophrenia in adults. Before transitioning to INVEGA HAFYERA™, patients must be adequately treated with INVEGA SUSTENNA® (1-month paliperidone palmitate) for at least four months, or INVEGA TRINZA® (3-month paliperidone palmitate) for at least one 3-month injection cycle.1
The FDA approval of INVEGA HAFYERA™ is based on the results of a 12-month, randomized, double-blind, non-inferiority Phase 3 global study that enrolled 702 adults (ages 18-70) living with schizophrenia from 20 countries. The results showed non-inferiority of INVEGA HAFYERA™ compared to INVEGA TRINZA® on the primary endpoint of time to first relapse at the end of the 12-month period. Results found that 92.5 percent of patients treated with INVEGA HAFYERA™ and 95 percent treated with INVEGA TRINZA® were relapse-free at 12 months.1 Relapse was defined as psychiatric hospitalization, increase in Positive and Negative Syndrome Scale [PANSS] total score, increase in individual PANSS item scores, self-injury, violent behavior, or suicidal/homicidal ideation.
The safety profile observed in the trial was consistent with previous studies of INVEGA SUSTENNA® and INVEGA TRINZA® with no new safety signals emerging.1 The most common adverse reactions (≥5%) in the INVEGA HAFYERA™ clinical trial were upper respiratory tract infection (12%), injection site reaction (11%), weight increase (9%), headache (7%), and parkinsonism (5%).1
Click-to-Tweet: #BREAKINGNEWS the @US_FDA approved new long-acting injectable treatment with fewer doses a year for adult patients living with #schizophrenia. Click here to learn more: https://bit.ly/3gDSI3j
“Before I found the right treatment plan for me, my symptoms often got in the way of things that I loved to do,” said Patrick, an adult living with schizophrenia and a participant in the clinical trial. “But since my doctor introduced me to Janssen’s long-acting injectable options and my symptoms are controlled, I have the clarity to focus on the present, but also the stability to plan for my future.”
Click-to-Tweet: Patrick, an adult living with schizophrenia, talks about his treatment journey and how a long-acting injectable has helped him stay on track with his goals: https://bit.ly/3gDSI3j
Schizophrenia is a complex and chronic brain disorder in which the symptoms and potential for relapse (or recurrence of symptoms) can impact many aspects of a person’s daily life. On average, an adult with schizophrenia experiences nine relapses in less than six years, often due to missed doses of medication.2 Adults living with schizophrenia and their loved ones face ongoing functional, emotional, and financial burdens. In addition, patients who experience more relapses may have more hospitalizations, which can lead to higher medical costs for patients, hospital systems, and payers.
“For too long, we’ve accepted relapse as a normal part of living with schizophrenia, while research continues to demonstrate that stronger medication adherence means better patient outcomes,” said Gustavo Alva*, M.D., DFAPA, Medical Director at ATP Clinical Research and 6-month paliperidone palmitate clinical trial investigator. “The Phase 3 trial results provide compelling evidence that 6-month paliperidone palmitate offers longer-term symptom control with the fewest doses per year, which may support greater patient adherence.”
Recently, the National Council for Mental Wellbeing and the American Psychiatric Association updated their schizophrenia treatment guidance and guidelines to expand the recommended use of long-acting injectables for appropriate adult patients living with schizophrenia.3,4,5
INVEGA HAFYERA™ is a long-acting injectable treatment that is administered by a healthcare provider in the upper buttocks area every six months. INVEGA HAFYERA™ dissolves slowly into the bloodstream after injection, resulting in continuous treatment and symptom control over six months.1
“Long-acting injectable treatments offer a number of advantages compared to oral medication for schizophrenia, including relief from needing to remember to take medication daily, lower discontinuation rates, and sustained treatment over longer periods,” said Bill Martin, Ph.D., Global Therapeutic Area Head, Neuroscience, Janssen Research & Development, LLC. “Today’s approval enables us to rethink how we manage this chronic disease by offering patients and caregivers the potential for a life less defined by schizophrenia medication.”
Please click here to read the full Prescribing Information, including Boxed WARNING, for INVEGA HAFYERA™.
INDICATION
INVEGA HAFYERA™ is a prescription medicine given by injection by a healthcare provider 1 time every 6 months and used for the treatment of schizophrenia in adults who have been adequately treated with either:
- A 1-time-each-month paliperidone palmitate extended-release injectable suspension for at least 4 months
- A 1-time-every-3-months paliperidone palmitate extended-release injectable suspension for at least 3 months
Learn more at www.janssen.com.
Johnson & Johnson wins FDA approval for long-acting schizophrenia med
Sep. 01, 2021 8:45 AM ET Johnson & Johnson (JNJ) By: Dulan Lokuwithana, SA News Editor1 Comment
- The Janssen Pharmaceutical Companies, a unit of Johnson & Johnson (NYSE:JNJ) announced that the FDA approved its long-acting atypical antipsychotic INVEGA HAFYERA for adults with schizophrenia.
- INVEGA HAFYERA (6-month paliperidone palmitate) is the first and only twice-yearly treatment available for schizophrenia, according to the company.
- https://seekingalpha.com/news/3736021-johnson-johnson-wins-fda-approval-for-long-acting-schizophrenia-med
- https://seekingalpha.com/symbol/JNJ
VOXZOGO® (vosoritide)
European Commission Approves BioMarin's VOXZOGO® (vosoritide) for the Treatment of Children with Achondroplasia from Age 2 Until Growth Plates Close
Achondroplasia is the Most Common Cause of DwarfismFirst Medicine Approved to Treat Children with Achondroplasia in EuropeAug 27, 2021
SAN RAFAEL, Calif., Aug. 27, 2021 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced today that the European Commission (EC) has granted marketing authorization for VOXZOGO® (vosoritide), a once daily injection to treat achondroplasia in children from the age of 2 until growth plates are closed, which occurs after puberty when children reach final adult height. Voxzogo is the first medicine to be approved to treat children with achondroplasia in Europe. Voxzogo, a modified C-type natriuretic peptide (CNP), directly targets the underlying pathophysiology of achondroplasia by down regulating fibroblast growth factor receptor 3 (FGFR3) signaling and consequently promoting endochondral bone formation.
"Today represents an important milestone for the European achondroplasia community. For the first time medical professionals in Europe can offer a meaningful targeted therapeutic treatment option for children and families affected by achondroplasia," said Klaus Mohnike, Professor of Paediatrics at Magdeburg University Hospital in Germany and investigator for the Voxzogo clinical program. "Achondroplasia is a serious, progressive, and lifelong condition, which can cause multi-system complications that in some cases require surgical intervention. This regulatory approval is based on improved height gain, one important determinant of day-to-day function for people with achondroplasia, and is a first step to understand the potential benefits of Voxzogo beyond height over the long term."
It is estimated that over 11,000 children across Europe, Middle East, and Africa are affected by achondroplasia and could be eligible for treatment with Voxzogo. Approximately a third of this population are in countries authorized under the EMA license. Also, the French National Agency for Medicines and Health Products Safety (ANSM) granted an Autorisation Temporaire d'Utilisation de cohorte (ATU cohort), or Temporary Authorization for Use to allow access of Voxzogo to begin immediately under an authorized process. An ATU allows access to drugs not yet approved in France, when provided for rare diseases with no alternative options, and when the benefit/risk is presumed positive. The list price in France under the ATU process is 712€ per vial and constitutes flat vial pricing across the spectrum of ages and weights translating to an estimated annual per patient cost of approximately 260,00€ or $300,000, assuming 100% compliance and excluding any discounts. We expect the initial German list price to be consistent with the French ATU price. The Company expects that these list prices will be subject, in one to two years, to material discounts after reimbursement negotiations in key markets such as Germany, France and Italy.
The EC based its decision on the totality of data from the Voxzogo clinical development program including the outcomes from the randomized, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of Voxzogo. The Phase 3 Study was further supported by the ongoing long-term safety and efficacy from the Phase 2 dose-finding study, which showed that growth rates have been sustained above participants' baseline rates and above the expected annualized growth velocity for untreated children with achondroplasia throughout the five-year observation period for which data are currently available. No acceleration of bone age was observed, suggesting that Voxzogo is not reducing the total duration of growth. The data package included results from an ongoing Phase 2 randomized double-blind study in infants and young children, including extensive pharmacokinetic and biomarker data, as well as preliminary growth data from participants in the 2 to 5-year age cohort. Data in sentinel study participants showed a positive effect on growth following two years of Voxzogo treatment in subjects aged 2 to 5 years. In addition, the data package included data from the Phase 3 extension study and extensive natural history data.
The U.S. New Drug Application (NDA) for Voxzogo is under review by the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of November 20, 2021. The Company successfully closed out the in-person FDA pre-approval inspection of its manufacturing facilities for Voxzogo earlier this year.
Regulatory Status
In January 2021, the Company received notice from the FDA that the NDA for Voxzogo had been granted Priority Review Designation based on the serious pediatric indication it addresses, and the lack of treatment options currently available. Consistent with FDA's policy on changes to review classification for an ongoing application review, the PDUFA action date is not affected by this designation. If approved, the Voxzogo NDA may qualify for a Priority Review Voucher (PRV). A PRV confers priority review to a subsequent drug application that would not otherwise qualify for that designation. The rare pediatric disease review voucher program is designed to encourage development of new drugs and biologics for the prevention or treatment of rare pediatric diseases.
Voxzogo received orphan drug designation from the FDA and EMA for the treatment of children with achondroplasia. The Orphan Drug Designation program is intended to advance the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions.
For additional information, please visit www.biomarin.com
European Commission approves Biomarin's achondroplasia treatment Voxzogo
Aug. 27, 2021 9:15 AM ET BioMarin Pharmaceutical Inc. (BMRN)
By: Aakash Babu, SA News Editor4 Comments
- The European Commission (EC) has granted marketing authorization for BioMarin Pharmaceutical's (NASDAQ:BMRN) Voxzogo (vosoritide) to treat certain children with achondroplasia, a common form of skeletal dysplasia leading to disproportionate short stature.
- https://seekingalpha.com/news/3734858-european-commission-approves-biomarins-achondroplasia-treatment-voxzogo
- https://seekingalpha.com/symbol/BMRN
- https://www.biomarin.com/our-treatments/pipeline/vosoritide-for-achondroplasia/
- https://www.biomarin.com/
FLUMIST® QUADRIVALENT (Influenza Vaccine Live, Intranasal)
FLUMIST® QUADRIVALENT (Influenza Vaccine Live, Intranasal) Vaccine Now Available in the US for Annual Flu Season
Needle-free vaccine option can help reduce influenza infections
The Centers for Disease Control and Prevention (CDC) recommends influenza vaccines as the primary protection against the flu virus to reduce the burden on the US healthcare system
August 31, 2021 07:00 AM Eastern Daylight Time
WILMINGTON, Del.--(BUSINESS WIRE)--AstraZeneca today announced that FLUMIST® QUADRIVALENT (Influenza Vaccine Live, Intranasal) doses are now available in the US for the 2021-2022 influenza season. FLUMIST QUADRIVALENT, the only Food and Drug Administration (FDA)-approved nasal-spray flu vaccine, is indicated for people ages 2 through 49. The bulk of AstraZeneca’s supply for the 2021-2022 flu season is expected to be available by mid-September.
In line with the 2021-2022 recommendations from the CDC’s Advisory Committee on Immunization Practices (ACIP) and the American Academy of Pediatrics (AAP), FLUMIST QUADRIVALENT continues to be an option for flu vaccination in the US this season. Vaccines may not prevent influenza in everyone who gets vaccinated.
The CDC continues to stress the importance of receiving a yearly flu vaccine, citing vaccination as the most effective protection against seasonal flu infection. Additionally, they reinforce that flu vaccination during the 2021-2022 season will help protect the public against preventable illness, as well as help alleviate burden on the healthcare system.
“As the world continues to navigate an unpredictable public health landscape with coinciding flu and COVID-19 viruses, AstraZeneca remains committed to following the science and putting people and their communities first by helping ensure families have access to a needle-free vaccination option,” said Mina Makar, Senior Vice President, US Respiratory & Immunology at AstraZeneca. “Improving patient and public health is at the heart of what we do, and we are proud to be doing our part in fighting the flu again this season by working with our partners to provide FLUMIST QUADRIVALENT to those eligible this year.”
Since FLUMIST QUADRIVALENT is the only FDA-approved flu vaccine to use a needle-free nasal spray administrative technique, it may be preferred by eligible patients. AstraZeneca’s live attenuated influenza vaccine (LAIV) (either as trivalent or quadrivalent formulations) has been approved by the FDA since 2003 and is covered by most health insurance plans. The most common side effects of FLUMIST QUADRIVALENT are runny or stuffy nose, sore throat, and fever over 100°F.
Please note, all available influenza vaccines are manufactured differently and different preparations have different indications as licensed by the FDA. Those interested in receiving a flu vaccine are encouraged to speak with a healthcare provider to determine if FLUMIST QUADRIVALENT is right for them and can use an online vaccine locator tool to find where it is available in their local area.
INDICATION
FLUMIST QUADRIVALENT is a vaccine indicated for active immunization of persons 2-49 years of age for the prevention of influenza disease caused by influenza A subtype viruses and type B viruses contained in the vaccine. FLUMIST QUADRIVALENT is for intranasal administration only.
Please see full Prescribing Information for FLUMIST QUADRIVALENT, including Patient Information.
For more information, please visit www.astrazeneca-us.com
AstraZeneca FluMist vaccine now available in the U.S.
Aug. 31, 2021 9:17 AM ET AstraZeneca PLC (AZN) By: Jonathan M Block, SA News Editor
- The FluMist Quadrivalent intranasal influenza vaccine from AstraZeneca (NASDAQ:AZN) is now available in the U.S. for the 2021-2022 flu season.
- The vaccine is approved from ages 2-49.
- https://seekingalpha.com/news/3735639-astrazeneca-flumist-vaccine-now-available-in-the-us
- https://seekingalpha.com/symbol/AZN
- https://www.astrazeneca.com/
- https://www.flumistquadrivalent.com/
biotecMAX™ April/May/June/July/Aug 2021 Insight
Repatha® (evolocumab)
New Data At ESC Congress 2021 Shows Repatha® (evolocumab) Improves Features Of Plaque Stability In Patients With Acute Coronary Syndrome (ACS)
New Positive Phase 3 Data for Repatha in ACS Patients who Initiated Treatment in the Acute Setting
Repatha in Addition to Optimized Statin Therapy Improves Features of Plaque Morphology Twice as Well as Statins Alone
Results May Provide Mechanistic Insight for Cardiovascular Event Reduction as Seen in Atherosclerotic Cardiovascular Disease Patients in the FOURIER Study
THOUSAND OAKS, Calif., Aug. 27, 2021 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced positive data from the HUYGENS Phase 3 study showing that Repatha® (evolocumab) in addition to optimized statin therapy, in comparison with optimized statin therapy alone, significantly improved features of plaque stability in patients with coronary artery disease (CAD). These data are being presented during an oral presentation at ESC Congress 2021, organized by the European Society of Cardiology, Aug. 27-30.
The HUYGENS study met its primary endpoint, with Repatha in addition to optimized statin therapy increasing fibrous cap thickness by 42.7 um in comparison with an increase of 21.5 um (75% increase versus 39%) on optimized statin therapy alone (p=0.01), as measured by optical coherence tomography (OCT). Thus, the addition of Repatha improved this feature twice as well as statins alone. Repatha also improved all of the study's secondary endpoints, including decreasing the maximum lipid arc by -57.5° versus -31.4° (p=0.01), as measured by OCT.
"The majority of acute coronary syndrome events are caused by plaque rupture, and those who have had a heart attack are especially vulnerable to additional episodes of plaque rupture, demonstrating the importance of maintaining the thickness of the fibrous cap to help stabilize plaques," said Stephen J. Nicholls, M.D., Ph.D., professor of Cardiology and director, Monash University Victorian Heart Institute, Melbourne, Australia and first author of the HUYGENS study. "These encouraging results reaffirm the potential of Repatha and highlight the benefits of Repatha in ACS patients who initiated treatment early."
Results from the randomized, double-blind 52-week study in ACS patients on optimized statin therapy demonstrate that Repatha treatment, initiated within a week after the ACS event, reduced LDL-C from 140 to 28 mg/dL (-80%) versus reductions from 142 to 87 mg/dL (-39%) with statin optimization alone. No new safety risks were identified. The most common treatment-emergent adverse events (>3%) were angina pectoris, myalgia, hypertension, diarrhea, fatigue and cough.
"Amgen continues to build a body of evidence to support the clinical profile of Repatha and demonstrate its benefit in patients at elevated risk of suffering another heart attack or stroke," said David M. Reese, M.D., executive vice president of Research and Development at Amgen. "This study builds on the findings from the GLAGOV study and provides evidence that low LDL-C levels can change characteristics of coronary plaque, which may explain the biology of cardiovascular event reduction we saw in the FOURIER study."
While HUYGENS did not evaluate cardiovascular outcomes, the results build on the growing body of evidence already supporting the clinical profile of Repatha. The HUYGENS study results add relevant insights to the science of plaque biology and contribute to our understanding of the important benefits of initiating Repatha after a heart attack. Fifty clinical trials, conducted with over 47,000 patients randomized to Repatha or placebo, have demonstrated the clinical benefits of Repatha, which include reduction in myocardial infarction and stroke, rapid (within four weeks) and dramatic LDL-C lowering over the long term (median 2.2 years), and consistent safety over a five-year treatment period generally consistent with the FOURIER study.7
About the Data
Previous studies include GLAGOV which showed Repatha, when added to optimal statin therapy, reduced plaque burden by decreasing plaque atheroma volume in patients with CAD.8 This was the first study to demonstrate that lowering LDL-C levels through PCSK9 inhibition reduces atherosclerotic plaque burden.
HUYGENS demonstrated that Repatha in addition to optimized statin therapy, in comparison with optimized statin therapy alone, significantly improved a key feature of plaque stability in patients with CAD by increasing the fibrous cap thickness. HUYGENS may offer mechanistic insight for the CV event reduction seen in the FOURIER outcomes study.9
For more information, visit www.amgen.com
About Repatha® (evolocumab)
Repatha is a human monoclonal antibody that inhibits proprotein convertase subtilisin/kexin type 9 (PCSK9). Repatha binds to PCSK9 and inhibits circulating PCSK9 from binding to the low-density lipoprotein (LDL) receptor (LDLR), preventing PCSK9-mediated LDLR degradation and permitting LDLR to recycle back to the liver cell surface. By inhibiting the binding of PCSK9 to LDLR, Repatha increases the number of LDLRs available to clear LDL from the blood, thereby lowering LDL-C levels.
Repatha is approved in 76 countries, including the U.S., Japan, China and in all 27 countries that are members of the European Union. Applications in other countries are pending.
Please see full Prescribing Information.
View original content to download multimedia:https://www.prnewswire.com/news-releases/new-data-at-esc-congress-2021-shows-repatha-evolocumab-improves-features-of-plaque-stability-in-patients-with-acute-coronary-syndrome-acs-301364057.html
SOURCE Amgen
Amgen's Repatha improves features of plaque stability in acute coronary syndrome patients
Aug. 27, 2021 4:59 AM ET Amgen Inc. (AMGN) By: Mamta Mayani, SA News Editor
- Amgen (NASDAQ:AMGN) announces positive data from the HUYGENS Phase 3 study showing that Repatha (evolocumab) in addition to optimized statin therapy, in comparison with optimized statin therapy alone, significantly improved features of plaque stability in patients with coronary artery disease (CAD).
- https://seekingalpha.com/news/3734734-amgens-repatha-improves-features-of-plaque-stability-in-acute-coronary-syndrome-patients
- https://seekingalpha.com/symbol/AMGN
- https://www.repatha.com/
- https://www.amgen.com/
TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor)
PDF Version Aug 26, 2021
Vertex Announces Publication in The New England Journal of Medicine of Phase 3 Results for TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in People With Cystic Fibrosis
BOSTON--(BUSINESS WIRE)--Aug. 26, 2021-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced publication in The New England Journal of Medicine (NEJM) of results from a Phase 3 study of TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in people with cystic fibrosis (CF) ages 12 years and older who have one copy of the F508del mutation and one gating (F/G) or residual function (F/RF) mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The manuscript includes data on primary and key secondary endpoints, which were previously reported and showed statistically significant and clinically meaningful improvements in lung function and sweat chloride, when compared to active control (either ivacaftor or tezacaftor/ivacaftor), as well as more detailed efficacy and safety data, including subgroup efficacy analyses.
“This study is the third of three Phase 3 clinical trials in the TRIKAFTA program in the 12 years and older age group. Consistent with the prior outcomes, these results show clinically meaningful improvements in pulmonary function, sweat chloride and Cystic Fibrosis Questionnaire-Revised (CFQ-R) respiratory domain scores," said Carmen Bozic, M.D., Executive Vice President and Chief Medical Officer, Vertex. “These results are especially notable given that all patients were treated with a CFTR modulator prior to initiating TRIKAFTA.”
“The outcomes within this study, in particular those from the subgroup efficacy analysis by F/G and F/RF, are remarkable because they demonstrate additional benefit on top of standard of care and build further confidence for clinicians to treat people with CF who may have these mutations,” said Steven Rowe, M.D., Director, Gregory Fleming James Cystic Fibrosis Research Center, University of Alabama at Birmingham.
Study 445-104
The data published today are from a global Phase 3, randomized, double-blind, parallel-group study. All patients had a 4-week run-in period of either ivacaftor or tezacaftor/ivacaftor. Following the run-in, 258 patients were randomized to receive TRIKAFTA® or to remain on their prior regimen of ivacaftor or tezacaftor/ivacaftor for 8 weeks. Baseline was measured at the end of the run-in period, prior to the start of the 8-week treatment period. TRIKAFTA® improved the percent predicted forced expiratory volume in 1 second (ppFEV1) by 3.7 percentage points (95% CI, 2.8 to 4.6; P<0.001) from baseline and by 3.5 percentage points (95% CI, 2.2 to 4.7; P<0.001) vs. active control and improved sweat chloride concentration by ‑22.3 mmol/liter (95% CI, ‑24.5 to ‑20.2; P<0.001) from baseline and by ‑23.1 mmol/liter (95% CI, ‑26.1 to ‑20.1; P<0.001) vs. active control. The change in the CFQ-R respiratory domain score was +10.3 points from baseline (95% CI, 8.0 to 12.7) and +8.7 points vs. active control (95% CI, 5.3 to 12.1). Subgroup analyses of patients with F/G and F/RF genotypes are also included in the manuscript. Safety data were consistent with those observed in previous Phase 3 studies with TRIKAFTA®.
INDICATION AND IMPORTANT SAFETY INFORMATION FOR TRIKAFTA® (elexacaftor/tezacaftor/ivacaftor and ivacaftor) TABLETS
What is TRIKAFTA?
TRIKAFTA is a prescription medicine used for the treatment of cystic fibrosis (CF) in patients aged 6 years and older who have at least one copy of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene or another mutation that is responsive to treatment with TRIKAFTA. Patients should talk to their doctor to learn if they have an indicated CF gene mutation. It is not known if TRIKAFTA is safe and effective in children under 6 years of age.
For company updates and to learn more about Vertex's history of innovation, visit www.vrtx.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210826005091/en/
Source: Vertex Pharmaceuticals Incorporated
https://seekingalpha.com/symbol/VRTX
FINTEPLA® (Fenfluramine)
Zogenix Receives Orphan Drug Designation for FINTEPLA® (Fenfluramine) in Japan
08.26.21PDF Version
- Zogenix plans to submit a new drug application (J-NDA) for FINTEPLA in Japan for the treatment of seizures associated with Dravet syndrome by year end.
- Dravet syndrome is a rare, life-long form of epilepsy marked by severe seizures, significant developmental delays, frequent hospitalizations and medical emergencies, and increased risk of premature death.
- Seizures associated with Dravet syndrome are highly resistant to existing anti-epileptic medicines, pointing to a major need for new treatment options.
EMERYVILLE, Calif., Aug. 26, 2021 (GLOBE NEWSWIRE) -- Zogenix (NASDAQ: ZGNX), a global biopharmaceutical company developing rare disease therapies, today announced that the Japanese Ministry of Health, Labour & Welfare (MHLW) has granted Orphan Drug Designation to FINTEPLA® (fenfluramine) oral solution, which Zogenix is developing in Japan as a potential treatment for seizures associated with Dravet syndrome.
Dravet syndrome is a rare infant- and childhood-onset epilepsy marked by frequent and severe treatment-resistant seizures, associated hospitalizations and medical emergencies, significant developmental and motor impairments, and an increased risk of sudden premature death.
The MHLW accreditation of Orphan Drug Designation follows the Japanese Pharmaceutical Affairs & Food Sanitation Council’s (PAFSC) First Committee on Drugs agreement on July 28 to grant orphan drug designation for FINTEPLA for Dravet syndrome. The PAFSC is organized under the MHLW and consists of several expert committees from various fields, who serve as the decision-making body for drug approval (J-NDA, s-NDA) as well as Orphan Drug designation. In Japan, Orphan Drug Designation may be granted to drug candidates designed to treat diseases with fewer than 50,000 patients or diseases that are designated as intractable and the need for improved medical care is high.
Zogenix is on track to submit a J-NDA for FINTEPLA for the treatment of seizures associated with Dravet syndrome later this year. If approved, the product will be made available in Japan through an exclusive distribution agreement with Nippon Shinyaku, Co., Ltd., a leading Japanese pharmaceutical product developer and distributor.
“The orphan drug designation of FINTEPLA in Japan is an important milestone in our mission to meet the unmet needs of severe, rare epilepsy patients around the world,” said Stephen J. Farr, Ph.D., President and Chief Executive Officer of Zogenix. “We are proud to have worked with patients, physicians, and regulators in Japan to achieve this milestone and reinforce our commitment to working with the MHLW to bring this much-needed potential treatment option to patients and families living with Dravet syndrome in Japan.”
The incidence of Dravet syndrome in Japan is estimated to be 1 in 20,000-40,000. There are an estimated 3,000 Dravet syndrome patients in Japan based on the Ministry of Health, Labour and Welfare (MHLW) Patient Survey.
About FINTEPLA®
FINTEPLA is a prescription medicine used to treat the seizures associated with Dravet syndrome in patients 2 years of age and older. FINTEPLA possesses dual activities to inhibit seizures: as a serotonergic agent, acting as a potent 5-HT releaser with agonist activity at 5-HT1D, 2A, and 2C receptors, and as a positive modulator of Sigma1R. Fenfluramine is approved in the United States and Europe and is in development in Japan for the treatment of seizures associated with Dravet syndrome.
Across multiple clinical studies, FINTEPLA demonstrated significant and sustained reduction of convulsive seizures associated with Dravet syndrome. In two pivotal Phase 3 trials, the reduction in convulsive seizure frequency per 28 days was statistically significantly greater for all dose groups of FINTEPLA compared to placebo. Please see important FINTEPLA prescribing and safety information at www.fintepla.com.
Zogenix gets orphan drug status for FINTEPLA in Japan
Aug. 26, 2021 8:25 AM ETZogenix, Inc. (ZGNX)By: Aakash Babu, SA News Editor3 Comments
- Zogenix (NASDAQ:ZGNX) announces that the Japanese Ministry of Health, Labour & Welfare (MHLW) has granted Orphan Drug Designation to FINTEPLA (fenfluramine) oral solution, which is being developed in Japan as a potential treatment for seizures associated with Dravet syndrome.
- https://seekingalpha.com/news/3734418-zogenix-gets-orphan-drug-status-for-fintepla-in-japan
- https://seekingalpha.com/symbol/ZGNX
- https://www.finteplahcp.com/
- https://www.zogenix.com/
Opdivo® (nivolumab)
U.S. Food and Drug Administration Approves Opdivo® (nivolumab) for the Adjuvant Treatment of Patients with High-Risk Urothelial Carcinoma
08/20/2021CATEGORY:
In CheckMate -274, Opdivo nearly doubled median disease-free survival compared to placebo in the intent-to-treat population1
Opdivo is now approved in earlier stages of disease for three types of cancer, including the first and only PD-1 inhibitor approved for urothelial carcinoma in the adjuvant setting
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that Opdivo®(nivolumab) 240 mg every two weeks or 480 mg every four weeks (injection for intravenous use) was approved by the U.S. Food and Drug Administration (FDA) for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection, regardless of prior neoadjuvant chemotherapy, nodal involvement or PD-L1 status.1 The approval is based on the Phase 3 CheckMate -274 trial, which compared Opdivo 240 mg (n=353) to placebo (n=356).1 This application was approved under the FDA’s Real-Time Oncology Review (RTOR) pilot program, which aims to ensure that safe and effective treatments are available to patients as early as possible.2
In the trial, among patients who received Opdivo, median disease-free survival (DFS) was nearly twice as long as in those who received placebo (20.8 months [95% Confidence Interval (CI): 16.5 to 27.6] versus 10.8 months [95% CI: 8.3 to 13.9]).1Opdivo reduced the risk of disease recurrence or death by 30% compared to placebo (Hazard Ratio [HR] 0.70, 95% CI: 0.57 to 0.86; P=0.0008).1 Among patients whose tumors express PD-L1 ≥1%, median DFS was not reached (95% CI: 21.2 to NE; n=140) for those who received Opdivo versus 8.4 months (95% CI: 5.6 to 21.2; n=142) for placebo; Opdivo reduced the risk of disease recurrence or death by 45% (HR 0.55, 95% CI: 0.39 to 0.77; P=0.0005).1
“This approval is a major milestone for patients who have undergone major surgery to remove the bladder or parts of the urinary tract and are in need of additional treatment approaches that can help reduce the risk of their UC returning,” said Matthew D. Galsky,* M.D., a CheckMate -274 primary investigator and Professor of Medicine, Director of Genitourinary Medical Oncology, Co-Director of the Center of Excellence for Bladder Cancer, and Associate Director for Translational Research at The Tisch Cancer Institute and the Icahn School of Medicine at Mount Sinai.3 “Nivolumabprovides a new FDA-approved treatment shown to reduce the risk of disease recurrence or death based on the safety and efficacy findings from CheckMate -274, and has the potential to become a new standard of care option in this setting.”1
About CheckMate -274
CheckMate -274 is a randomized, double-blind, placebo-controlled, multi-center trial evaluating Opdivo as an adjuvant treatment in patients who had undergone radical resection of urothelial carcinoma (UC) originating in the bladder or upper urinary tract and were at high risk of recurrence.1 The UC pathologic staging criteria that defines high risk patients was ypT2-ypT4a or ypN+ for patients who received neoadjuvant cisplatin chemotherapy or pT3-pT4a or pN+ for patients who did not receive neoadjuvant cisplatin and who also either were ineligible for or refused adjuvant cisplatin chemotherapy.1
Patients were randomized (n=353 and n=356 to the Opdivo and placebo arms, respectively) to receive Opdivo 240 mg or placebo by intravenous infusion over 30 minutes every two weeks until recurrence or unacceptable toxicity for a maximum treatment duration of one year.1 Eligible patients were randomized in a 1:1 ratio to Opdivo or placebo and were stratified by pathologic nodal status (N+ vs. N0/x with <10 nodes removed vs. N0 with ≥10 nodes removed), tumor cells expressing PD-L1 (≥1% vs. <1%/indeterminate as determined by the central lab using the PD L1 IHC 28-8 pharmDx assay), and use of neoadjuvant cisplatin (yes vs. no).1 The major efficacy outcome measures were investigator-assessed DFS in all randomized patients and in patients with tumors expressing PD-L1 ≥1%.1 DFS was defined as time to first recurrence (local urothelial tract, local non-urothelial tract, or distant metastasis), or death.1 Additional efficacy outcome measures included overall survival.1 The FDA-approved dosing for Opdivo is 240 mg every two weeks (30-minute intravenous infusion) or 480 mg every four weeks (30-minute intravenous infusion) until disease recurrence or unacceptable toxicity for up to one year.1
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the adjuvant treatment of patients with urothelial carcinoma (UC) who are at high risk of recurrence after undergoing radical resection of UC.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
For more information about Bristol Myers Squibb, visit us at BMS.com
Bristol Myers wins FDA approval for Opdivo as adjuvant therapy for bladder cancer
Aug. 20, 2021 12:09 PM ET Bristol-Myers Squibb Company (BMY) By: Dulan Lokuwithana, SA News Editor
- Bristol Myers Squibb (BMY +1.0%) announced that the FDA approved Opdivo (nivolumab) for the adjuvant treatment of patients with urothelial carcinoma (UC) who have a high risk of cancer return after surgical resection.
- https://seekingalpha.com/news/3732759-bristol-myers-wins-fda-approval-for-opdivo-as-adjuvant-therapy-for-bladder-cancer
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
RINVOQ® (upadacitinib)
August 24, 2021
European Commission Approves RINVOQ® (upadacitinib) as First JAK Inhibitor in the European Union for the Treatment of Both Adults and Adolescents with Moderate to Severe Atopic Dermatitis
- Approval supported by data from one of the largest registrational Phase 3 programs in atopic dermatitis evaluating RINVOQ (upadacitinib) monotherapy or with topical corticosteroids[1]
- RINVOQ met all primary and secondary endpoints, demonstrating rapid and significant improvement in skin clearance and itch reduction compared to placebo at week 16 and earlier time points (p<0.001)[1]
- Results at week 16 continued to be maintained through week 52[1]
- RINVOQ has a safety profile supported by clinical programs evaluating more than 10,500 patients* across approved indications, and on-market experience in rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis[2-9]
- Milestone marks the fourth EC-approved indication for RINVOQ[1]
NORTH CHICAGO, Ill., Aug. 24, 2021 /PRNewswire/ -- AbbVie (NYSE: ABBV) today announced the European Commission (EC) approved RINVOQ® (upadacitinib), an oral, selective and reversible JAK inhibitor, for the treatment of moderate to severe atopic dermatitis in adults and adolescents 12 years and older who are candidates for systemic therapy.1 The recommended dose of RINVOQ for atopic dermatitis in adults is 15 mg or 30 mg once daily based on individual patient presentation, and 15 mg once daily for adolescents (12-17 years of age) and adults 65 years and older.1 RINVOQ can be used with or without topical corticosteroids (TCS).1
"This is a significant milestone for AbbVie in our pursuit to transform care in atopic dermatitis," said Michael Severino, M.D., vice chairman and president, AbbVie. "We are excited to provide an additional treatment option in Europe to help alleviate the burden of unrelenting itch and rash that many of these patients struggle with in daily life, despite available treatment options."
The EC approval is supported by data from one of the largest registrational Phase 3 programs in atopic dermatitis with more than 2,500 adults and adolescents with moderate to severe disease.1 These studies evaluated the efficacy and safety of RINVOQ monotherapy (Measure Up 1 [MU1] and Measure Up 2 [MU2]) and with topical corticosteroids (AD Up [AU]) compared to placebo.1 In all three studies, the co-primary endpoints were at least a 75 percent improvement in the Eczema Area and Severity Index (EASI 75) and validated Investigator's Global Assessment for Atopic Dermatitis (vIGA-AD) score of 0/1 (clear or almost clear) at week 16.1
Highlights From the Global Phase 3 Atopic Dermatitis Clinical Trial Program1
Across the Phase 3 studies, all primary and secondary endpoints were met with 15 mg and 30 mg doses of RINVOQ compared to placebo. Highlights include:
- Significantly more patients achieved EASI 75 at week 16 in the RINVOQ 15 mg group (MU1: 70%; MU2: 60%; AU: 65%) and the RINVOQ 30 mg group (MU1: 80%; MU2: 73%; AU: 77%), compared to placebo (MU1: 16%; MU2: 13%; AU: 26%).
- Significantly more patients achieved vIGA-AD 0/1 at week 16 in the RINVOQ 15 mg group (MU1: 48%; MU2: 39%; 40: 31%) and the RINVOQ 30 mg group (MU1: 62%; MU2: 52%; AU: 59%) compared to placebo (MU1: 8%; MU2: 5%; AU: 11%).
- Significantly more patients achieved clinically meaningful itch reduction (improvement in Worst Pruritus NRS ≥4) in the RINVOQ 15 mg group (MU1: 52%; MU2: 42%; AU: 52%) and the RINVOQ 30 mg group (MU1: 60%; MU2: 60%; AU: 64%) compared to placebo (MU1: 12%; MU2: 9%; AU: 15%) at week 16.
- Clinically meaningful itch reduction (improvement in Worst Pruritus NRS ≥4) and skin clearance (EASI 75) were observed as early as week 1 and week 2, respectively, in patients treated with either dose of RINVOQ compared to those treated with placebo.
- Results at week 16 continued to be maintained through week 52 in patients treated with either dose of RINVOQ.
The most commonly reported adverse reactions (≥5% of patients) with RINVOQ 15 mg or 30 mg were upper respiratory tract infection (25.4%), acne (15.1%), herpes simplex (8.4%), headache (6.3%) and increased blood creatine phosphokinase (CPK; 5.5%).1 The most common serious adverse reactions were serious infections (<1.0%).1
The Marketing Authorization means that RINVOQ is approved in all member states of the European Union, as well as Iceland, Liechtenstein, Norway and Northern Ireland. RINVOQ is already approved for the treatment of moderate to severe atopic dermatitis in Russia, Saudi Arabia, United Arab Emirates, New Zealand and Chile, and is currently under review in the U.S. by the Food and Drug Administration (FDA).
*10,500 patients includes all patients across all arms (active treatment and placebo) in 8 Phase 3 trials in rheumatoid arthritis, 2 in psoriatic arthritis, 1 in ankylosing spondylitis and 5 in atopic dermatitis.2-9 This includes 344 adolescent patients (aged 12 to 17 years) in the Phase 3 Measure Up 1, Measure Up 2 and, AD Up studies in atopic dermatitis.1,2,5 Of the total number of patients included in these trials, 6,280 were randomized to receive RINVOQ at either dose.2-9
About RINVOQ® (upadacitinib)
Discovered and developed by AbbVie scientists, RINVOQ is a selective and reversible JAK inhibitor that is being studied in several immune-mediated inflammatory diseases.1,10-20 In human cellular assays, RINVOQ preferentially inhibits signaling by JAK1 or JAK1/3 with functional selectivity over cytokine receptors that signal via pairs of JAK2.1 RINVOQ 15 mg is also approved by the U.S. Food and Drug Administration (FDA) for adults with moderately to severely active rheumatoid arthritis, and by the European Commission for adults with moderate to severe active rheumatoid arthritis, adults with active psoriatic arthritis (PsA) and adults with active ankylosing spondylitis (AS). Phase 3 trials of RINVOQ in rheumatoid arthritis, atopic dermatitis, psoriatic arthritis, axial spondyloarthritis, Crohn's disease, ulcerative colitis, giant cell arteritis and Takayasu arteritis are ongoing.12-20
Please see the RINVOQ full SmPC for complete prescribing information at http://www.EMA.europa.eu.
European Commission approves AbbVie's atopic dermatitis treatment RINVOQ
Aug. 24, 2021 8:12 AM ETAbbVie Inc. (ABBV)
By: Aakash Babu, SA News Editor9 Comments
- European Commission (EC) has approved AbbVie's (NYSE:ABBV) RINVOQ for the treatment of moderate to severe atopic dermatitis in adults and adolescents 12 years and older who are candidates for systemic therapy.
- https://seekingalpha.com/news/3733438-european-commission-approves-abbvies-atopic-dermatitis-treatment-rinvoq
- https://seekingalpha.com/symbol/ABBV
- https://www.rinvoq.com/
EYLEA® (aflibercept) Injection
August 24, 2021 at 7:00 AM EDT Back
REGENERON ANNOUNCES ENCOURAGING TOPLINE PHASE 2 DATA OF HIGH-DOSE AFLIBERCEPT IN WET AGE-RELATED MACULAR DEGENERATION
TARRYTOWN, N.Y., Aug. 24, 2021 /PRNewswire/ --
Phase 3 trials in wet AMD and diabetic macular edema fully recruited, with results expected in the second half of 2022
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that an ongoing Phase 2 proof-of-concept trial evaluating an investigational 8 mg dose of aflibercept met its primary safety endpoint, with no new safety signals observed compared to the currently-approved 2 mg dose of EYLEA® (aflibercept) Injection in patients with wet age-related macular degeneration (wet AMD). In this small trial involving 106 patients, a higher proportion of patients in the aflibercept 8 mg group had no retinal fluid (43.4%, n=23/53) compared to patients treated with EYLEA 2 mg (26.4%, n=14/53) (p=0.067) at week 16, the primary efficacy endpoint. At this timepoint patients had received three initial doses (administered at weeks 0, 4 and 8), after which dosing was extended.
Aflibercept 8 mg is currently being evaluated in two large Phase 3 trials in wet AMD and diabetic macular edema (DME), which are expected to report results in the second half of 2022. The trials will assess the safety and efficacy of aflibercept 8 mg for up to two years, with visual acuity as the primary efficacy endpoint at 48 weeks, measured by the Early Treatment Diabetic Retinopathy Study (ETDRS) Best Corrected Visual Acuity (BCVA). Both trials will assess aflibercept 8 mg compared to EYLEA 2 mg, testing dosing intervals of every 12 weeks and every 16 weeks.
"We are cautiously optimistic that these early data suggest that a higher dose of aflibercept may potentially benefit patients with wet AMD, and we look forward to Phase 3 data next year, which will be crucial to understand its overall efficacy and safety," said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer of Regeneron. "Having worked for nearly two decades in retinal disease, we know that large, robust data sets are required to fully understand whether a medicine can achieve three critical things: improved visual and anatomic outcomes, convenient dosing, and a safety profile that is consistent with EYLEA."
During the initial 16 weeks of the Phase 2 trial, adverse events (AEs) in the study eye occurred in 17.0% (9 of 53) of aflibercept 8 mg patients and 22.6% (12 of 53) of EYLEA 2 mg patients. Serious ocular AEs occurred in two patients overall, one in the aflibercept 8 mg group (retinal tear) and one in the EYLEA 2 mg group (visual acuity reduced). There were no AEs of intraocular inflammation (including occlusive retinal vasculitis), anti-platelet trialists' collaboration (APTC)-defined arterial thromboembolic events or deaths in either patient group.
Wet AMD is the leading cause of vision loss among people 50 years and older in the U.S. Existing anti-VEGF treatments including EYLEA have helped change the course of disease for millions of patients worldwide, and efforts to develop new medicines are focused on further enhancing clinical effectiveness while extending the time between treatment doses. This new, concentrated high-dose aflibercept formulation enables a greater amount of medicine to be administered with each treatment, potentially extending the time between doses while retaining the efficacy and safety profile seen with EYLEA 2 mg.
Aflibercept 8 mg is being jointly developed by Regeneron and Bayer.
About the Phase 2 Trial
The Phase 2 randomized, single-masked trial (NCT04126317) enrolled 106 treatment-naïve patients with wet AMD. The trial was designed to investigate the safety, efficacy and tolerability of high-dose aflibercept (8 mg) compared to the existing approved dose of EYLEA (2 mg). Patients were randomized into two groups, with one group receiving aflibercept 8 mg (n=53) and the other group receiving EYLEA 2 mg (n=53). Patients in both groups received three initial injections (weeks 0, 4 and 8), before the primary endpoint was assessed at week 16, after which dosing was extended to every 12 weeks, or more frequently if required due to persistent or worsening disease. Efficacy was assessed via the presence of retinal fluid in the center subfield on optical coherence tomography (OCT) at this timepoint. The trial will continue through week 44.
Trial participants were at least 50 years of age (mean: 77 years), baseline retinal thickness was 502.1 microns, and the BCVA ETDRS letter score was between 24 to 78 in the study eye (mean: 59 letters).
About the Phase 3 Clinical Program
There are two ongoing pivotal trials to investigate the efficacy and safety of aflibercept 8 mg versus EYLEA 2 mg. In DME, Regeneron is sponsoring the Phase 2/3 multi-center, randomized, double-masked PHOTON trial (NCT04429503). In wet AMD, Bayer is sponsoring the Phase 3 multi-center, randomized, double-masked PULSAR trial (NCT04423718) in treatment naïve patients. Across both trials, patients are randomized into one of three treatment groups, testing aflibercept 8 mg with dosing regimens at either 12- or 16-week intervals or EYLEA 2 mg with an 8-week dosing regimen.
INDICATIONS
EYLEA® (aflibercept) Injection 2 mg (0.05 mL) is indicated for the treatment of patients with Neovascular (Wet) Age-related Macular Degeneration (AMD), Macular Edema following Retinal Vein Occlusion (RVO), Diabetic Macular Edema (DME), and Diabetic Retinopathy (DR).
For additional information about the company, please visit www.regeneron.com
View original content:https://www.prnewswire.com/news-releases/regeneron-announces-encouraging-topline-phase-2-data-of-high-dose-aflibercept-in-wet-age-related-macular-degeneration-301361118.html
SOURCE Regeneron Pharmaceuticals, Inc.
Regeneron's aflibercept met primary safety endpoint in mid-stage wet AMD study
Aug. 24, 2021 9:15 AM ET
Regeneron Pharmaceuticals, Inc. (REGN)Bayer Aktiengesellschaft (BAYRY)Bayer Aktiengesellschaft (BAYZF)
By: Mamta Mayani, SA News Editor
- Regeneron Pharmaceuticals (NASDAQ:REGN) announces that an ongoing Phase 2 trial evaluating an 8 mg dose of aflibercept met its primary safety endpoint, with no new safety signals observed compared to the currently-approved 2 mg dose of EYLEA (aflibercept) Injection in patients with wet age-related macular dhttps://seekingalpha.com/news/3733500-regenerons-aflibercept-met-primary-safety-endpoint-in-mid-stage-wet-amd-studyegeneration (wet AMD).
- https://seekingalpha.com/news/3733500-regenerons-aflibercept-met-primary-safety-endpoint-in-mid-stage-wet-amd-study
- https://seekingalpha.com/symbol/REGN
- https://www.regeneron.com/
- https://eylea.us/
CABOMETYX® (cabozantinib) in Combination with OPDIVO® (nivolumab)
Press Release
Exelixis Announces Partner Takeda and Ono Receive Approval in Japan for CABOMETYX® (cabozantinib) in Combination with OPDIVO® (nivolumab) for the Treatment of Unresectable or Metastatic Renal Cell CarcinomaPDF Version
– Approval based on the phase 3 CheckMate -9ER pivotal trial, which showed CABOMETYX in combination with OPDIVO improved overall survival and doubled median progression-free survival and objective response rate versus sunitinib –
ALAMEDA, Calif.--(BUSINESS WIRE)--Aug. 25, 2021-- Exelixis, Inc. (Nasdaq: EXEL) today announced Takeda Pharmaceutical Company Limited (Takeda), its partner responsible for the clinical development and commercialization of CABOMETYX® (cabozantinib) in Japan, and Ono Pharmaceutical Co., Ltd. (Ono) received approval from the Japanese Ministry of Health, Labor and Welfare to manufacture and market CABOMETYX in combination with OPDIVO® (nivolumab) as a treatment for unresectable or metastatic renal cell carcinoma (RCC).
“We’re excited our partner Takeda, along with Ono, will be able to bring CABOMETYX in combination with OPDIVO to patients with advanced kidney cancer in Japan following regulatory approvals as a first-line treatment in the U.S. and EU earlier this year,” said Michael M. Morrissey, Ph.D., Exelixis’ President and Chief Executive Officer. “With approximately 25,000 new cases of kidney cancer diagnosed in Japan annually, we’re pleased that this important new treatment option will now be available to Japanese patients in need of new therapies.”
The approval is based on CheckMate -9ER, a phase 3 pivotal trial evaluating CABOMETYX in combination with OPDIVO in previously untreated patients with advanced or metastatic RCC compared with sunitinib. In CheckMate -9ER, CABOMETYX in combination with OPDIVO demonstrated superior overall survival (OS) and doubled median progression-free survival (PFS) and objective response rate (ORR) versus sunitinib, with a favorable safety profile.
Per the terms of Exelixis and Takeda’s collaboration and license agreement, Exelixis is eligible to receive a milestone payment of $20 million from Takeda upon the first commercial sale of CABOMETYX in combination with OPDIVO for the treatment of RCC. Exelixis continues to be eligible to receive additional development, regulatory and first-sale milestones for potential future cabozantinib indications and is also eligible for sales revenue milestones and royalties on net sales of cabozantinib in Japan.
Takeda previously received approvals to manufacture and market CABOMETYX in Japan as a treatment for patients with curatively unresectable or metastatic RCC and for patients with unresectable hepatocellular carcinoma (HCC) that has progressed after prior systemic therapy.
About CheckMate -9ER
CheckMate -9ER is an open-label, randomized (1:1), multi-national phase 3 trial evaluating patients with previously untreated advanced or metastatic RCC with a clear cell component. A total of 651 patients (22% favorable risk, 58% intermediate risk, 20% poor risk; 25% PD-L1 ≥1%) were randomized to CABOMETYX at a dose of 40 mg once-daily and OPDIVO (n = 323) versus sunitinib (n = 328). The primary endpoint is PFS; secondary endpoints include OS and ORR. The primary efficacy analysis compares the doublet combination regimen of CABOMETYX and OPDIVO versus sunitinib in all randomized patients. The trial is sponsored by Bristol Myers Squibb and Ono Pharmaceutical Co. and co-funded by Exelixis, Ipsen and Takeda Pharmaceutical Company Limited.
About RCC
The American Cancer Society’s 2021 statistics cite kidney cancer as among the top ten most commonly diagnosed forms of cancer among both men and women in the U.S.1 Clear cell RCC is the most common form of kidney cancer in adults.2 If detected in its early stages, the five-year survival rate for RCC is high; for patients with advanced or late-stage metastatic RCC, however, the five-year survival rate is only 13%.1 Approximately 32,000 patients in the U.S. and 71,000 worldwide will require systemic treatment for advanced kidney cancer in 2021.3
About 70% of RCC cases are known as “clear cell” carcinomas, based on histology.4 The majority of clear cell RCC tumors have below-normal levels of a protein called von Hippel-Lindau, which leads to higher levels of MET, AXL and VEGF.5,6 These proteins promote tumor angiogenesis (blood vessel growth), growth, invasiveness and metastasis.7,8,9,10 MET and AXL may provide escape pathways that drive resistance to VEGF receptor inhibitors.6,7
About CABOMETYX® (cabozantinib)
In the U.S., CABOMETYX tablets are approved for the treatment of patients with advanced RCC; for the treatment of patients with HCC who have been previously treated with sorafenib; and for patients with advanced RCC as a first-line treatment in combination with OPDIVO. CABOMETYX tablets have also received regulatory approvals in the European Union and additional countries and regions worldwide. In 2016, Exelixis granted Ipsen exclusive rights for the commercialization and further clinical development of cabozantinib outside of the U.S. and Japan. In 2017, Exelixis granted exclusive rights to Takeda Pharmaceutical Company Limited for the commercialization and further clinical development of cabozantinib for all future indications in Japan. Exelixis holds the exclusive rights to develop and commercialize cabozantinib in the U.S.
For more information about Exelixis, please visit www.exelixis.com
Exelixis, the Exelixis logo, CABOMETYX, COMETRIQ and COTELLIC are registered U.S. trademarks of Exelixis. MINNEBRO is a registered trademark of Daiichi Sankyo Company, Limited.
OPDIVO® is a registered trademark of Bristol-Myers Squibb Company.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210825005760/en/ Source: Exelixis, Inc.
Exelixis and Takeda win Japanese approval for CABOMETYX in renal cell carcinoma
Aug. 25, 2021 4:39 PM ET Exelixis, Inc. (EXEL), TAK By: Dulan Lokuwithana, SA News Editor3 Comments
- Exelixis (NASDAQ:EXEL) and Takeda (NYSE:TAK) announced that the two companies along with their partner Ono Pharmaceutical received the regulatory approval for CABOMETYX (cabozantinib) in combination with OPDIVO (nivolumab) as a treatment of unresectable or metastatic renal cell carcinoma (RCC).
- https://seekingalpha.com/news/3734223-exelixis-and-takeda-win-japanese-approval-for-cabometyx-in-renal-cell-carcinoma
- https://seekingalpha.com/symbol/EXEL
- https://seekingalpha.com/symbol/TAK
- https://www.exelixis.com/
- https://www.cabometyx.com/
- https://www.opdivo.com/advanced-kidney-cancer/opdivo-cabometyx-combination
Tislelizumab (BGB-A317)
BeiGene Announces Acceptance of a Supplemental Biologics License Application in China for Tislelizumab in Nasopharyngeal Cancer
Aug 22, 2021 7:00 PM
CAMBRIDGE, Mass. & BEIJING--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a global biotechnology company focused on developing and commercializing innovative medicines worldwide, today announced that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has accepted a supplemental Biologics License Application (sBLA) for anti-PD-1 antibody tislelizumab in combination with chemotherapy as a first-line treatment for patients with recurrent or metastatic nasopharyngeal cancer (NPC).
“Treatment options for NPC, one of the most common head and neck cancers in China and many parts of Asia, are limited, with chemotherapy continuing to dominate front-line care. Supported by the positive RATIONALE 309 trial, the NMPA acceptance of this sBLA, which is the ninth for tislelizumab in China, represents an incredible milestone in its development history and serves as a validation of this potentially differentiated checkpoint inhibitor,” commented Yong (Ben) Ben, M.D., Chief Medical Officer, Immuno-Oncology at BeiGene. “We look forward to bringing this important immunotherapy to the underserved NPC patient community in China.”
The sBLA is supported by clinical results from a randomized, double-blind, Phase 3 clinical trial RATIONALE 309 (NCT03924986) to evaluate the efficacy and safety of tislelizumab combined with gemcitabine and cisplatin versus placebo combined with gemcitabine and cisplatin as a first-line treatment for patients with recurrent or metastatic NPC. The primary endpoint of this trial is progression-free survival (PFS) as assessed by independent review committee (IRC) in the intention-to-treat (ITT) population; secondary endpoints include overall survival (OS), IRC-assessed overall response rate (ORR) and duration of response (DoR), and investigator-assessed PFS. A total of 263 Asian patients were enrolled and randomized 1:1 to either the tislelizumab plus chemotherapy arm or the placebo plus chemotherapy arm.
As announced in May 2021, RATIONALE 309 met the primary endpoint of PFS at the planned interim analysis. The safety profile of tislelizumab was consistent with its known risks, with no new safety signals identified with the addition of chemotherapy. BeiGene expects to present results from the RATIONALE 309 trial at an upcoming medical conference.
About Tislelizumab
Tislelizumab (BGB-A317) is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to FcγR on macrophages. In pre-clinical studies, binding to FcγR on macrophages has been shown to compromise the anti-tumor activity of PD-1 antibodies through activation of antibody-dependent macrophage-mediated killing of T effector cells. Tislelizumab is the first drug from BeiGene’s immuno-oncology biologics program and is being developed internationally as a monotherapy and in combination with other therapies for the treatment of a broad array of both solid tumor and hematologic cancers.
The China National Medical Products Administration (NMPA) has granted tislelizumab approval in five indications, including full approval for first-line treatment of patients with advanced squamous non-small cell lung cancer (NSCLC) in combination with chemotherapy and for first-line treatment of patients with advanced non-squamous NSCLC in combination with chemotherapy; and conditional approval for the treatment of patients with classical Hodgkin’s lymphoma (cHL) who received at least two prior therapies, for the treatment of patients with locally advanced or metastatic urothelial carcinoma (UC) with PD-L1 high expression whose disease progressed during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy, and for the treatment of patients with hepatocellular carcinoma (HCC) who have received at least one systemic therapy. Full approval for these indications is contingent upon results from ongoing randomized, controlled confirmatory clinical trials.
In addition, four supplemental Biologics License Applications for tislelizumab have been accepted by the Center for Drug Evaluation (CDE) of the NMPA and are under review for second- or third-line treatment of patients with locally advanced or metastatic NSCLC who progressed on prior platinum-based chemotherapy, for patients with previously treated, locally advanced unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair-deficient (dMMR) solid tumors, for the treatment of patients with locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who have disease progression following or are intolerant to first-line standard chemotherapy, and for first-line treatment of patients with recurrent or metastatic nasopharyngeal cancer (NPC).
BeiGene has initiated or completed 17 potentially registration-enabling clinical trials in China and globally, including 13 Phase 3 trials and four pivotal Phase 2 trials.
In January 2021, BeiGene and Novartis entered into a collaboration and license agreement granting Novartis rights to develop, manufacture, and commercialize tislelizumab in North America, Europe, and Japan.
Tislelizumab is not approved for use outside of China.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210822005013/en/
Source: BeiGene, Ltd.
BeiGene's tislelizumab sBLA accepted in China for nasopharyngeal cancer
Aug. 22, 2021 11:46 PM ET
By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) announces that the Center for Drug Evaluation of China has accepted a supplemental Biologics License Application (sBLA) for anti-PD-1 antibody tislelizumab in combination with chemotherapy as a first-line treatment for patients with recurrent or metastatic nasopharyngeal cancer (NPC).
- https://seekingalpha.com/news/3732892-beigenes-tislelizumab-sbla-accepted-in-china-for-nasopharyngeal-cancer
- https://seekingalpha.com/symbol/BGNE
- https://www.beigene.com/en-us
Orencia (abatacept)
U.S. Food and Drug Administration Accepts for Priority Review Bristol Myers Squibb’s Application for Orencia (abatacept) for the Prevention of Acute Graft Versus Host Disease (aGvHD)
08/23/2021CATEGORY:
U.S. Food and Drug Administration assigned an action date of December 23, 2021
The sBLA is supported by the Phase 2 ABA2 Trial evaluating Orencia in adults and children to prevent aGvHD
If approved, Orencia would become the first therapy for the prevention of aGvHD
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE:BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental Biologics License Application (sBLA) for Orencia (abatacept) for the prevention of moderate to severe acute graft versus host disease (aGvHD) in patients 6 years of age and older receiving unrelated donor hematopoietic stem cell transplantation (HSCT). The FDA granted the application Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) goal date of December 23, 2021.
“While stem cell transplants are an effective treatment for aggressive leukemias and other hematological malignancies, patients who receive stem cell transplants from unrelated and human leukocyte antigens (HLA)-mismatched donors are at high risk for developing aGvHD,” said study lead investigator Leslie Kean, M.D., PhD, Director of the Pediatric Stem Cell Transplantation Program, Boston Children's Hospital/Dana-Farber Cancer Institute. “There is a tremendous need to expand the stem cell donor pool by lowering the risk of aGvHD in both adults and children receiving unrelated donor stem cell transplants.”
Stem cell transplants include infusion of donor T-cells, a type of white blood cell that recognizes and destroys foreign invaders in the recipient’s body, including cancer cells. GvHD occurs when the donor T-cells also recognize the patient’s healthy cells as foreign and start attacking healthy tissues and organs. To initiate this attack, T-cells require activation through a signaling process called co-stimulation. Between 30 and 70 percent of transplant recipients develop aGvHD, depending on donor type, transplant technique, and other features. Orencia, a therapy currently approved to treat various arthritic conditions, binds to and inhibits protein targets involved in co-stimulation, thus inhibiting T-cell activation.
“For patients who receive unrelated donor stem cell transplants, in particular for racial and ethnic minority patient populations, there is a heightened risk of developing aGvHD, a potentially life-threatening medical complication for which there are no approved preventive therapies,” said Mary Beth Harler, M.D., head of Immunology and Fibrosis Development, Bristol Myers Squibb. “We look forward to working with the FDA to bring Orencia to this new patient population and employ pathbreaking science in an effort to address unmet needs of underserved patients.”
The sBLA submitted to the FDA is based on results from the Phase 2 ABA2 trial and a registry trial based on real world evidence. The ABA2 trial assessed the impact of Orencia on the prevention of severe aGvHD, when added to a standard GvHD prophylactic regimen administered to patients with hematologic malignancies receiving a stem cell transplant from an unrelated, HLA-matched or mismatched donor. A mismatch in HLA increases the risk of GvHD. Results from ABA2 showed that treatment with Orencia resulted in a significant reduction in severe aGvHD and associated morbidity without an increase in disease relapse. The findings of the real-world analysis were consistent with those of ABA2.
About ABA2
The ABA2 study was a multicenter, Phase 2 investigator sponsored trial conducted by Dr. Leslie Kean of Boston Children’s Hospital/Dana Farber Cancer Institute. ABA2 had two cohorts: a single arm cohort for patients receiving transplants from mismatched unrelated donors (MMUD) (“7/8” cohort), and a randomized, double blind, placebo-controlled cohort for patients receiving transplants from 8/8 matched unrelated donors (MUD) (“8/8” cohort). All subjects received a calcineurin inhibitor (CNI), with dosing starting on day -2 and continuing through at least Day 100 as tolerated, and methotrexate (MTX) on days one, three, six and 11 (transplant day is Day 0). Orencia-treated subjects received 10 mg/kg Orencia on days -1, 5, 14 and 28.
In the ABA2 clinical trial, addition of Orencia to SOC aGvHD prophylaxis of MTX+CNI resulted in a significantly higher aGvHD-free survival (GFS) rate compared to registry controls in the single-arm 7/8 HLA-matched cohort, and numerically higher severe GFS rate in the double-blind, placebo-controlled 8/8 HLA-matched cohort at 180 days post-transplant.
About ORENCIA ®
ORENCIA® is an immunomodulator that disrupts the continuous cycle of T-cell activation.
U.S. Indications/Usage and Important Safety Information for ORENCIA® (abatacept)
Indications and Usage
Adult Rheumatoid Arthritis: ORENCIA® (abatacept) is indicated for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA).
Polyarticular Juvenile Idiopathic Arthritis: ORENCIA® (abatacept) is indicated for the treatment of patients 2 years of age and older with moderately to severely active polyarticular juvenile idiopathic arthritis (pJIA).
Adult Psoriatic Arthritis: ORENCIA® (abatacept) is indicated for the treatment of adult patients with active psoriatic arthritis (PsA).
Limitations of Use: The concomitant use of ORENCIA with other potent immunosuppressants [e.g., biologic disease-modifying antirheumatic drugs (bDMARDS), Janus kinase (JAK) inhibitors] is not recommended.
For more information about Bristol Myers Squibb, visit us at BMS.com
FDA grants accelerated review to Bristol Myers' Orencia application for prevention of aGvHD
Aug. 23, 2021 7:19 AM ET Bristol-Myers Squibb Company (BMY)
By: Mamta Mayani, SA News Editor1 Comment
- Under priority review, the FDA has accepted Bristol Myers Squibb's (NYSE:BMY) supplemental Biologics License Application (sBLA) for Orencia (abatacept) for the prevention of moderate to severe acute graft versus host disease (aGvHD) in patients 6 years of age and older receiving unrelated donor hematopoietic stem cell transplantation (HSCT).
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
- https://www.orencia.com/
Jardiance® (empagliflozin)
US FDA approves Jardiance® (empagliflozin) to treat adults living with heart failure with reduced ejection fraction
August 18, 2021Download PDF- New treatment options are critical, as approximately half of all people with heart failure die within five years of diagnosis
- Heart failure accounts for more than one million hospitalizations a year in the U.S.
RIDGEFIELD, Conn. and INDIANAPOLIS, Aug. 18, 2021 /PRNewswire/ -- Jardiance® (empagliflozin) 10 mg has been approved by the U.S. Food and Drug Administration to reduce the risk of cardiovascular death plus hospitalization for heart failure in adults with heart failure with reduced ejection fraction (HFrEF), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced today.
About EMPEROR-Reduced
EMPEROR–Reduced (NCT03057977) was a phase III international, randomized, double–blind trial that enrolled 3,730 adults with and without type 2 diabetes. All participants had heart failure (New York Heart Association [NYHA] functional class II, III or IV) for at least 3 months and a left ventricular ejection fraction of 40% or less at their most recent assessment. At randomization, 75% of patients were NYHA class II, 24% were class III and 0.5% were class IV.
Participants were randomized to once-daily Jardiance 10 mg (n=1,863) or placebo (n=1,867), on top of treatment with guideline-directed heart failure therapy. Median follow-up time was 16 months. The composite primary endpoint was defined as time to first event of cardiovascular death or hospitalization for heart failure.
What is JARDIANCE? (www.jardiance.com)
JARDIANCE is a prescription medicine used to:
- lower blood sugar along with diet and exercise in adults with type 2 diabetes
- reduce the risk of cardiovascular death in adults with type 2 diabetes who also have known cardiovascular disease
- reduce the risk of cardiovascular death and hospitalization for heart failure (when the heart is weak and cannot pump enough blood to the rest of your body) in adults with heart failure
JARDIANCE is not for people with type 1 diabetes. It may increase their risk of diabetic ketoacidosis (increased ketones in the blood or urine).
JARDIANCE is not for use to lower blood sugar in adults with type 2 diabetes who have severe kidney problems, because it may not work.
For more information, please see Prescribing Information and Medication Guide.
Boehringer Ingelheim and Eli Lilly and Company
In January 2011, Boehringer Ingelheim and Eli Lilly and Company announced an alliance that centers on compounds representing several of the largest diabetes treatment classes. Depending on geographies, the companies either co-promote or separately promote the respective molecules each contributing to the alliance. The alliance leverages the strengths of two of the world's leading pharmaceutical companies to focus on patient needs. By joining forces, the companies demonstrate their commitment, not only to the care of people with diabetes, but also to investigating the potential to address areas of unmet medical need. Clinical trials have been initiated to evaluate the impact of Jardiance on people living with heart failure or chronic kidney disease.
For more information, please visit www.boehringer-ingelheim.us,
For the latest updates, visit http://www.lillydiabetes.com/
To learn more about Lilly, please visit us at lilly.com
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SOURCE Eli Lilly and Company
FDA approves Lilly's Jardiance to treat certain patients with heart failure
Aug. 18, 2021 2:21 PM ET Eli Lilly and Company (LLY) By: Aakash Babu, SA News Editor1 Comment
- Eli Lilly (LLY -0.5%) and development partner Boehringer Ingelheim announce that the U.S. FDA has approved Jardiance (empagliflozin) 10 mg to reduce the risk of cardiovascular death plus hospitalization for heart failure in adults with heart failure with reduced ejection fraction ((HFrEF)).
- https://seekingalpha.com/news/3731988-fda-approves-lillys-jardiance-to-treat-certain-patients-with-heart-failure
- https://seekingalpha.com/symbol/LLY
- https://www.lilly.com/
- https://www.jardiance.com/
Landmark trial demonstrates Jardiance® (empagliflozin) is the first therapy to show statistically significant improvement in heart failure outcomes in adults with preserved ejection fraction
August 27, 2021Download PDF- In this clinical first for adults with heart failure with preserved ejection fraction, Jardiance demonstrated an impressive 21% relative risk reduction in the composite primary endpoint of cardiovascular death or hospitalization for heart failure
- The benefit in the primary endpoint was independent of ejection fraction or diabetes status
- Jardiance also reduced the relative risk of first and recurrent hospitalizations for heart failure by 27% and significantly slowed kidney function decline
- Results from the EMPEROR-Preserved phase III trial were presented today at the European Society of Cardiology Congress 2021 and published in The New England Journal of Medicine
RIDGEFIELD, Conn. and INDIANAPOLIS, Aug. 27, 2021 /PRNewswire/ -- Full results from the landmark EMPEROR-Preserved phase III trial demonstrated that Jardiance® (empagliflozin) showed an impressive 21% relative risk reduction for the composite primary endpoint of cardiovascular death or hospitalization for heart failure in adults with heart failure with preserved ejection fraction (HFpEF) compared with placebo. The benefit was independent of ejection fraction or diabetes status, establishing Jardiance as the first and only treatment to significantly improve outcomes for the full spectrum of heart failure patients. The results were presented today at the European Society of Cardiology (ESC) Congress 2021 and published in The New England Journal of Medicine, Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced.
Key secondary endpoint analyses from the trial showed that Jardiance also reduced the relative risk of first and recurrent hospitalizations for heart failure by 27% and significantly slowed kidney function decline.
EVRENZO™ (roxadustat)
Astellas Receives European Commission Approval for First-in-Class EVRENZO™ (roxadustat) for Adult Patients with Symptomatic Anemia of Chronic Kidney Disease
Roxadustat is the first orally administered hypoxia-inducible factor (HIF) prolyl hydroxylase (PH) inhibitor available for adult patients with anemia associated with chronic kidney disease in Europe
TOKYO, Aug. 19, 2021 /PRNewswire/ -- Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") and FibroGen, Inc. (Nasdaq: FGEN, CEO: Enrique Conterno, "FibroGen") today announced that the European Commission (EC) has approved EVRENZO™ (roxadustat) for the treatment of adult patients with symptomatic anemia associated with chronic kidney disease (CKD).
"We are very pleased EVRENZO has been approved as the first oral HIF-PH inhibitor to treat adult patients with symptomatic anemia associated with CKD in the European Union," said Steven Benner, M.D., M.H.S., President of Development, Astellas. "Today's approval provides patients, regardless of dialysis status, with a first-in-class treatment option to address the multifaceted nature of this condition. We look forward to making roxadustat available to adult patients with anemia of CKD in countries across the European Union."
Roxadustat is the first orally administered HIF-PH inhibitor available in the European Union. Roxadustat increases hemoglobin (Hb) levels through a different mechanism of action compared to injectable erythropoiesis-stimulating agents (ESAs) which are typically co-administered with intravenous iron. As a HIF-PH inhibitor, roxadustat activates the body's natural response to reduced oxygen levels in the blood. This response involves the regulation of multiple, coordinated processes that allow management of anemia with a reduced use of intravenous iron.
"HIF-PH inhibitors represent a major advance in the treatment of anemia of CKD," said Mark Eisner, M.D., M.P.H., Chief Medical Officer, FibroGen. "Roxadustat provides a novel breakthrough for patients who suffer from this condition."
This approval follows the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) positive opinion to authorize roxadustat in June6 based on results from a comprehensive pivotal Phase 3 program comprising of eight multicenter and randomized studies, which involved 9,600 patients worldwide.7-12 The results of this program showed roxadustat was efficacious in achieving and maintaining target Hb levels (10-12g/dL) in patients with symptomatic anemia of CKD regardless of dialysis status and irrespective of prior ESA treatment.7-11 The safety profile observed in the roxadustat development program is reflective of the CKD populations studied and comparable to ESAs.7-12
https://www.fibrogen.com/roxadustat
For more information, please visit our website at https://www.astellas.com/en.
For more information, please visit www.fibrogen.com.
FibroGen rises as EU approval of Roxadustat prompts Raymond James upgrade
Aug. 20, 2021 8:06 AM ET FibroGen, Inc. (FGEN)Astellas Pharma Inc. (ALPMF)
By: Dulan Lokuwithana, SA News Editor
- Raymond James has upgraded FibroGen (NASDAQ:FGEN) to market perform from underperform after the company joined Astellas Pharma (OTCPK:ALPMF) on Thursday to announce the European approval of EVRENZO (roxadustat) for adults with anemia linked to chronic kidney disease (CKD).
- https://seekingalpha.com/news/3732643-fibrogen-rises-as-eu-approval-of-roxadustat-prompts-raymond-james-upgrade
- https://seekingalpha.com/symbol/FGEN
- https://seekingalpha.com/symbol/ALPMF
- https://www.astellas.com/en/
Abecma (Idecabtagene Vicleucel)
Bristol Myers Squibb Receives European Commission Approval for Abecma (Idecabtagene Vicleucel), the First Anti-BCMA CAR T Cell Therapy for Relapsed and Refractory Multiple Myeloma
08/19/2021CATEGORY:
Abecma represents the only cell therapy approved for multiple myeloma
Approval of Abecma is based on the pivotal KarMMa trial of patients worldwide, including five European countries, which demonstrated rapid, deep and durable responses with a well-understood and predictable safety profile
Abecma expands upon Bristol Myers Squibb’s leadership in cell therapy research and multiple myeloma, offering an innovative option to patients in need
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the European Commission (EC) has granted Conditional Marketing Authorization for Abecma (idecabtagene vicleucel; ide-cel), a first-in-class B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.
Abecma is the first and only CAR T cell therapy approved that is directed to recognize and bind to BCMA, a protein that is nearly universally expressed on cancer cells in multiple myeloma, leading to the death of BCMA-expressing cells.1Abecma is delivered via a single infusion with a target dose of 420 x 106 CAR-positive viable T cells within a range of 260 to 500 x 106 CAR-positive viable T cells. Abecma is approved for use in all European Union (EU) member states.*
“The EC approval of Abecma is an important milestone for the treatment of multiple myeloma, and moves us closer to offering a first-in-class, personalized therapy to patients in Europe battling this incurable disease after exhausting prior treatment options with the three standards of care,” said Samit Hirawat, M.D., chief medical officer, Bristol Myers Squibb. “With this third regulatory approval for Abecma worldwide, we are proud to be advancing the science of cell therapy and continuing to bring this first anti-BCMA CAR T cell therapy to patients in need.”
Abecma Clinical Trial Results
The efficacy of Abecma is based on results from the pivotal KarMMa study in which 128 patients with relapsed and refractory multiple myeloma who had received at least three prior therapies including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and were refractory to the last treatment regimen were treated with Abecma.7
In the study, the overall response rate (ORR) was 73% (95% CI: 66-81), and 33% of patients achieved a complete response (CR; 95% CI: 25-41). Onset of response was rapid with a median time to response of one month. In addition, responses were durable, with a median duration of response of 10.6 months (95% CI: 8.0 – 11.4), and 23 months (95% CI: 11.4 – 23.3) for those who achieved a CR.7
In a pooled safety analysis of 184 patients treated with Abecma in the KarMMa and CRB-401 studies, cytokine release syndrome (CRS) occurred in 81% of patients, with Grade >3 CRS, using the Lee grading system, occurring in 5.4% of patients. There was one case of fatal (Grade 5) CRS reported. The median time to onset of CRS was one day (range: 1-17 days) and the median duration of CRS was five days (range: 1-63 days). Any grade neurotoxicity (NT) of the 128 patients receiving Abecma in the KarMMa study occurred in 18% of patients, including Grade 3 events in 3.1% of patients, with no Grade 4 or 5 events occurring. The median time to onset of NT was two days (range: 1-10 days) and the median duration was three days (range: 1-26 days).7
The most common (>20%) adverse reactions in the pooled safety analysis included neutropenia, CRS, anaemia, thrombocytopenia, infections - pathogen unspecified, leucopenia, fatigue, diarrhoea, hypokalaemia, hypophosphataemia, nausea, lymphopenia, pyrexia, cough, hypocalcaemia, infections - viral, headache, hypomagnesaemia, upper respiratory tract infection, arthralgia, and oedema peripheral. The most common Grade 3 or 4 adverse reactions were neutropenia (88.6%), anaemia (58.2%), thrombocytopenia (53.5%), leucopenia (45.1%), lymphopenia (30.4%), infections - pathogen unspecified (17.9%), hypophosphataemia (17.4%), febrile neutropenia (14.7%), hypocalcaemia (7.1%), infections - viral (7.1%), pneumonia (6.0%), CRS (5.4%), hypertension (5.4%) and hyponatraemia (5.4%).7
Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide, and Summary of Product Characteristics for ABECMA.
About Abecma
Abecma is the first-in-class B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, first approved in the U.S. in March 2021 for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 monoclonal antibody. Abecma is also approved in Canada for relapsed and refractory multiple myeloma. Abecma recognizes and binds to BCMA on the surface of multiple myeloma cells leading to CAR T cell proliferation, cytokine secretion, and subsequent cytolytic killing of BCMA-expressing cells.
Abecma is being jointly developed and commercialized in the U.S. as part of a Co-Development, Co-Promotion, and Profit Share Agreement with Bristol Myers Squibb and bluebird bio. Bristol Myers Squibb will assume sole responsibility for Abecma drug product manufacturing and commercialization outside of the U.S.
For more information about Bristol Myers Squibb, visit us at BMS.com
EC approves Bristol Myers Squibb's Abecma to treat certain patients with multiple myeloma
Aug. 19, 2021 9:50 AM ETBristol-Myers Squibb Company (BMY)By: Aakash Babu, SA News Editor
- The European Commission (EC) has granted conditional marketing authorization to Bristol Myers Squibb's (BMY +0.1%) Abecma for the treatment of certain patients with relapsed and refractory multiple myeloma.
- https://seekingalpha.com/news/3732327-ec-approves-bristol-myers-squibbs-abecma-to-treat-certain-patients-with-multiple-myeloma
- https://seekingalpha.com/symbol/BMY
- https://seekingalpha.com/symbol/BLUE
biotecMAX™ April/May/June/July/Aug 2021 Insight
Opdivo (nivolumab) + Yervoy (ipilimumab) and Opdivo + Chemotherapy
EMA Validates Bristol Myers Squibb’s Applications for Opdivo (nivolumab) + Yervoy (ipilimumab) and Opdivo + Chemotherapy as First-Line Treatments for Unresectable Advanced, Recurrent or Metastatic Esophageal Squamous Cell Carcinoma
08/17/2021CATEGORY:
Applications based on positive results from the Phase 3 CheckMate -648 trial, in which both Opdivo-based combinations demonstrated a significant survival benefit over chemotherapy alone
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced that the European Medicines Agency (EMA) has validated its Type II Variation Marketing Authorization Applications (MAA) for both Opdivo (nivolumab) in combination with Yervoy (ipilimumab) and Opdivo in combination with fluoropyrimidine- and platinum-containing chemotherapy as first-line treatments for adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC). Validation of these applications confirm that the submissions are complete and begins the EMA’s centralized review process.
“Outcomes for patients with advanced esophageal squamous cell carcinoma treated with chemotherapy alone remain poor, and there is a clear need for additional options beyond this long-standing standard of care,” said Ian M. Waxman, M.D., development lead, gastrointestinal cancers, Bristol Myers Squibb. “The validation of our applications moves us a step closer to potentially bringing these two Opdivo-based regimens to patients in the EU who may benefit.”
The applications are based on results from the pivotal Phase 3 CheckMate -648 trial, in which both Opdivo-based treatment combinations — Opdivo plus Yervoy and Opdivo plus chemotherapy — demonstrated a statistically significant and clinically meaningful overall survival (OS) benefit compared to chemotherapy at the pre-specified interim analysis in patients with unresectable advanced or metastatic esophageal squamous cell carcinoma (ESCC) with tumor cell PD-L1 expression ≥1%, as well as in the all-randomized population. Opdivo plus Yervoy is the first dual immunotherapy combination to demonstrate a superior survival benefit versus chemotherapy in this setting. The safety profiles of Opdivo and chemotherapy and of Opdivo plus Yervoy were consistent with the known safety profiles of the individual components.
Results from CheckMate -648 were presented in an oral session during the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting and were selected for the official ASCO press program.
Bristol Myers Squibb thanks the patients and investigators involved in the CheckMate -648 trial.
About CheckMate -648
CheckMate -648 is a randomized Phase 3 study evaluating Opdivo plus Yervoy or Opdivo plus fluorouracil and cisplatin against fluorouracil plus cisplatin alone in patients with unresectable advanced or metastatic esophageal squamous cell carcinoma. The primary endpoints of the trial are overall survival (OS) and progression-free survival (PFS) by blinded independent central review (BICR) in patients whose tumors express PD-L1 ≥1% for both Opdivo-based combinations versus chemotherapy. Secondary endpoints of the trial include OS and PFS by BICR in the all-randomized population.
In the Opdivo plus Yervoy arm, patients received treatment with Opdivo 3 mg/kg every 2 weeks and Yervoy 1 mg/kg every 6 weeks up to 24 months or until disease progression or unacceptable toxicity. In the Opdivo plus chemotherapy arm, patients received treatment with Opdivo 240 mg on Day 1 and Day 15, fluorouracil 800 mg/m²/day on Day 1 through Day 5 (for 5 days), and cisplatin 80 mg/m² on Day 1 of four-week cycle. Patients received Opdivo for up to 24 months or until disease progression or unacceptable toxicity, and chemotherapy until disease progression or unacceptable toxicity.
About Opdivo
Opdivo is a programmed death-1 (PD-1) immune checkpoint inhibitor that is designed to uniquely harness the body’s own immune system to help restore anti-tumor immune response. By harnessing the body’s own immune system to fight cancer, Opdivo has become an important treatment option across multiple cancers.
Opdivo’s leading global development program is based on Bristol Myers Squibb’s scientific expertise in the field of Immuno-Oncology and includes a broad range of clinical trials across all phases, including Phase 3, in a variety of tumor types. To date, the Opdivo clinical development program has treated more than 35,000 patients. The Opdivo trials have contributed to gaining a deeper understanding of the potential role of biomarkers in patient care, particularly regarding how patients may benefit from Opdivo across the continuum of PD-L1 expression.
In July 2014, Opdivo was the first PD-1 immune checkpoint inhibitor to receive regulatory approval anywhere in the world. Opdivo is currently approved in more than 65 countries, including the United States, the European Union, Japan and China. In October 2015, the Company’s Opdivo and Yervoy combination regimen was the first Immuno-Oncology combination to receive regulatory approval for the treatment of metastatic melanoma and is currently approved in more than 50 countries, including the United States and the European Union.
About Yervoy
Yervoy is a recombinant, human monoclonal antibody that binds to the cytotoxic T-lymphocyte-associated antigen-4 (CTLA-4). CTLA-4 is a negative regulator of T-cell activity. Yervoy binds to CTLA-4 and blocks the interaction of CTLA-4 with its ligands, CD80/CD86. Blockade of CTLA-4 has been shown to augment T-cell activation and proliferation, including the activation and proliferation of tumor infiltrating T-effector cells. Inhibition of CTLA-4 signaling can also reduce T-regulatory cell function, which may contribute to a general increase in T-cell responsiveness, including the anti-tumor immune response. On March 25, 2011, the U.S. Food and Drug Administration (FDA) approved Yervoy 3 mg/kg monotherapy for patients with unresectable or metastatic melanoma. Yervoy is approved for unresectable or metastatic melanoma in more than 50 countries. There is a broad, ongoing development program in place for Yervoy spanning multiple tumor types.
INDICATIONS
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with unresectable or metastatic melanoma.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer (NSCLC) whose tumors express PD-L1 (≥1%) as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab) and 2 cycles of platinum-doublet chemotherapy, is indicated for the first-line treatment of adult patients with metastatic or recurrent non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
OPDIVO® (nivolumab) is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with progression on or after platinum-based chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving OPDIVO.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of adult patients with unresectable malignant pleural mesothelioma (MPM).
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the first-line treatment of patients with intermediate or poor risk advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab), in combination with cabozantinib, is indicated for the first-line treatment of patients with advanced renal cell carcinoma (RCC).
OPDIVO® (nivolumab) is indicated for the treatment of patients with advanced renal cell carcinoma (RCC) who have received prior anti-angiogenic therapy.
OPDIVO® (nivolumab) is indicated for the treatment of adult patients with classical Hodgkin lymphoma (cHL) that has relapsed or progressed after autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin or after 3 or more lines of systemic therapy that includes autologous HSCT. This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab) is indicated for the treatment of patients with recurrent or metastatic squamous cell carcinoma of the head and neck (SCCHN) with disease progression on or after platinum-based therapy.
OPDIVO® (nivolumab) is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma who have disease progression during or following platinum-containing chemotherapy or have disease progression within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), as a single agent, is indicated for the treatment of adult and pediatric (12 years and older) patients with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of adults and pediatric patients 12 years and older with microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) metastatic colorectal cancer (CRC) that has progressed following treatment with a fluoropyrimidine, oxaliplatin, and irinotecan. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
OPDIVO® (nivolumab), in combination with YERVOY® (ipilimumab), is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph nodes or metastatic disease who have undergone complete resection.
OPDIVO® (nivolumab) is indicated for the treatment of patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC) after prior fluoropyrimidine- and platinum-based chemotherapy.
OPDIVO® (nivolumab) is indicated for the adjuvant treatment of completely resected esophageal or gastroesophageal junction cancer with residual pathologic disease in patients who have received neoadjuvant chemoradiotherapy (CRT).
OPDIVO® (nivolumab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the treatment of patients with advanced or metastatic gastric cancer, gastroesophageal junction cancer, and esophageal adenocarcinoma.
For more information about Bristol Myers Squibb, visit us at BMS.com
EMA validates Bristol Myers' Opdivo combo applications in esophageal cancer
Aug. 17, 2021 7:07 AM ET Bristol-Myers Squibb Company (BMY) By: Mamta Mayani, SA News Editor
- Bristol Myers Squibb (NYSE:BMY) announces that the EMA has validated its Type II Variation Marketing Authorization Applications (MAA) for both Opdivo (nivolumab) in combination with Yervoy (ipilimumab) and Opdivo + fluoropyrimidine- and platinum-containing chemotherapy as first-line treatments for adult patients with unresectable advanced, recurrent or metastatic esophageal squamous cell carcinoma (ESCC).
- https://seekingalpha.com/news/3731281-ema-validates-bristol-myers-opdivo-combo-applications-in-esophageal-cancer
- https://seekingalpha.com/symbol/BMY
- https://www.bms.com/
VENTANA MMR RxDx Panel
Roche receives FDA approval for first companion diagnostic to identify dMMR solid tumour patients eligible for anti-PD-1 immunotherapy
- Cancer is the second leading cause of death worldwide, with nearly 10 million deaths annually.1, 2 In the U.S., approximately 1.9 million new cancer cases are expected to be diagnosed in 2021.1
- Based on cancer biomarkers, the first-of-its-kind VENTANA MMR RxDx Panel helps determine which solid tumour patients may benefit from GSK immunotherapy.
- Roche/GSK collaboration represents an important step towards a personalised healthcare strategy for certain solid tumour patients.
Basel, 18 August 2021 – Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced U.S. Food and Drug Administration (FDA) approval of the VENTANA MMR RxDx Panel, advancing the company's commitment to personalised healthcare through tests that determine which patients are most likely to benefit from specific and targeted therapies. The VENTANA MMR RxDx Panel is the first companion diagnostic test to aid in identifying patients whose solid tumours are deficient in DNA mismatch repair (MMR), who may be eligible for JEMPERLI (dostarlimab-gxly) monotherapy, an anti-PD-1 immunotherapy from GSK. The test evaluates a panel of MMR proteins in tumours to provide important treatment information to clinicians.
MMR is a naturally occurring mechanism that scans our DNA, correcting errors that cause disease. When MMR is deficient (dMMR), cells mutate, which can lead to cancer. While MMR deficiency is most common in endometrial cancer, other high prevalence dMMR tumour types include gastric, colorectal, small intestine, cervical and neuroendocrine cancers. In the U.S., prevalence of dMMR across patients with solid tumours has been estimated at 14 percent.3 PD-1 inhibitors can be effective treatment in cancers with MMR deficiency.
FDA approval of the VENTANA MMR RxDx Panel provides clinicians with access to a fully automated panel of MMR biomarkers tested by immunohistochemistry (IHC), enabling impactful treatment decisions for patients. JEMPERLI was approved by the FDA on 17 August 2021 for the treatment of adult patients with dMMR recurrent or advanced solid tumours, as determined by an FDA-approved test, that have progressed on or following prior treatment and who have no satisfactory alternative treatment options. This indication received accelerated approval based on tumour response rate and durability of response. Continued approval for this indication may depend on verification and description of clinical benefit in a confirmatory trial(s).
The VENTANA MMR RxDx Panel and JEMPERLI were earlier approved by the FDA for use in endometrial cancer in April 2021.
Read more about Roche innovation in MMR biomarker testing.
About the VENTANA MMR RxDx Panel
The VENTANA MMR RxDx Panel is a label expansion of Roche’s current on-market VENTANA MMR IHC Panel. The VENTANA MMR RxDx Panel is intended for the assessment of expression of MMR proteins in formalin-fixed, paraffin-embedded (FFPE) tumour tissue stained with OptiView DAB IHC Detection Kit and ancillary reagents in the panel for VENTANA anti-MLH1 (M1), VENTANA anti-MSH2 (G219-1129) and VENTANA anti-MSH6 (SP93) and OptiView DAB IHC Detection Kit with the OptiView Amplification Kit and ancillary reagents for VENTANA anti-PMS2 (A16-4) on a BenchMark ULTRA instrument. DNA mismatch repair (MMR) proteins have been clinically proven to be predictive biomarkers for PD-1 targeted therapy; specifically, a loss of expression of one or more MMR proteins might predict an increased likelihood of response to such therapy.4,5,6 PD-1 inhibitors can be effective in cancers with MMR deficiency.4,6 MMR is a conserved molecular mechanism that functions to correct the improper base substitutions that spontaneously occur during DNA replication. Defects in the MMR machinery have been attributed to mutations in the MMR proteins.
Roche announces FDA approval of companion diagnostic for dMMR solid tumors
Aug. 18, 2021 12:04 PM ET Roche Holding AG (RHHBY)AnaptysBio, Inc. (ANAB)GlaxoSmithKline plc (GSK)
By: Dulan Lokuwithana, SA News Editor
- Roche (OTCQX:RHHBY +0.2%) said that the FDA approved VENTANA MMR RxDx Panel, a companion diagnostic to help identify patients with solid tumors that are deficient in DNA mismatch repair (MMR) who may be eligible for anti-PD-1 immunotherapy.
- https://seekingalpha.com/news/3731933-roche-announces-fda-approval-of-companion-diagnostic-for-dmmr-solid-tumors
- https://seekingalpha.com/symbol/RHHBY
- https://www.roche.com/
- https://diagnostics.roche.com/global/en/products/tests/ventana-mmr-rxdx-panel.html
Impella 2.5® and Impella CP® devices
FDA Grants Breakthrough Device Designation to Impella ECP, the World’s Smallest Heart Pump
Play Video
This animation shows Impella ECP, the world's smallest heart pump. Impella ECP is placed percutaneously into the heart's left ventricle, expands, and supports the heart’s pumping function, providing flow greater than 3.5 L/min.
August 18, 2021 08:02 AM Eastern Daylight Time
DANVERS, Mass.--(BUSINESS WIRE)--The United States Food and Drug Administration (FDA) has granted breakthrough device designation to Abiomed’s (NASDAQ: ABMD) Impella ECP expandable percutaneous heart pump. The designation means the FDA will prioritize Impella ECP’s regulatory review processes including design iterations, clinical study protocols and pre-market approval (PMA) application.
Impella ECP is the smallest heart pump in the world and the first to be compatible with small bore access and closure techniques. It measures 9 French (3 millimeters) in diameter upon insertion and removal from the body. While in the heart, it expands to support the heart’s pumping function, providing flow greater than 3.5 L/min.
The FDA granted breakthrough device designation in part based on positive clinical data from the first 21 Impella ECP patients treated as part of an FDA regulated early feasibility study. In granting the designation, the FDA determined Impella ECP meets the FDA’s stringent requirements for a breakthrough device.
“This is yet another validation from the FDA of the clinical benefits of Impella technology and an affirmation of the innovative nature of Impella ECP which, due to its smaller vascular access size, has the potential to provide even safer procedures and be available to more patients who need hemodynamic support for coronary revascularization,” said Chuck Simonton, MD, Abiomed’s chief medical officer.
In the United States, an estimated 440,000 patients are indicated and yet undertreated for high-risk PCI. Impella ECP’s size may enable more physicians to provide critical hemodynamic support to coronary artery disease patients who need it.
The first Impella ECP patient in the world is Robert Matthews, an 80-year-old retired auto worker from Detroit, a father of four, and grandfather of eleven. For more than 20 years, Robert lived with heart disease and endured multiple procedures. In 2020, he was referred to Amir Kaki, MD, an interventional cardiologist and director of mechanical circulatory support at Ascension St. John Hospital in Detroit. Dr. Kaki discovered multiple blockages and poor heart function and identified Robert as an appropriate candidate for a Protected PCI with Impella. Robert became the first patient in the world treated with Impella ECP when Dr. Kaki inserted the heart pump prior to opening blockages and placing stents.
Two days later, Robert returned home, and his family and friends immediately noticed his renewed energy. Today, Robert is grateful for the cutting-edge technology that restored his quality of life.
Caution: Impella ECP is an investigational device, limited by federal law to investigational use only.
ABOUT IMPELLA HEART PUMPS
The Impella 2.5® and Impella CP® devices are U.S. FDA approved to treat certain advanced heart failure patients undergoing elective and urgent percutaneous coronary interventions (PCI), such as stenting or balloon angioplasty, to reopen blocked coronary arteries.
The Impella 2.5, Impella CP, Impella CP with SmartAssist®, Impella 5.0®, Impella LD®, and Impella 5.5® with SmartAssist® are U.S. FDA approved to treat heart attack or cardiomyopathy patients in cardiogenic shock, and have the unique ability to enable native heart recovery, allowing patients to return home with their own heart.
For additional information, please visit: www.abiomed.com.
Impella®
The World's Smallest Heart Pump
https://www.abiomed.com/products-and-services/impella
FDA grants breakthrough device status to Abiomed's Impella ECP heart pump
Aug. 18, 2021 8:41 AM ETAbiomed, Inc. (ABMD)
By: Aakash Babu, SA News Editor2 Comments
- The U.S. FDA has granted breakthrough device designation to Abiomed's (NASDAQ:ABMD) Impella ECP expandable percutaneous heart pump.
- https://seekingalpha.com/news/3731803-fda-grants-breakthrough-device-status-to-abiomeds-impella-ecp-heart-pump
- https://seekingalpha.com/symbol/ABMD
- https://www.heartrecovery.com/products-and-services/impella/impella-25
- https://www.abiomed.com/
KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib)
FDA Approves KEYTRUDA® (pembrolizumab) Plus LENVIMA® (lenvatinib) Combination for First-Line Treatment of Adult Patients With Advanced Renal Cell Carcinoma (RCC)
August 11, 2021 6:30 pm ET
KEYTRUDA Plus LENVIMA Is Now Approved for Two Types of Cancer, Including Advanced RCC
Based on Phase 3 CLEAR/KEYNOTE-581 Trial, KEYTRUDA Plus LENVIMA Significantly Reduced Risk of Disease Progression or Death by 61% Versus Sunitinib
KENILWORTH, N.J. & WOODCLIFF LAKE, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, and Eisai today announced that the U.S. Food and Drug Administration (FDA) has approved the combination of KEYTRUDA, Merck’s anti-PD-1 therapy, plus LENVIMA, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC). The approval is based on results from the pivotal Phase 3 CLEAR (Study 307)/KEYNOTE-581 trial, in which KEYTRUDA plus LENVIMA demonstrated statistically significant improvements versus sunitinib in the efficacy outcome measures of progression-free survival (PFS), overall survival (OS) and confirmed objective response rate (ORR).
For PFS, KEYTRUDA plus LENVIMA reduced the risk of disease progression or death by 61% (HR=0.39 [95% CI: 0.32-0.49]; p<0.0001) with a median PFS of 23.9 months versus 9.2 months for sunitinib. For OS, KEYTRUDA plus LENVIMA reduced the risk of death by 34% (HR=0.66 [95% CI: 0.49-0.88]; p=0.0049) versus sunitinib. Additionally, the confirmed ORR was 71% (95% CI: 66-76) (n=252) for patients who received KEYTRUDA plus LENVIMA versus 36% with sunitinib (95% CI: 31-41) (n=129). KEYTRUDA plus LENVIMA achieved a complete response (CR) rate of 16% and partial response (PR) rate of 55% versus a CR rate of 4% and a PR rate of 32% for those who received sunitinib.
Data Supporting the Approval
The approvalwas based on data from the CLEAR (Study 307)/KEYNOTE-581 trial (ClinicalTrials.gov, NCT02811861), a Phase 3, multicenter, open-label, randomized trial conducted in 1,069 patients with advanced RCC in the first-line setting. Patients were enrolled regardless of PD-L1 tumor expression status. Patients with active autoimmune disease or a medical condition that required immunosuppression were ineligible. Randomization was stratified by geographic region (North America and Western Europe vs. “Rest of the World”) and Memorial Sloan Kettering Cancer Center (MSKCC) prognostic groups (favorable vs. intermediate vs. poor risk).
Patients were randomized (1:1:1) to one of the following treatment arms:
- LENVIMA (20 mg orally once daily) in combination with KEYTRUDA (200 mg intravenously [IV] every three weeks for up to 24 months); or
- LENVIMA (18 mg orally once daily) in combination with everolimus (5 mg orally once daily); or
- Sunitinib (50 mg orally once daily for four weeks on treatment, followed by two weeks off treatment).
Treatment continued until unacceptable toxicity or disease progression. Administration of KEYTRUDA plus LENVIMA was permitted beyond Response Evaluation Criteria in Solid Tumors (RECIST)-defined disease progression if the patient was clinically stable and considered by the investigator to be deriving clinical benefit. KEYTRUDA was continued for a maximum of 24 months; however, treatment with LENVIMA could be continued beyond 24 months. Assessment of tumor status was performed at baseline and then every eight weeks.
The study population characteristics were: median age of 62 years (range: 29 to 88 years), 42% age 65 or older; 75% male; 74% White, 21% Asian, 1% Black, and 2% other races; 18% and 82% of patients had a baseline Karnofsky Performance Status (KPS) of 70 to 80 and 90 to 100, respectively; patient distribution by MSKCC risk categories was 27% favorable, 64% intermediate, and 9% poor. Common sites of metastases in patients were lung (68%), lymph node (45%), and bone (25%).
About KEYTRUDA® (pembrolizumab) Injection, 100 mg
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,500 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of patients with melanoma with involvement of lymph node(s) following complete resection.
Non-Small Cell Lung Cancer
KEYTRUDA, in combination with pemetrexed and platinum chemotherapy, is indicated for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations.
KEYTRUDA, in combination with carboplatin and either paclitaxel or paclitaxel protein-bound, is indicated for the first-line treatment of patients with metastatic squamous NSCLC.
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with NSCLC expressing PD-L1 [tumor proportion score (TPS) ≥1%] as determined by an FDA-approved test, with no EGFR or ALK genomic tumor aberrations, and is
- stage III where patients are not candidates for surgical resection or definitive chemoradiation, or
- metastatic.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with metastatic NSCLC whose tumors express PD-L1 (TPS ≥1%) as determined by an FDA-approved test, with disease progression on or after platinum-containing chemotherapy. Patients with EGFR or ALK genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving KEYTRUDA.
Head and Neck Squamous Cell Cancer
KEYTRUDA, in combination with platinum and fluorouracil (FU), is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent head and neck squamous cell carcinoma (HNSCC).
KEYTRUDA, as a single agent, is indicated for the first-line treatment of patients with metastatic or with unresectable, recurrent HNSCC whose tumors express PD-L1 [combined positive score (CPS) ≥1)] as determined by an FDA-approved test.
KEYTRUDA, as a single agent, is indicated for the treatment of patients with recurrent or metastatic HNSCC with disease progression on or after platinum-containing chemotherapy.
Classical Hodgkin Lymphoma
KEYTRUDA is indicated for the treatment of adult patients with relapsed or refractory classical Hodgkin lymphoma (cHL).
KEYTRUDA is indicated for the treatment of pediatric patients with refractory cHL, or cHL that has relapsed after 2 or more lines of therapy.
Primary Mediastinal Large B-Cell Lymphoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with refractory primary mediastinal large B-cell lymphoma (PMBCL), or who have relapsed after 2 or more prior lines of therapy. KEYTRUDA is not recommended for treatment of patients with PMBCL who require urgent cytoreductive therapy.
Urothelial Carcinoma
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic urothelial carcinoma (mUC) who are not eligible for cisplatin-containing chemotherapy and whose tumors express PD-L1 (CPS ≥10), as determined by an FDA-approved test, or in patients who are not eligible for any platinum-containing chemotherapy regardless of PD-L1 status. This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
KEYTRUDA is indicated for the treatment of patients with locally advanced or mUC who have disease progression during or following platinum-containing chemotherapy or within 12 months of neoadjuvant or adjuvant treatment with platinum-containing chemotherapy.
KEYTRUDA is indicated for the treatment of patients with Bacillus Calmette-Guerin-unresponsive, high-risk, non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ with or without papillary tumors who are ineligible for or have elected not to undergo cystectomy.
Microsatellite Instability-High or Mismatch Repair Deficient Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options.
This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established.
Microsatellite Instability-High or Mismatch Repair Deficient Colorectal Cancer
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic MSI-H or dMMR colorectal cancer (CRC).
Gastric Cancer
KEYTRUDA, in combination with trastuzumab, fluoropyrimidine- and platinum-containing chemotherapy, is indicated for the first-line treatment of patients with locally advanced unresectable or metastatic HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Esophageal Cancer
KEYTRUDA is indicated for the treatment of patients with locally advanced or metastatic esophageal or GEJ (tumors with epicenter 1 to 5 centimeters above the GEJ) carcinoma that is not amenable to surgical resection or definitive chemoradiation either:
- in combination with platinum- and fluoropyrimidine-based chemotherapy, or
- as a single agent after one or more prior lines of systemic therapy for patients with tumors of squamous cell histology that express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Cervical Cancer
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Hepatocellular Carcinoma
KEYTRUDA is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with sorafenib. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Merkel Cell Carcinoma
KEYTRUDA is indicated for the treatment of adult and pediatric patients with recurrent locally advanced or metastatic Merkel cell carcinoma (MCC). This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.
Renal Cell Carcinoma
KEYTRUDA, in combination with axitinib, is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
KEYTRUDA, in combination with LENVIMA, is indicated for the first-line treatment of adult patients with advanced RCC.
Endometrial Carcinoma
KEYTRUDA, in combination with LENVIMA, is indicated for the treatment of patients with advanced endometrial carcinoma that is not MSI-H or dMMR, who have disease progression following prior systemic therapy in any settings and are not candidates for curative surgery or radiation.
Tumor Mutational Burden-High Cancer
KEYTRUDA is indicated for the treatment of adult and pediatric patients with unresectable or metastatic tumor mutational burden-high (TMB-H) [≥10 mutations/megabase] solid tumors, as determined by an FDA-approved test, that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with TMB-H central nervous system cancers have not been established.
Cutaneous Squamous Cell Carcinoma
KEYTRUDA is indicated for the treatment of patients with recurrent or metastatic cutaneous squamous cell carcinoma (cSCC) or locally advanced cSCC that is not curable by surgery or radiation.
Triple-Negative Breast Cancer
KEYTRUDA is indicated for the treatment of patients with high-risk early-stage TNBC in combination with chemotherapy as neoadjuvant treatment, and then continued as a single agent as adjuvant treatment after surgery.
KEYTRUDA, in combination with chemotherapy, is indicated for the treatment of patients with locally recurrent unresectable or metastatic TNBC whose tumors express PD-L1 (CPS ≥10) as determined by an FDA-approved test.
Please see Prescribing Information for KEYTRUDA (pembrolizumab) at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_pi.pdf and Medication Guide for KEYTRUDA at http://www.merck.com/product/usa/pi_circulars/k/keytruda/keytruda_mg.pdf.
About LENVIMA® (lenvatinib); available as 10 mg and 4 mg capsules
LENVIMA® (lenvatinib) is a kinase inhibitor that is indicated:
- For the treatment of patients with locally recurrent or metastatic, progressive, radioactive iodine-refractory differentiated thyroid cancer (DTC)
- In combination with KEYTRUDA, for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC)
- In combination with everolimus, for the treatment of adult patients with advanced renal cell carcinoma (RCC) following one prior anti-angiogenic therapy
- For the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)
- In combination with KEYTRUDA, for the treatment of patients with advanced endometrial carcinoma (EC) that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
LENVIMA, discovered and developed by Eisai, is a kinase inhibitor that inhibits the kinase activities of vascular endothelial growth factor (VEGF) receptors VEGFR1 (FLT1), VEGFR2 (KDR), and VEGFR3 (FLT4). LENVIMA inhibits other kinases that have been implicated in pathogenic angiogenesis, tumor growth, and cancer progression in addition to their normal cellular functions, including fibroblast growth factor (FGF) receptors FGFR1-4, the platelet derived growth factor receptor alpha (PDGFRα), KIT, and RET. The combination of LENVIMA and everolimus showed increased anti-angiogenic and anti-tumor activity as demonstrated by decreased human endothelial cell proliferation, tube formation, and VEGF signaling in vitro and tumor volume in mouse xenograft models of human renal cell cancer greater than each drug alone. In syngeneic mouse tumor models, lenvatinib decreased tumor-associated macrophages, increased activated cytotoxic T cells, and demonstrated greater antitumor activity in combination with an anti-PD-1 monoclonal antibody compared to either treatment alone.
For more information, visit www.merck.com
For more information about Eisai, please visit www.eisai.com (for global headquarters: Eisai Co., Ltd.), us.eisai.com (for U.S. headquarters: Eisai, Inc.) or www.eisai.eu (for Europe, Middle East, Africa, Russia, Australia, and New Zealand headquarters: Eisai Europe Ltd.), and connect with us on Twitter (U.S. and global) and LinkedIn (for U.S. and EMEA).
View source version on businesswire.com: https://www.businesswire.com/news/home/20210811005902/en/
Source: Merck & Co., Inc.
FDA Approves LENVIMA® (lenvatinib) Plus KEYTRUDA® (pembrolizumab) Combination for First-Line Treatment of Adult Patients With Advanced Renal Cell Carcinoma (RCC)LENVIMA Plus KEYTRUDA Is Now Approved for Two Types of Cancer, Including Advanced RCC
Based on Phase 3 CLEAR/KEYNOTE-581 Trial, LENVIMA Plus KEYTRUDA Significantly Reduced Risk of Disease Progression or Death by 61% Versus Sunitinib
- August 12, 2021
TOKYO and KENILWORTH, N.J., Aug. 12, 2021 – Eisai (Headquarters: Tokyo, CEO: Haruo Naito) and Merck & Co., Inc., Kenilworth, N.J., U.S.A. (known as MSD outside the United States and Canada)
https://www.eisai.com/news/2021/news202169.html
FDA approves Merck's Keytruda/Lenvima combo for renal cell carcinoma
Aug. 12, 2021 2:12 AM ET Merck & Co., Inc. (MRK) By: Mamta Mayani, SA News Editor2 Comments
- Merck (NYSE:MRK) and Eisai announce that the FDA has approved the combination of Keytruda plus Lenvima, the orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).
- https://www.merck.com/news/fda-approves-keytruda-pembrolizumab-plus-lenvima-lenvatinib-combination-for-first-line-treatment-of-adult-patients-with-advanced-renal-cell-carcinoma-rcc/
- https://seekingalpha.com/symbol/MRK
- https://www.eisai.com/index.html
QARZIBA® (Dinutuximab Beta)
BeiGene and EUSA Pharma Announce China NMPA Approval of QARZIBA® (Dinutuximab Beta) for Patients with High-Risk Neuroblastoma
Aug 17, 2021 7:00 AM
CAMBRIDGE, Mass. & BEIJING & HEMEL HEMPSTEAD, England--(BUSINESS WIRE)-- BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160) and EUSA Pharma (UK), Ltd. today announced that the China National Medical Products Administration (NMPA) has granted QARZIBA® (dinutuximab beta) conditional approval for the treatment of high-risk neuroblastoma in patients aged 12 months and above who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with a history of relapsed or refractory (R/R) neuroblastoma with or without residual disease. Dinutuximab beta is a targeted immunotherapy approved by the European Medicines Agency (EMA).i
“Dinutuximab beta represents an important biologic therapy for pediatric patients in China, having been listed in the first batch of New Drugs in Urgent Clinical Need Marketed Overseas by the NMPA,” commented Xiaobin Wu, Ph.D., President, Chief Operating Officer, and General Manager of China at BeiGene. “For these young patients fighting neuroblastoma in China, we are proud to bring the first approved treatment.”
“We are delighted that the benefit of dinutuximab beta has been recognized in China. This approval represents an important milestone in our mission and collaboration with BeiGene of bringing innovative cancer and rare disease therapies to patients,” said Carsten Thiel, Ph.D., Chief Executive Officer of EUSA Pharma.
The approval of dinutuximab beta in China for the treatment of patients with high-risk neuroblastoma was supported by clinical results available from key trials conducted by SIOPEN (The International Society of Paediatric Oncology Europe Neuroblastoma Group) in collaboration with APEIRON Biologics and EUSA Pharma. These randomized controlled trials evaluated the efficacy of dinutuximab beta by comparing the administration of dinutuximab beta with and without interleukin-2 (IL-2) in the first-line treatment of patients with high-risk neuroblastoma and in two single-arm studies in the R/R setting. In the SIOPEN trial (HR-NBL1), the five-year event-free survival (EFS) rate in patients treated with dinutuximab beta was 57% vs. 42% of historical controls (p<0.01) and the five-year overall survival (OS) rate was 64% vs. 50% (p≤0.0001).ii The safety of dinutuximab beta has been evaluated in 514 patients. The most common adverse reactions were pyrexia and pain that occurred despite analgesic treatment. Other frequent adverse reactions were hypersensitivity, vomiting, diarrhea, capillary leak syndrome, and hypotension.
About QARZIBA® (dinutuximab beta)
QARZIBA® is a monoclonal antibody that is specifically directed against the carbohydrate moiety of disialoganglioside 2 (GD2), which is overexpressed on neuroblastoma cells. Dinutuximab beta was approved by the European Commission in 2017 (See EMA Summary of Product Characteristics (SmPC)) and is indicated for the treatment of high-risk neuroblastoma in patients aged 12 months and above, who have previously received induction chemotherapy and achieved at least a partial response, followed by myeloablative therapy and stem cell transplantation, as well as patients with a history of relapsed or refractory neuroblastoma, with or without residual disease. Prior to the treatment of relapsed neuroblastoma, any actively progressing disease should be stabilized by other suitable measures.
For more information please visit www.eusapharma.com.
To learn more about BeiGene, please visit www.beigene.com
View source version on businesswire.com: https://www.businesswire.com/news/home/20210817005501/en/
Source: BeiGene, Ltd.
BeiGene's Qarziba OK'd in China for patients with high-risk neuroblastoma
Aug. 17, 2021 7:31 AM ET BeiGene, Ltd. (BGNE)
By: Mamta Mayani, SA News Editor
- BeiGene (NASDAQ:BGNE) and EUSA Pharma (UK) announce that the China National Medical Products Administration (NMPA) has granted QARZIBA (dinutuximab beta) conditional approval for the treatment of high-risk neuroblastoma in patients aged 12 months and above.
- Dinutuximab beta is a targeted immunotherapy approved by the EMA.
- https://seekingalpha.com/news/3731303-beigenes-qarziba-okd-in-china-for-phttps://seekingalpha.com/symbol/BGNEatients-with-high-risk-neuroblastoma
- https://eusapharma.com/research-areas
Lyumjev® Rapid-Acting Insulin for Use with Omnipod® Products
Aug 16, 2021
Insulet Announces FDA Clearance of Lyumjev® Rapid-Acting Insulin for Use with Omnipod® Products in the United States
ACTON, Mass.--(BUSINESS WIRE)--Aug. 16, 2021-- Insulet Corporation (NASDAQ: PODD) (Insulet or the Company), the leader in tubeless insulin pump technology with its Omnipod® brand of products, today announced FDA clearance for use of Eli Lilly and Company’s (NYSE: LLY) Lyumjev® (insulin lispro-aabc injection) 100 units/mL with Insulet’s Omnipod® Insulin Management System and Omnipod DASH® Insulin Management System. Insulet is the only insulin pump manufacturer in the United States to obtain this indication, providing more flexibility for people with diabetes.
Lyumjev, approved by the FDA in June 2020, is a formulation of insulin lispro developed by Eli Lilly and Company to speed the absorption of insulin into the blood stream. As a rapid-acting insulin, Lyumjev helps control glucose levels after meals in adults with diabetes, so it more closely matches a body’s typical insulin response to a meal.
The FDA has approved an expanded label for the rapid-acting insulin to include administration through continuous subcutaneous insulin infusion (CSII) with an insulin pump, providing more options for people with diabetes to manage their glucose levels and reduce post-meal elevations. Omnipod is the only insulin pump cleared for use with Lyumjev in the United States.
“Rapid-acting insulin is a popular option for people who want added flexibility in their meal-time diabetes regimen,” said Dr. Trang Ly MBBS, FRACP, PhD, Insulet Senior Vice President and Medical Director. “We are thrilled to provide additional choice to the diabetes community, making their lives with diabetes even simpler.”
Insulet collaborated with Lilly to add Lyumjev to the label of the Omnipod Insulin Management System and the Omnipod DASH Insulin Management System.
Please see Lyumjev Full Prescribing Information including Patient Prescribing Information. For additional questions, please call the Lilly Answer Center at 1-800-545-5979.
For more information, please visit insulet.com and omnipod.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20210816005133/en/
Source: Insulet Corporation
FDA clears Insulet's Omnipod insulin management systems for use with Eli Lilly's Lyumjev
Aug. 16, 2021 7:39 AM ET Eli Lilly and Company (LLY), PODD By: Aakash Babu, SA News Editor
- The U.S. FDA has cleared the use of Insulet's (NASDAQ:PODD) Omnipod and Omnipod Dash insulin management systems with Eli Lilly's Lyumjev (insulin lispro-aabc injection) 100 units/mL in certain patients with type 1 and type 2 diabetes.
- https://seekingalpha.com/symbol/PODD
- https://seekingalpha.com/symbol/LLY
- https://www.lyumjev.com/
- https://www.insulet.com/
- https://www.omnipod.com/
KEYTRUDA® (pembrolizumab)
FDA Accepts Application for Merck’s KEYTRUDA® (pembrolizumab) as Single Agent for Certain Patients With MSI-H/dMMR Advanced Endometrial Carcinoma
August 10, 2021 6:45 am ET
Data Supporting Application to Be Presented at ESMO Congress 2021
KENILWORTH, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside the United States and Canada, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review a new supplemental Biologics License Application (sBLA) seeking approval for KEYTRUDA, Merck’s anti-PD-1 therapy, as a single agent for the treatment of patients with advanced endometrial carcinoma that is microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation. The application is based on overall response data from Cohorts D and K of the KEYNOTE-158 trial, which will be presented for the first time at the European Society for Medical Oncology (ESMO) Congress 2021 (Abstract #795P). The FDA has set a Prescription Drug User Fee Act (PDUFA), or target action, date of March 28, 2022.
“The FDA’s acceptance of our application adds to our momentum of advancing new treatment options to address the most challenging cancers facing women,” said Dr. Scot Ebbinghaus, vice president, clinical research, Merck Research Laboratories. “KEYTRUDA monotherapy is already playing a role for the treatment of certain patients with advanced endometrial carcinoma through the tumor-agnostic MSI-H indication which received accelerated approval four years ago. We look forward to sharing the latest results from KEYNOTE-158, including updated data for KEYTRUDA in certain types of MSI-H/dMMR advanced endometrial carcinoma, at the ESMO Congress in September.”
KEYTRUDA is approved for the treatment of adult and pediatric patients with unresectable or metastatic MSI-H or dMMR solid tumors that have progressed following prior treatment and who have no satisfactory alternative treatment options. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval may be contingent upon verification and description of clinical benefit in the confirmatory trials. The safety and effectiveness of KEYTRUDA in pediatric patients with MSI-H central nervous system cancers have not been established. This accelerated approval was based on efficacy data from patients enrolled in one of five open-label, multi-cohort trials, including KEYNOTE-158.