Moorestown, New Jersey, United States
biotecMAX™ 2023 Insight
TEZSPIRE® (tezepelumab-ekko)
TEZSPIRE® APPROVED FOR SELF-ADMINISTRATION IN THE U.S. WITH A NEW PRE-FILLED PEN
Now Offers Patients the Choice of Administration at Home or in a Doctor's Office
THOUSAND OAKS, Calif., Feb. 2, 2023 /PRNewswire/ -- Amgen (NASDAQ:AMGN) and AstraZeneca today announced the U.S. Food and Drug Administration (FDA) has approved TEZSPIRE® (tezepelumab-ekko) for self-administration in a pre-filled, single-use pen for patients aged 12 years and older with severe asthma.1 First approved by the FDA in December 2021, TEZSPIRE is the only biologic approved for severe asthma with no phenotype (e.g., eosinophilic or allergic) or biomarker limitation within its approved label.2-9
"People with severe asthma will now have the flexibility to administer TEZSPIRE at home or continue to receive their medicine in their doctor's office," said Murdo Gordon, executive vice president of Global Commercial Operations at Amgen. "This approval reinforces our continued efforts to improve accessibility to TEZSPIRE, a first-in-class medicine proven to consistently and significantly reduce exacerbations across a broad population of people with severe asthma."
The approval by the FDA was based on results from the PATHFINDER clinical trial program, which included results from the PATH-BRIDGE Phase 1 trial and the PATH-HOME trial Phase 3 trial.10 The majority (92%) of healthcare providers, patients and caregivers were able to successfully administer TEZSPIRE both in the clinic and at home throughout the PATH-HOME trial. The improvements in asthma control and the safety profile of TEZSPIRE observed in the PATH-HOME trial were consistent with previous clinical trials.10
"Severe asthma continues to be a very complex condition to manage, so we welcome the TEZSPIRE pre-filled pen as an option that will empower patients and healthcare providers with increased choice," said Kenneth Mendez, president and chief executive officer of the Asthma and Allergy Foundation of America. "We believe self-administration alternatives can play an important role in patients' lives and address unmet needs for those living with severe asthma."
The most common adverse reactions (incidence ≥3% and more common than placebo) of TEZSPIRE are pharyngitis, arthralgia, and back pain.1
TEZSPIRE self-administration and the TEZSPIRE pre-filled pen are also approved in the European Union (EU) and are under regulatory review in several other countries around the world. TEZSPIRE is currently approved for the treatment of severe asthma in the U.S., EU, Japan and other countries.
TEZSPIRE® (tezepelumab-ekko) U.S. Indication
TEZSPIRE is indicated for the add-on maintenance treatment of adult and pediatric patients aged 12 years and older with severe asthma.
TEZSPIRE is not indicated for the relief of acute bronchospasm or status asthmaticus.
TEZSPIRE® (tezepelumab-ekko) Important Safety Information
CONTRAINDICATIONS
Known hypersensitivity to tezepelumab-ekko or excipients.
Please see the full Prescribing Information including Patient Information and Instructions for Use.
About TEZSPIRE® (tezepelumab-ekko)
TEZSPIRE is a 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, TEZSPIRE targets and blocks 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.11,12 TSLP is released in response to multiple triggers associated with asthma exacerbations, including allergens, viruses and other airborne particles.11,12
Expression of TSLP is increased in the airways of patients with asthma and has been correlated with disease severity.11,13 Blocking TSLP may prevent the release of pro-inflammatory cytokines by immune cells, resulting in the prevention of asthma exacerbations and improved asthma control.11,13 By working at the top of the cascade, TEZSPIRE helps stop inflammation at the source and has the potential to treat a broad population of severe asthma patients.11,13
TEZSPIRE is also in development for other potential indications including chronic obstructive pulmonary disease, chronic rhinosinusitis with nasal polyps, chronic spontaneous urticaria and eosinophilic esophagitis (EoE). In October 2021, tezepelumab was granted Orphan Drug Designation by the FDA for the treatment of EoE.
About the Amgen and AstraZeneca Collaboration
In 2020, Amgen and AstraZeneca updated the 2012 collaboration agreement for TEZSPIRE. 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, Amgen and AstraZeneca will jointly commercialize TEZSPIRE in North America. Amgen will record product sales in the U.S., with AstraZeneca recording its share of U.S. profits as Collaboration Revenue. Outside of the U.S., AstraZeneca will record product sales, with Amgen recording profit share as Other/Collaboration revenue.
About Amgen
Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.
Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people's lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world's leading independent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.
Amgen is one of the 30 companies that comprise the Dow Jones Industrial Average and is also part of the Nasdaq-100 index. In 2022, Amgen was named one of the "World's Best Employers" by Forbes and one of "America's 100 Most Sustainable Companies" by Barron's.
For more information, visit Amgen.com and follow us on Twitter, LinkedIn, Instagram, TikTok and YouTube.
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SOURCE Amgen
Amgen, AstraZeneca's asthma therapy Tezspire's self-administration option gets FDA approval
Feb. 02, 2023 9:42 AM ET
By: Ravikash, SA News Editor
- The U.S. Food and Drug Administration (FDA) approved a self-administration option of Amgen (NASDAQ:AMGN) and AstraZeneca's (NASDAQ:AZN) asthma therapy Tezspire.
- https://seekingalpha.com/news/3931285-amgen-astrazenecas-asthma-therapy-tezspires-self-administration-option-gets-fda-approval
- AstraZeneca PLC (AZN), AMGN
- https://www.tezspire.com/
DUPIXENT® (DUPILUMAB)
January 30, 2023 at 1:00 AM EST
DUPIXENT® (DUPILUMAB) APPROVED BY EUROPEAN COMMISSION AS THE FIRST AND ONLY TARGETED MEDICINE INDICATED FOR EOSINOPHILIC ESOPHAGITIS
Approximately 60% of patients aged 12 years and older treated with Dupixent 300 mg weekly in the pivotal trial experienced histological disease remission; patients also significantly improved their ability to swallow compared to placebo
Dupixent is now an option for the approximately 50,000 adults and adolescents living with severe uncontrolled eosinophilic esophagitis in the European Union (EU)
Dupixent now approved to treat five diseases with underlying type 2 inflammation in the EU
TARRYTOWN, N.Y. and PARIS, Jan. 30, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that the European Commission (EC) expanded the marketing authorization for Dupixent® (dupilumab) in the European Union (EU) to treat eosinophilic esophagitis (EoE) in adults and adolescents 12 years and older, weighing at least 40 kg, who are inadequately controlled by, are intolerant to, or who are not candidates for conventional medicinal therapy. EoE is a chronic, progressive inflammatory disease that damages the esophagus and prevents it from working properly. With this approval, Dupixent is the first and only targeted medicine specifically indicated to treat EoE in Europe and the U.S.
“This latest approval establishes Dupixent as the only targeted medicine specifically indicated for eosinophilic esophagitis in the European Union. Dupixent is also the only biologic shown in pivotal trials to help patients achieve histological remission, reduce difficulty swallowing and improve health-related quality of life – all of which are crucial to reducing the burden of this debilitating disease,” said George D. Yancopoulos, M.D., Ph.D., President and Chief Scientific Officer at Regeneron, and a principal inventor of Dupixent. “Since its first approval, Dupixent has redefined the treatment of certain chronic diseases with underlying type 2 inflammation and is now indicated for five conditions in the European Union. We remain committed to investigating Dupixent’s potential in additional diseases in which IL-4 and IL-13 may play a key role.”
“The impact of EoE on a patient’s daily life cannot be overstated – the narrowing and scarring of the esophagus can make something as simple as eating a painful and distressing experience, and may lead to choking and food impaction,” said Naimish Patel, M.D., Head of Global Development, Immunology and Inflammation at Sanofi. “With this latest approval for Dupixent, adults and adolescents in the EU suffering from the chronic and often debilitating symptoms of EoE now have the first and only targeted treatment option clinically proven to reduce both esophageal inflammation and damage, as well as improve swallowing ability, pain and health-related quality of life.”
The EC decision is supported by 52-week data from a Phase 3 trial consisting of three parts (Part A, B and C). Part A and Part B investigated Dupixent 300 mg weekly (Part A n=42; Part B n=80) compared to placebo (Part A n=39; Part B n=79) for 24 weeks. Part C (n=188) observed patients who had continued on or switched to Dupixent from Parts A and B for an additional 28 weeks.
Dupixent patients in Parts A and B, respectively, experienced:
- An approximately 10 times higher rate of histological disease remission (60% and 59%), a co-primary endpoint, compared to placebo (5% and 6%).
- A 69% and 64% reduction in disease symptoms compared to 32% and 41% with placebo. Disease symptoms were measured using the Dysphagia Symptom Questionnaire (DSQ), on which Dupixent patients experienced a 21.9- and 23.8-point clinically meaningful improvement compared to a 9.6- and 13.9-point improvement for placebo, a co-primary endpoint. Swallowing improvement was observed as early as four weeks.
- A greater than seven-fold reduction in abnormal endoscopic findings from baseline (-3.2 and -4.5 points) compared to placebo (-0.3 and -0.6 points).
- Nominally significant improvements in swallowing-related pain and health-related quality of life, as well as less frequent non-swallowing symptoms.
Histological disease remission, swallowing improvement and reduction in abnormal endoscopic findings were consistent with the overall population in patients who were uncontrolled, or not responsive to or not eligible for swallowed topical corticosteroids. Longer term efficacy in Part C was similar to results observed in Parts A and B.
The safety results of the trial were generally consistent with the known safety profile of Dupixent in its approved indications. The most common side effects across indications were injection site reactions, conjunctivitis, conjunctivitis allergic, arthralgia, oral herpes and eosinophilia. Adverse events more commonly observed in EoE patients treated with Dupixent (n=122) compared to placebo (n=117) included infections (32% vs. 25%). An additional adverse reaction of injection site bruising was reported in the EoE trial. The safety profile through 52 weeks was generally consistent with the safety profile observed at 24 weeks.
About the Dupixent Eosinophilic Esophagitis Trial
The three-part Phase 3 randomized, double-blind, placebo-controlled trial evaluated the efficacy and safety of Dupixent in patients aged 12 years and older with EoE. All patients had previously not responded to proton pump inhibitors, and, across Parts A and B, 74% of patients were previously treated with swallowed topical corticosteroids.
At 24 weeks, the co-primary endpoints in Parts A and B assessed patient-reported measures of difficulty swallowing (change from baseline in the DSQ on a 0-84 scale) and esophageal inflammation (proportion of patients achieving histological disease remission, defined as peak esophageal intraepithelial eosinophil count of ≤6 eos/hpf).
Additional endpoints included abnormal endoscopic findings (EoE Endoscopic Reference Score [EoE-EREFS] on a 0-18 scale), swallowing-related pain (DSQ pain score), health-related quality of life (EoE Impact Questionnaire [EoE-IQ]) and frequency of other non-dysphagia symptoms (EoE Symptom Questionnaire [EoE-SQ]).
About Dupixent
Dupixent is an injection administered under the skin (subcutaneous injection) at different injection sites. In the EU for adolescents and adults with EoE, Dupixent is administered at 300 mg every week. It is available as both a pre-filled pen and pre-filled syringe at the 300 mg dose. Dupixent is intended for use under the guidance of a healthcare professional and can be given in a clinic or at home by self-administration after training by a healthcare professional.
Dupixent, which was invented using Regeneron's proprietary VelocImmune® technology, is a fully human monoclonal antibody that inhibits the signaling of the interleukin-4 (IL-4) and interleukin-13 (IL-13) pathways and is not an immunosuppressant. The Dupixent development program has shown significant clinical benefit and a decrease in type 2 inflammation in Phase 3 trials, establishing that IL-4 and IL-13 are key and central drivers of the type 2 inflammation that plays a major role in multiple related and often co-morbid diseases. These diseases include approved indications for Dupixent, such as atopic dermatitis, asthma, chronic rhinosinusitis with nasal polyposis (CRSwNP), prurigo nodularis and EoE.
Dupixent has received regulatory approvals in one or more countries around the world for use in certain patients with atopic dermatitis, asthma, CRSwNP, EoE or prurigo nodularis in different age populations. Dupixent is currently approved for one or more of these indications in more than 60 countries, including in Europe, the U.S. and Japan. More than 500,000 patients have been treated with Dupixent globally.
About Regeneron's VelocImmune Technology
Regeneron's VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron's co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved or authorized fully human monoclonal antibodies. This includes REGEN-COV® (casirivimab and imdevimab), Dupixent, Libtayo® (cemiplimab-rwlc), Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb) and Inmazeb® (atoltivimab, maftivimab and odesivimab-ebgn).
Dupilumab Development Program
Dupilumab is being jointly developed by Regeneron and Sanofi under a global collaboration agreement. To date, dupilumab has been studied across more than 60 clinical trials involving more than 10,000 patients with various chronic diseases driven in part by type 2 inflammation.
In addition to the currently approved indications, Regeneron and Sanofi are studying dupilumab in a broad range of diseases driven by type 2 inflammation or other allergic processes in Phase 3 trials, including pediatric EoE, hand and foot atopic dermatitis, chronic inducible urticaria-cold, chronic spontaneous urticaria, chronic pruritus of unknown origin, chronic obstructive pulmonary disease with evidence of type 2 inflammation, chronic rhinosinusitis without nasal polyposis, allergic fungal rhinosinusitis, allergic bronchopulmonary aspergillosis, and bullous pemphigoid. These potential uses of dupilumab are currently under clinical investigation, and the safety and efficacy in these conditions have not been fully evaluated by any regulatory authority.
U.S. INDICATIONS
DUPIXENT is a prescription medicine used:
- to treat adults and children 6 months of age and older with moderate-to-severe eczema (atopic dermatitis or AD) that is not well controlled with prescription therapies used on the skin (topical), or who cannot use topical therapies. DUPIXENT can be used with or without topical corticosteroids. It is not known if DUPIXENT is safe and effective in children with atopic dermatitis under 6 months of age.
- with other asthma medicines for the maintenance treatment of moderate-to-severe eosinophilic or oral steroid dependent asthma in adults and children 6 years of age and older whose asthma is not controlled with their current asthma medicines. DUPIXENT helps prevent severe asthma attacks (exacerbations) and can improve your breathing. DUPIXENT may also help reduce the amount of oral corticosteroids you need while preventing severe asthma attacks and improving your breathing. DUPIXENT is not used to treat sudden breathing problems. It is not known if DUPIXENT is safe and effective in children with asthma under 6 years of age.
- with other medicines for the maintenance treatment of chronic rhinosinusitis with nasal polyposis (CRSwNP) in adults whose disease is not controlled. It is not known if DUPIXENT is safe and effective in children with chronic rhinosinusitis with nasal polyposis under 18 years of age.
- to treat adults and children 12 years of age and older, who weigh at least 88 pounds (40 kg), with eosinophilic esophagitis (EoE). It is not known if DUPIXENT is safe and effective in children with eosinophilic esophagitis under 12 years of age and who weigh at least 88 pounds (40 kg).
- to treat adults with prurigo nodularis (PN). It is not known if DUPIXENT is safe and effective in children with prurigo nodularis under 18 years of age.
Please see accompanying full Prescribing Information including Patient Information.
About Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led for 35 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to nine FDA-approved treatments and numerous product candidates in development, almost all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, pain, hematologic conditions, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite® technologies, such as VelocImmune®, which uses unique genetically humanized mice to produce optimized fully human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center, which is conducting one of the largest genetics sequencing efforts in the world.
For more information, please visit www.Regeneron.com or follow @Regeneron on Twitter.
About Sanofi
We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions.
Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY.
Source: Regeneron Pharmaceuticals, Inc.
Sanofi, Regeneron Dupixent gets approval in EU for expanded use in eosinophilic esophagitis
Jan. 30, 2023 4:26 AM ET
By: Ravikash, SA News Editor
The European Commission (EC) approved Sanofi (NASDAQ:SNY) and Regeneron Pharmaceuticals' (NASDAQ:REGN) Dupixent to treat eosinophilic esophagitis (EoE) in patients 12 years and older, weighing at least 40 kg, who are inadequately controlled by, are intolerant to, or who are not eligible for conventional medicinal therapy.
Hemlibra® (emicizumab)
European Commission approves label expansion of Roche’s Hemlibra to include people with moderate haemophilia A in the EUFebruary 01, 2023
- Hemlibra, already approved for severe haemophilia A in the EU, will now also provide an effective and convenient prophylactic treatment option for people with moderate haemophilia A
- Moderate haemophilia A can have a significant impact on the lives of people affected, with only 15% living a bleed-free life1
- The approval is based on the HAVEN 6 results, where Hemlibra demonstrated effective bleed control and a favourable safety profile in people with moderate haemophilia A without inhibitors2
Basel, 01 February 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission approved the expansion of the Hemlibra® (emicizumab) European Union (EU) marketing authorisation. The label will now include the routine prophylaxis of bleeding episodes in people with haemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors, who have moderate disease (FVIII ≥1% and ≤ 5%) with a severe bleeding phenotype.3 Haemophilia A affects around 900,000 people worldwide,4,5 approximately 14% of whom have a moderate form of the disease.6
“We welcome the European Commission's decision to approve Hemlibra also for people with moderate haemophilia A in the EU, since even moderate disease can produce bleeds that cause irreversible joint damage and impact quality of life,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “As its benefit expands to broader populations, we remain committed to determining how Hemlibra might help even more people with haemophilia A to live a bleed-free life.”
While the treatment and care of people with severe haemophilia A are well-established, there is less information and guidance on prophylaxis for moderate haemophilia A.7 Additionally, the severity of haemophilia A, traditionally measured by an individual’s factor VIII levels, is not always reflective of bleeding behaviour. Many people with moderate haemophilia A may not be receiving prophylactic treatments and could have a worsened clinical burden. Approximately 85% of people with moderate haemophilia A have bleeds within a given year and one in three have long-term joint problems, many of which require surgery and impact quality of life.1,7,8
This approval is based on the results of the phase III HAVEN 6 trial, in which Hemlibra demonstrated effective bleed control and a favourable safety profile in people with non-severe haemophilia A without factor VIII inhibitors, where prophylaxis was clinically indicated.2 The decision was also based on real world data. This label expansion will provide an effective and convenient prophylactic treatment option with a favourable safety profile for people in the EU with moderate haemophilia A with a severe bleeding phenotype.
Hemlibra is approved as a prophylactic treatment option for people with haemophilia A with factor VIII inhibitors in more than 110 countries and for people without factor VIII inhibitors in more than 100 countries worldwide. It has been studied in one of the largest clinical trial programmes in people with haemophilia A with and without factor VIII inhibitors, including eight phase III studies.
About HAVEN 6
HAVEN 6 is a phase III clinical study designed to evaluate the safety, efficacy, pharmacokinetics and pharmacodynamics of Hemlibra in people with non-severe haemophilia A without factor VIII inhibitors. Data from the primary analysis of HAVEN 6 was presented at the 30th International Society on Thrombosis and Haemostasis (ISTH) Annual Congress, on 11 July 2022.2 The analysis included data from 72 participants, including three women, 51 (70.8%) of whom had moderate haemophilia A without factor VIII inhibitors. The data indicate that Hemlibra has effective bleed control and a favourable safety profile in people with non-severe haemophilia A without factor VIII inhibitors, with no new safety signals identified. Hemlibra achieved clinically meaningful bleed control, with 66.7% of participants experiencing no bleeds that required treatment, 81.9% experiencing no spontaneous bleeds that required treatment, and 88.9% experiencing no joint bleeds that required treatment. Model-based annualised bleed rates (ABR) remained low throughout the evaluation period at 0.9 (95% CI: 0.55-1.52).2
About Hemlibra® (emicizumab)
Hemlibra is a bispecific factor IXa- and factor X-directed antibody. It is designed to bring together factor IXa and factor X, proteins involved in the natural coagulation cascade, and restore the blood clotting process for people with haemophilia A. Hemlibra is a prophylactic (preventative) treatment that can be administered by an injection of a ready-to-use solution under the skin (subcutaneously) once-weekly, every two weeks, or every four weeks (after an initial once-weekly dose for the first four weeks). Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and is being co-developed globally by Chugai, Roche and Genentech. It is marketed in the United States by Genentech as Hemlibra (emicizumab-kxwh), with kxwh as the suffix designated in accordance with Nonproprietary Naming of Biological Products Guidance for Industry issued by the U.S. Food and Drug Administration.9
About Roche in haematology
Roche has been developing medicines for people with malignant and non-malignant blood diseases for more than 20 years; our experience and knowledge in this therapeutic area runs deep. Today, we are investing more than ever in our effort to bring innovative treatment options to patients across a wide range of haematological diseases. Our approved medicines include MabThera®/Rituxan® (rituximab), Gazyva®/Gazyvaro® (obinutuzumab), Polivy® (polatuzumab vedotin), Venclexta®/Venclyxto® (venetoclax) in collaboration with AbbVie, Hemlibra® (emicizumab) and Lunsumio® (mosunetuzumab). Our pipeline of investigational haematology medicines includes T-cell engaging bispecific antibodies glofitamab, targeting both CD20 and CD3, and cevostamab, targeting both FcRH5 and CD3; Tecentriq® (atezolizumab), a monoclonal antibody designed to bind with PD-L1 and crovalimab, an anti-C5 antibody engineered to optimise complement inhibition. Our scientific expertise, combined with the breadth of our portfolio and pipeline, also provides a unique opportunity to develop combination regimens that aim to improve the lives of patients even further.
About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.
In recognising our endeavour to pursue a long-term perspective in all we do, Roche has been named one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices for the thirteenth consecutive year. This distinction also reflects our efforts to improve access to healthcare together with local partners in every country we work.
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.
Roche's hemophilia A therapy Hemlibra gets approval in EU for expanded use
Feb. 02, 2023 5:21 AM ET
Roche Holding AG (RHHBY), RHHBF
By: Ravikash, SA News Editor
- Roche's (OTCQX:RHHBY) (OTCQX:RHHBF) said on Wednesday that the European Commission approved the expanded use of Hemlibra for the routine prevention of bleeding episodes in patients with hemophilia A (congenital factor VIII deficiency) without factor VIII inhibitors who have moderate disease with severe bleeding phenotype.
- https://seekingalpha.com/news/3931060-roches-hemophilia-a-therapy-hemlibra-gets-approval-in-eu-for-expanded-use
- Roche Holding AG (RHHBY), RHHBF
- https://www.roche.com/
- https://www.hemlibra.com/
- https://www.gene.com/
CARVYKTI™ (ciltacabtagene autoleucel)
Janssen Announces Unblinding of Phase 3 CARTITUDE-4 Study of CARVYKTI® (cilta-cel) as Primary Endpoint Met in Treatment of Patients with Relapsed and Refractory Multiple Myeloma
Jan 27, 2023 United States
Study unblinded following recommendation of Independent Data Monitoring Committee
RARITAN, New Jersey, January 27, 2023 – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced that the Phase 3 CARTITUDE-4 study evaluating CARVYKTI® (ciltacabtagene autoleucel; cilta-cel) versus pomalidomide, bortezomib, and dexamethasone (PVd) or daratumumab, pomalidomide, and dexamethasone (DPd) for the treatment of patients with relapsed and lenalidomide-refractory multiple myeloma met its primary endpoint of significant improvement in progression-free survival (PFS) at the first pre-specified interim analysis. As a result of meeting the primary endpoint, the Independent Data Monitoring Committee recommended the unblinding of the study.
“The CARTITUDE-4 study represents the first Phase 3 program in our comprehensive clinical development strategy for CARVYKTI, and further demonstrates our commitment to advance the treatment of patients with relapsed/refractory multiple myeloma,” said Jordan Schecter, M.D., Vice President, Clinical Development Cellular Therapy Program, Janssen Research & Development, LLC. “We look forward to the presentation of the data from the CARTITUDE-4 study at a future medical meeting.”
CARTITUDE-4 (NCT04181827) is the first randomized Phase 3 study evaluating the efficacy and safety of CARVYKTI®. The study compares CARVYKTI® with standard of care treatments PVd or DPd in adult patients with relapsed and lenalidomide-refractory multiple myeloma who received one to three prior lines of therapy. The primary endpoint of the study is PFS; safety, overall survival, minimal residual disease negative rate and overall response rate are secondary endpoints. Patients enrolled in the CARTITUDE-4 study will continue to be followed for primary, secondary and exploratory endpoints for the duration of the study.
Results from the CARTITUDE-4 study will be presented at an upcoming scientific congress and shared with health authorities in planned submission applications.
About CARVYKTI® (ciltacabtagene autoleucel)
CARVYKTI® (cilta-cel) received U.S. Food and Drug Administration approval in February 2022 for the treatment of adults with relapsed or refractory multiple myeloma after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.[1] In May 2022, the European Commission granted conditional marketing authorization of CARVYKTI® (cilta-cel) for the treatment of adults 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. In September 2022, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved CARVYKTI® (cilta-cel) for the treatment of adults with relapsed or refractory multiple myeloma in patients that have no history of CAR-positive T cell infusion therapy targeting BCMA and who have received three or more lines of therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 monoclonal antibody, and in whom multiple myeloma has not responded to or has relapsed following the most recent therapy.
CARVYKTI® is a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding chimeric antigen receptor (CAR) that directs the CAR positive T-cells to eliminate cells that express BCMA. BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B cells and plasma cells. The CARVYKTI® CAR protein features two BCMA-targeting single domains designed to confer high avidity against human BCMA. Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.
In December 2017, Janssen Biotech, Inc. entered into an exclusive worldwide license and collaboration agreement with Legend Biotech USA, Inc. to develop and commercialize CARVYKTI®.
For more information, visit www.CARVYKTI.com.
CARVYKTI® INDICATIONS AND USAGE
CARVYKTI® (ciltacabtagene autoleucel) is a B-cell maturation antigen (BCMA)-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory multiple myeloma, after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
Further information is available at www.CARVYKTIrems.com or 1-844-672-0067.
Please read full Prescribing Information including Boxed Warning for CARVYKTI®.
About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we're creating a future where disease is a thing of the past. We're the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.
Learn more at www.janssen.com. Follow us at @JanssenUS and @JanssenGlobal. Janssen Research & Development, LLC and Janssen Biotech, Inc. are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
JNJ/ Legend CAR-T therapy succeeds in Phase 3 multiple myeloma trial
Jan. 27, 2023 9:09 AM ET
Legend Biotech Corporation (LEGN), JNJ
By: Dulan Lokuwithana, SA News Editor
- Legend Biotech (NASDAQ:LEGN) announced Friday that Carvykti, the CAR-T cell therapy that the company commercializes with Johnson & Johnson (NYSE:JNJ), met the primary endpoint in a Phase 3 trial for patients with multiple myeloma.
- https://seekingalpha.com/news/3928956-jnj-legend-car-t-therapy-succeeds-late-stage-multiple-myeloma-trial
- Legend Biotech Corporation (LEGN), JNJ
- https://www.carvykti.com/
- https://www.janssen.com/
ENHERTU® (fam-trastuzumab deruxtecan-nxki) for injection
Enhertu approved in the EU as the first HER2-directed therapy for patients with HER2-low metastatic breast cancer
PUBLISHED26 January 2023 26 January 2023 07:00 GMT
Approval based on DESTINY-Breast04 results where AstraZeneca and Daiichi Sankyo’s Enhertu reduced the risk of disease progression or death by 50% and increased overall survival by more than 6 months vs. chemotherapy
AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) has been approved in the European Union (EU) as monotherapy for the treatment of adult patients with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-) breast cancer who have received prior chemotherapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy.
Enhertu is a specifically engineered HER2-directed antibody drug conjugate (ADC) being jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.
The approval by the European Commission follows the positive opinion of the Committee for Medicinal Products for Human Use and is based on results from the DESTINY-Breast04 Phase III trial, which were presented at the American Society of Clinical Oncology 2022 Annual Meeting and published in The New England Journal of Medicine.1
In the trial, Enhertu significantly reduced the risk of disease progression or death by 50% versus physician’s choice of chemotherapy (based on a hazard ratio [HR] of 0.50; 95% confidence interval [CI]: 0.40-0.63; p<0.0001) in patients with HER2-low metastatic breast cancer with hormone receptor (HR)-positive or HR-negative disease. A median progression-free survival (PFS) of 9.9 months was seen with Enhertu versus 5.1 months in those treated with chemotherapy, as assessed by blinded independent central review (BICR). A 36% reduction in the risk of death was seen with Enhertu compared to chemotherapy (hazard ratio of 0.64; 95% CI: 0.49-0.84; p=0.001) with a median overall survival (OS) of 23.4 months versus 16.8 months.
Javier Cortés, MD, PhD, Head, International Breast Cancer Center, Barcelona, Spain, said: “The European approval of Enhertu in the HER2-low metastatic breast cancer population marks the first time we will have the opportunity to treat patients with lower levels of HER2 expression with a HER2-directed therapy. Enhertu has shown a significant improvement in outcomes compared to chemotherapy for these patients, reinforcing its potential to become a new standard of care.”
Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “Historically, patients with breast cancer who have tumours with low levels of HER2 expression have been classified as HER2-negative, giving them limited treatment options beyond chemotherapy. This approval reinforces the important role Enhertu may have for patients with HER2-low disease and highlights the need to evolve the way breast cancer is treated to improve patient outcomes.”
Ken Keller, Global Head of Oncology Business, and President and CEO, Daiichi Sankyo, Inc, said: “The approval of Enhertu in HER2-low metastatic breast cancer represents a significant clinical advance for patients in Europe with both HR-positive and HR-negative disease who previously have had limited treatment options in the late-line setting. This milestone also supports our vision to bring Enhertu to more patients across the HER2 spectrum, which requires a change to the breast cancer classification system that has been guiding treatment for more than two decades.”
The safety profile observed in patients treated with Enhertu in the DESTINY-Breast04 trial was consistent with that seen in other trials of Enhertu in breast cancer with no new safety signals identified.
Financial considerations
Following EU approval, an amount of $150m is due from AstraZeneca to Daiichi Sankyo as a milestone payment for this HER2-low breast cancer post-chemotherapy indication. The milestone payment will be capitalised as an addition to the upfront payment made by AstraZeneca to Daiichi Sankyo in 2019 and subsequent capitalised milestones.
Sales of Enhertu in most EU territories are recognised by Daiichi Sankyo. AstraZeneca reports its share of gross profit margin from Enhertu sales in those territories as collaboration revenue in the Company’s financial statements. AstraZeneca will record product sales in respect of sales made in territories where AstraZeneca is the selling party.
Further details on the financial arrangements were set out in the March 2019 announcement of the collaboration.
DESTINY-Breast04
DESTINY-Breast04 is a global, randomised, open-label, Phase III trial evaluating the efficacy and safety of Enhertu (5.4mg/kg) versus physician’s choice of chemotherapy (capecitabine, eribulin, gemcitabine, paclitaxel or nab-paclitaxel) in patients with HR-positive or HR-negative, HER2-low unresectable and/or metastatic breast cancer previously treated with one or two prior lines of chemotherapy. Patients were randomised 2:1 to receive either Enhertu or chemotherapy.
The primary endpoint of DESTINY-Breast04 is PFS in patients with HR-positive disease based on BICR. Key secondary endpoints include PFS based on BICR in all randomised patients (HR-positive and HR-negative disease), OS in patients with HR-positive disease and OS in all randomised patients (HR-positive and HR-negative disease). Other secondary endpoints include PFS based on investigator assessment, objective response rate based on BICR and on investigator assessment, duration of response based on BICR and safety.
DESTINY-Breast04 enrolled 557 patients at multiple sites in Asia, Europe and North America. For more information about the trial, visit ClinicalTrials.gov.
Enhertu
Enhertu is a HER2-directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, Enhertu is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced programme in AstraZeneca’s ADC scientific platform. Enhertu consists of a HER2 monoclonal antibody attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a stable tetrapeptide-based cleavable linker.
Enhertu (5.4mg/kg) is approved in more than 40 countries for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received a (or one or more) prior anti-HER2-based regimen, either in the metastatic setting or in the neoadjuvant or adjuvant setting, and have developed disease recurrence during or within six months of completing therapy based on the results from the DESTINY-Breast03 trial.
Enhertu (5.4mg/kg) is approved in more than 30 countries for the treatment of adult patients with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ ISH-) breast cancer who have received a prior systemic therapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy based on the results of the DESTINY-Breast04 trial.
Enhertu (5.4mg/kg) is approved under accelerated approval in the US for the treatment of adult patients with unresectable or metastatic non-small cell lung cancer whose tumours have activating HER2 (ERBB2) mutations, as detected by an FDA-approved test, and who have received a prior systemic therapy based on the results from the DESTINY-Lung02 trial. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.
Enhertu (6.4mg/kg) is approved in more than 30 countries for the treatment of adult patients with locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma who have received a prior trastuzumab-based regimen based on the results from the DESTINY-Gastric01 trial and/or DESTINY-Gastric02 trials.
Enhertu development programme
A comprehensive global development programme is underway evaluating the efficacy and safety of Enhertu monotherapy across multiple HER2-targetable cancers including breast, gastric, lung and colorectal cancers. Trials in combination with other anticancer treatments, such as immunotherapy, are also underway.
Daiichi Sankyo collaboration
Daiichi Sankyo Company, Limited (TSE: 4568) [referred to as Daiichi Sankyo] and AstraZeneca entered into a global collaboration to jointly develop and commercialise Enhertu (a HER2-directed ADC) in March 2019, and datopotamab deruxtecan (DS-1062; a TROP2-directed ADC) in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is responsible for the manufacturing and supply of Enhertu and datopotamab deruxtecan.
AstraZeneca in breast cancer
Driven by a growing understanding of breast cancer biology, AstraZeneca is starting to challenge, and redefine, the current clinical paradigm for how breast cancer is classified and treated to deliver even more effective treatments to patients in need – with the bold ambition to one day eliminate breast cancer as a cause of death.
AstraZeneca has a comprehensive portfolio of approved and promising compounds in development that leverage different mechanisms of action to address the biologically diverse breast cancer tumour environment.
With Enhertu (trastuzumab deruxtecan), a HER2-directed ADC, AstraZeneca and Daiichi Sankyo are aiming to improve outcomes in previously treated HER2-positive and HER2-low metastatic breast cancer and are exploring its potential in earlier lines of treatment and in new breast cancer settings.
In HR-positive breast cancer, AstraZeneca continues to improve outcomes with foundational medicines Faslodex (fulvestrant) and Zoladex (goserelin) and aims to reshape the HR-positive space with next-generation SERD and potential new medicine camizestrant as well as a potential first-in-class AKT kinase inhibitor, capivasertib. AstraZeneca is also collaborating with Daiichi Sankyo to explore the potential of TROP2-directed ADC, datopotamab deruxtecan, in this setting.
PARP inhibitor Lynparza (olaparib) is a targeted treatment option that has been studied in early and metastatic breast cancer with an inherited BRCA mutation. AstraZeneca with MSD (Merck & Co., Inc. in the US and Canada) continue to research Lynparza in these settings and to explore its potential in earlier disease.
To bring much-needed treatment options to patients with triple-negative breast cancer, an aggressive form of breast cancer, AstraZeneca is evaluating the potential of datopotamab deruxtecan alone and in combination with immunotherapy Imfinzi (durvalumab), capivasertib in combination with chemotherapy, and Imfinzi in combination with other oncology medicines, including Lynparza and Enhertu.
AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.
The Company's focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.
AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca, Daiichi Sankyo's Enhertu gets approval in EU for breast cancer subtype
Jan. 26, 2023 4:36 AM ET
AstraZeneca PLC (AZN), DSKYF, DSNKY
By: Ravikash, SA News Editor
- The European Commission (EC) approved AstraZeneca (NASDAQ:AZN) and Daiichi Sankyo's (OTCPK:DSKYF) (OTCPK:DSNKY) drug Enhertu to treat certain patients with a type of breast cancer.
- https://seekingalpha.com/news/3928225-astrazeneca-daiichi-sankyos-enhertu-gets-approval-in-eu-for-breast-cancer-subtype
- AstraZeneca PLC (AZN), DSKYF, DSNKY
- https://enhertu.com/en
- https://www.astrazeneca.com/
TRULICITY (dulaglutide) injection
Trulicity
dulaglutide
Table of contents
Opinion
On 26 January 2023, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion recommending a change to the terms of the marketing authorisation for the medicinal product Trulicity. The marketing authorisation holder for this medicinal product is Eli Lilly Nederland B.V.
The CHMP adopted an extension to the existing indication to include children from 10 years of age. For information, the full indication will be as follows:1
Type 2 Diabetes Mellitus
Trulicity is indicated for the treatment of adultspatients 10 years and above with insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise
- as monotherapy when metformin is considered inappropriate due to intolerance or contraindications
- in addition to other medicinal products for the treatment of diabetes.
For study results with respect to combinations, effects on glycaemic control and cardiovascular events, and the populations studied, see sections 4.4, 4.5 and 5.1.
Detailed recommendations for the use of this product will be described in the updated summary of product characteristics (SmPC), which will be published in the revised European public assessment report (EPAR), and will be available in all official European Union languages after a decision on this change to the marketing authorisation has been granted by the European Commission.
1 New text in bold, removed text as strikethrough
- List itemCHMP post-authorisation summary of opinion for Trulicity (II-65) (PDF/107.79 KB) (new)
Adopted
First published: 27/01/2023
EMA/CHMP/26443/2023
Lilly's diabetes drug Trulicity gets EMA committee nod for use in adolescents
Jan. 27, 2023 7:14 AM ET
By: Ravikash, SA News Editor1 Comment
- A committee of the European Medicines Agency (EMA) recommended the expanded approval of Eli Lilly's (NYSE:LLY) blockbuster drug Trulicity to treat patients 10 years of age and above with insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise.
- https://seekingalpha.com/news/3928883-lillys-diabetes-drug-trulicity-gets-ema-committee-nod-for-use-in-adolescents
- Eli Lilly and Company (LLY)
- https://www.trulicity.com/
- https://www.lilly.com/
KEYTRUDA® (pembrolizumab) Plus Chemotherapy
Merck’s KEYTRUDA® (pembrolizumab) Plus Chemotherapy Significantly Improved Overall Survival Versus Chemotherapy in First-Line Advanced or Unresectable Biliary Tract Cancer in KEYNOTE-966 Trial
January 25, 2023 6:45 am ET
RAHWAY, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced positive results from the Phase 3 KEYNOTE-966 trial. In the final analysis of this trial, KEYTRUDA, Merck’s anti-PD-1 therapy, in combination with standard of care chemotherapy (gemcitabine and cisplatin) demonstrated a statistically significant and clinically meaningful improvement in overall survival (OS) versus chemotherapy alone for the first-line treatment of patients with advanced or unresectable biliary tract cancer (BTC). The safety profile of KEYTRUDA in this trial was consistent with that observed in previously reported studies. Results will be presented at an upcoming medical meeting and will be submitted to regulatory authorities.
“Biliary tract cancer is typically diagnosed at an advanced stage, and these patients face a poor prognosis, with five-year survival rates estimated to be approximately 5% to 15%,” said Dr. Eliav Barr, senior vice president, head of global clinical development and chief medical officer, Merck Research Laboratories. “We are very encouraged by these overall survival results that show the potential benefit of KEYTRUDA in addition to chemotherapy for biliary cancer patients who are in urgent need of new treatment options.”
Merck has an extensive clinical development program evaluating KEYTRUDA in gastrointestinal cancers and is continuing to study KEYTRUDA for multiple uses in gastric, hepatobiliary, esophageal, pancreatic and colorectal cancers.
About KEYNOTE-966
KEYNOTE-966 is a randomized, double-blind Phase 3 trial (ClinicalTrials.gov, NCT04003636) evaluating KEYTRUDA in combination with gemcitabine and cisplatin compared to placebo plus gemcitabine and cisplatin for the first-line treatment of advanced and/or unresectable BTC. The primary endpoint was OS, and the secondary endpoints included progression-free survival, objective response rate, duration of response and safety. The trial enrolled 1,069 patients who were randomized to receive KEYTRUDA (200 mg every three weeks for up to approximately two years) plus gemcitabine and cisplatin, or placebo plus gemcitabine and cisplatin.
About Biliary Tract Cancer (BTC)
Biliary tract cancer is a group of rare and highly aggressive cancers in the gallbladder and bile ducts. Biliary tract cancer is the second most common type of primary liver cancer after hepatocellular carcinoma, accounting for 15% of all liver cancers. It is estimated there are approximately 211,000 new cases of BTC diagnosed and 174,000 deaths from the disease each year globally. Biliary tract cancer is most frequently diagnosed in patients between 50 to 70 years old, and 70% of BTC patients are diagnosed at an advanced stage. Patients diagnosed with BTC face a very poor prognosis, as the five-year survival rate is estimated to be between 5% and 15%.
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 adult and pediatric (12 years and older) patients with stage IIB, IIC, or III melanoma 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.
Non-muscle Invasive Bladder Cancer
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, 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 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) as determined by an FDA-approved test.
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 gastroesophageal junction (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, in combination with chemotherapy, with or without bevacizumab, is indicated for the treatment of patients with persistent, recurrent, or metastatic cervical cancer whose tumors express PD-L1 (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 cervical cancer with disease progression on or after chemotherapy whose tumors express PD-L1 (CPS ≥1) as determined by an FDA-approved test.
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 is indicated for the adjuvant treatment of patients with RCC at intermediate-high or high risk of recurrence following nephrectomy, or following nephrectomy and resection of metastatic lesions.
Endometrial Carcinoma
KEYTRUDA, as a single agent, is indicated for the treatment of patients with advanced endometrial carcinoma that is MSI-H or dMMR, as determined by an FDA-approved test, who have disease progression following prior systemic therapy in any setting 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 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.
About Merck
At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
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 .
Source: Merck & Co., Inc.
Merck's Keytruda shows survival benefit in biliary tract cancer, meeting phase 3 study goal
Jan. 25, 2023 7:50 AM ET
By: Ravikash, SA News Editor
Merck's (NYSE:MRK) Keytruda, with standard of care chemotherapy, met the main goal of overall survival (OS) in patients with advanced or unresectable biliary tract cancer (BTC) in a phase 3 trial.
FDA Approves KEYTRUDA® (pembrolizumab) as Adjuvant Treatment Following Surgical Resection and Platinum-Based Chemotherapy for Patients With Stage IB (T2a ≥4 Centimeters), II, or IIIA Non-Small Cell Lung Cancer (NSCLC)
January 27, 2023 6:45 am ET
Approval based on KEYNOTE-091 trial, which demonstrated a clinically meaningful improvement in disease-free survival with KEYTRUDA in these patients following surgical resection and platinum-based chemotherapy versus placebo
Approval marks fifth indication for KEYTRUDA-based regimens in NSCLC and 34th indication for KEYTRUDA in the US
RAHWAY, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside of the United States and Canada, announced today that the U.S. Food and Drug Administration (FDA) has approved KEYTRUDA, Merck’s anti-PD-1 therapy, as a single agent, for adjuvant treatment following surgical resection and platinum-based chemotherapy for adult patients with stage IB (T2a ≥4 centimeters [cm]), II, or IIIA non-small cell lung cancer (NSCLC).
biotecMAX™ 2023 Insight
JARDIANCE® (empagliflozin tablets)
US FDA accepts supplemental New Drug Application for Jardiance® for adults with chronic kidney disease
January 20, 2023Download PDF
The supplemental New Drug Application is based on results from the landmark EMPA-KIDNEY phase III trial, which showed Jardiance® (empagliflozin) tablets significantly reduced the risk of kidney disease progression* or cardiovascular death in adults with CKD by 28% (absolute risk reduction [ARR]: 3.8%) compared with placebo, both on top of standard of care.
RIDGEFIELD, Conn. and INDIANAPOLIS, Jan. 20, 2023 /PRNewswire/ -- The U.S. Food and Drug Administration (FDA) has accepted a supplemental New Drug Application (sNDA) for Jardiance® (empagliflozin) tablets, which is being investigated as a potential treatment to reduce the risk of kidney disease progression and cardiovascular death in adults with chronic kidney disease (CKD), Boehringer Ingelheim and Eli Lilly and Company (NYSE: LLY) announced.
"There is a significant need for additional therapies that reduce the risk of kidney disease progression and hospitalizations in adults with CKD," said Mohamed Eid, M.D., M.P.H., M.H.A., vice president, Clinical Development & Medical Affairs, Cardio-Renal-Metabolism & Respiratory Medicine, Boehringer Ingelheim Pharmaceuticals, Inc. "This application acceptance is an important step forward for the approximately 37 million people in the U.S. living with CKD."
The sNDA is based on results from the landmark EMPA-KIDNEY phase III trial, in which Jardiance significantly reduced the risk of kidney disease progression* or cardiovascular death in adults with CKD by 28% (ARR: 3.8%) compared with placebo, both on top of standard of care. Results were presented during the American Society of Nephrology (ASN)'s Kidney Week 2022 and simultaneously published in The New England Journal of Medicine. EMPA-KIDNEY is the first SGLT2 inhibitor CKD trial to show a significant reduction in risk of hospitalization for any cause, with a 14% relative risk reduction with Jardiance versus placebo (24.8 vs. 29.2 events/100 patient-years, respectively), both on top of standard of care, in a pre-specified key secondary endpoint. Reductions in other key secondary endpoints of hospitalization for heart failure or cardiovascular death or all-cause death were not statistically significant. Hospitalizations account for 35% to 55% of total healthcare costs for people with CKD in the U.S.
EMPA-KIDNEY, the largest and broadest dedicated SGLT2 inhibitor trial in CKD to date, provides additional data for patients commonly seen in clinical practice. The trial enrolled 6,609 participants, including people without diabetes (56%), those with various underlying causes of CKD and those across the spectrum of eGFR and urine albumin-creatinine ratio (measures of kidney function and excess albumin in the urine, respectively). Overall, the safety data in EMPA-KIDNEY were consistent with the previously known safety profile of Jardiance.
Initially approved in 2014, Jardiance is a once-daily tablet used along with diet and exercise to lower blood sugar in adults with type 2 diabetes; and to reduce the risk of cardiovascular death in adults with type 2 diabetes and known cardiovascular disease. Jardiance is also indicated to reduce the risk of cardiovascular death and hospitalization for heart failure in adults with heart failure. Jardiance is not for patients with type 1 diabetes, or to improve glycemic control in adults with type 2 diabetes with an eGFR <30 mL/min/1.73 m2. Jardiance is contraindicated in people with hypersensitivity to empagliflozin or any of the excipients in Jardiance, and in patients on dialysis. Please see additional Important Safety Information below.
"This marks another exciting milestone for Jardiance, potentially extending its ability to positively impact the approximately one billion people diagnosed with a cardio, renal or metabolic condition," said Jeff Emmick, M.D., Ph.D., vice president, Product Development, Lilly. "We look forward to working with the FDA during the review process and eagerly await a decision later this year on the indication for CKD, which doubles a person's risk for hospitalization."
In March 2020, the FDA granted Fast Track designation to the clinical investigation of Jardiance to reduce the risk of kidney disease progression and cardiovascular death in adults with CKD. According to the FDA, Fast Track designation is designed to facilitate the development of drugs and expedite treatments that may address serious conditions and fill an unmet medical need. Jardiance is not indicated for the treatment of CKD.
*Kidney disease progression: Defined as end-stage kidney disease (the initiation of maintenance dialysis or receipt of a kidney transplant), a sustained decline in estimated glomerular filtration rate (eGFR) to below 10 mL/min/1.73 m2, kidney death or a sustained decline of at least 40% in eGFR from randomization).
About EMPA-KIDNEY: The study of heart and kidney protection with Jardiance
EMPA-KIDNEY (NCT03594110) is a multinational, randomized, double-blind, placebo-controlled clinical trial, designed to evaluate the effect of Jardiance on kidney disease progression and cardiovascular mortality risk. The primary outcome is defined as time to a first event of either cardiovascular death or kidney disease progression, defined as end-stage kidney disease (the need for kidney replacement therapy such as dialysis or kidney transplantation), a sustained decline in eGFR to <10 mL/min/1.73 m2, kidney death or a sustained decline of ≥40 percent in eGFR from randomization. Key secondary outcomes include cardiovascular death or hospitalization for heart failure, all-cause hospitalization and all-cause mortality. EMPA-KIDNEY includes 6,609 adults randomized from eight countries with established CKD both with and without diabetes, as well as with and without albuminuria, receiving either Jardiance 10 mg or placebo, on top of current standard of care.
What is JARDIANCE?
JARDIANCE is a prescription medicine used to:
- reduce the risk of cardiovascular death and hospitalization for heart failure in adults with heart failure, when the heart cannot pump enough blood to the rest of your body
- reduce the risk of cardiovascular death in adults with type 2 diabetes who also have known cardiovascular disease
- lower blood sugar along with diet and exercise in adults with type 2 diabetes
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.
https://www.jardiance.com/type-2-diabetes/
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.
About Boehringer Ingelheim
Boehringer Ingelheim is working on breakthrough therapies that improve the lives of humans and animals. As a leading research-driven biopharmaceutical company, the company creates value through innovation in areas of high unmet medical need. Founded in 1885 and family-owned ever since, Boehringer Ingelheim takes a long-term perspective. Around 52,000 employees serve more than 130 markets in the three business areas, Human Pharma, Animal Health, and Biopharmaceutical Contract Manufacturing. Learn more at www.boehringer-ingelheim.us.
About Lilly
Lilly unites caring with discovery to create medicines that make life better for people around the world. We've been pioneering life-changing discoveries for nearly 150 years, and today our medicines help more than 47 million people across the globe. Harnessing the power of biotechnology, chemistry and genetic medicine, our scientists are urgently advancing new discoveries to solve some of the world's most significant health challenges, redefining diabetes care, treating obesity and curtailing its most devastating long-term effects, advancing the fight against Alzheimer's disease, providing solutions to some of the most debilitating immune system disorders, and transforming the most difficult-to-treat cancers into manageable diseases. With each step toward a healthier world, we're motivated by one thing: making life better for millions more people. That includes delivering innovative clinical trials that reflect the diversity of our world and working to ensure our medicines are accessible and affordable. To learn more, visit Lilly.com and Lilly.com/newsroom or follow us on Facebook, Instagram and LinkedIn.
This press release contains forward-looking statements (as that term is defined in the Private Securities Litigation Reform Act of 1995) about Jardiance® as a treatment for adults with type 2 diabetes, to reduce the risk of cardiovascular death in adults with type 2 diabetes and known cardiovascular disease, and to reduce the risk of cardiovascular death and hospitalization for heart failure in adults with heart failure, and as a potential treatment for adults with cardio-kidney-metabolic conditions and reflects Lilly's current beliefs and expectations. However, as with any pharmaceutical product, there are substantial risks and uncertainties in the process of drug research, development and commercialization. Among other things, there can be no guarantee that planned or ongoing studies will be completed as planned, that future study results will be consistent with the results to date or that Jardiance® will receive additional regulatory approvals. For a further discussion of these and other risks and uncertainties that could cause actual results to differ from Lilly's expectations, please see Lilly's most recent Forms 10-K and 10-Q filed with the U.S. Securities and Exchange Commission. Lilly undertakes no duty to update forward-looking statements.
Jardiance®, EMPEROR-Reduced®, EMPEROR-Preserved®, EMPA-REG OUTCOME® and EMPACT-MI® are registered trademarks of Boehringer Ingelheim.
View original content to download multimedia:https://www.prnewswire.com/news-releases/us-fda-accepts-supplemental-new-drug-application-for-jardiance-for-adults-with-chronic-kidney-disease-301726873.html
SOURCE Eli Lilly and Company
Lilly's diabetes drug Jardiance to get FDA review for chronic kidney disease indication
Jan. 20, 2023 10:02 AM ET
By: Jonathan Block, SA News Editor
- The US FDA has accepted a supplemental New Drug Application for Eli Lilly's Jardiance (empagliflozin) as a treatment to cut the risk of kidney disease progression and cardiovascular death in those with chronic kidney disease (CKD).
- https://seekingalpha.com/news/3926360-lillys-diabetes-drug-jardiance-to-get-fda-review-for-chronic-kidney-disease-indication
- Eli Lilly and Company (LLY)
- https://www.jardiance.com/type-2-diabetes/
- https://www.lilly.com/
BRUKINSA® (zanubrutinib)
BRUKINSA® (zanubrutinib) Receives Marketing Authorization for Chronic Lymphocytic Leukemia (CLL) and Marginal Zone Lymphoma (MZL) in Great Britain by MHRA
Jan 19, 2023 7:00 AM
MHRA marketing authorizations follow recent European Commission marketing authorizations
BRUKINSA is the only treatment authorized for MZL in Great Britain
CAMBRIDGE, Mass. & BASEL, Switzerland & BEIJING--(BUSINESS WIRE)-- BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company, today announced that the Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorizations for BRUKINSA (zanubrutinib) in Great Britain for both the treatment of adult patients with chronic lymphocytic leukemia (CLL) and the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy.
“BRUKINSA is a highly selective BTK inhibitor that has demonstrated clinically meaningful improvements over the first generation of BTK inhibitor in relapsed CLL,” said Dr Renata Walewska, Department of Hematology, University Hospitals Dorset, Bournemouth, UK. “The authorization of BRUKINSA for MZL and CLL in Great Britain is a significant step forward for eligible patients and their physicians as there is no targeted treatment currently authorized for MZL patients other than chemoimmunotherapy, and it represents an alternative to current BTKi treatments for patients with CLL.”
The MHRA authorization for CLL is based on two global Phase 3 clinical trials: SEQUOIA (NCT03336333)1, comparing BRUKINSA against bendamustine plus rituximab (BR) in patients with previously untreated CLL, and ALPINE (NCT03734016)2, comparing BRUKINSA® against IMBRUVICA® (ibrutinib) in patients with relapsed/refractory (R/R) CLL.
The MHRA authorization for MZL is based on results from the multicenter, global, single-arm, open-label, Phase 2 MAGNOLIA3 trial in patients with R/R MZL who received at least one anti-CD-20 based regimen.
“As a BTK inhibitor designed to maximize BTK occupancy and minimize off-target binding, we believe BRUKINSA presents a very promising treatment option for eligible patients with MZL and CLL,” said Mehrdad Mobasher, M.D., M.P.H., Chief Medical Officer, Hematology at BeiGene.
“As we strive for delivering cancer medicines more quickly to more patients around the world, we are pleased with the progress we’ve made in bringing BRUKINSA to more eligible patients with hematological malignancies in Great Britain following these authorizations,” added Dr. Robert Mulrooney, General Manager, UK & Ireland at BeiGene.
Earlier this year, the National Institute for Health and Care Excellence (NICE), recommended BRUKINSA for the treatment of Waldenström’s macroglobulinemia (WM) in adults who have had at least one treatment, only if bendamustine plus rituximab is also suitable. BRUKINSA has also been recommended by the Scottish Medicines Consortium for the treatment of adult patients with WM who have received at least one prior therapy, or in first-line treatment for patients unsuitable for chemoimmunotherapy.
BRUKINSA is currently authorized in the European Union, and Northern Ireland, as per rulings set out in the Northern Ireland Protocol,for the treatment of adult patients with WM who have received at least one prior therapy or as the first-line treatment for patients unsuitable for chemoimmunotherapy; for the treatment of adult patients with CLL and for adult patients with MZL who have received at least one prior anti-CD20-based therapy.
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. BRUKINSA was specifically designed to deliver targeted 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 supported by a broad clinical program which includes more than 4,700 subjects in 35 trials in more than 30 geographies. To date, BRUKINSA is approved in over 60 markets, including the United States, China, the European Union, Great Britain, Canada, Australia, South Korea, Iceland, Norway and Switzerland.
About BeiGene
BeiGene is a global biotechnology company that is developing and commercializing innovative and affordable oncology medicines to improve treatment outcomes and access for far more patients worldwide. With a broad portfolio, we are expediting development of our diverse pipeline of novel therapeutics through our internal capabilities and collaborations. We are committed to radically improving access to medicines for far more patients who need them. Our growing global team of more than 9,000 colleagues spans five continents, with administrative offices in Cambridge, U.S.; Basel, Switzerland and Beijing, China. To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.
View source version on businesswire.com: https://www.businesswire.com/news/home/20230119005082/en/
Source: BeiGene
BeiGene Brukinsa gets approval in UK for blood cancer subtypes
Jan. 19, 2023 7:48 AM ET
By: Ravikash, SA News Editor
- U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA) approved BeiGene's (NASDAQ:BGNE) Brukinsa to treat adult patients with chronic lymphocytic leukemia (CLL) and adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy.
- https://seekingalpha.com/news/3925820-beigenes-brukinsa-gets-approval-in-uk-for-blood-cancer-subtypes
- BeiGene, Ltd. (BGNE)
- https://www.brukinsa.com/hcp/
Tezspire (tezepelumab)
Tezspire approved for self-administration in the EU in a new pre-filled pen
PUBLISHED13 January 2023 13 January 2023 07:00 GMT
New administration option for first and only severe asthma biologic approved in the
EU with no phenotype or biomarker limitations
AstraZeneca’s Tezspire (tezepelumab) has received a positive opinion from the European Medicine Agency’s Committee for Medicinal Products for Human Use (CHMP) for self-administration in a pre-filled, single-use pen for patients aged 12 years and older with severe asthma. The CHMP opinion can be implemented without the need for a European Commission decision due to the nature of the Type-II label variation.
The approval for self-administration was based on results from the PATHFINDER clinical trial programme, which included results from the PATH-BRIDGE Phase I trial and the PATH-HOME Phase III trial.1,2 The majority (92%) of healthcare providers, patients and caregivers were able to successfully administer Tezspire both in the clinic and at home throughout the PATH-HOME trial.2 The improvements in asthma control and the safety profile of Tezspire observed in the PATH-HOME trial were consistent with previous clinical trials.2 Tezspire is the only biologic approved for severe asthma with no phenotype (e.g. eosinophilic or allergic) or biomarker limitation within its approved label.3
Professor Ian Pavord, Professor of Respiratory Medicine at the University of Oxford and Honorary Consultant Physician at the Oxford University Hospitals, said: “Severe asthma continues to have a debilitating impact for people living with the disease. I believe the approval of the Tezspire pre-filled pen will be welcome news for physicians and patients in Europe as it offers increased choice and greater flexibility when administering this important medicine.”
Mene Pangalos, Executive Vice President, BioPharmaceuticals R&D, AstraZeneca, said: “Tezspire is the first and only biologic approved in Europe for patients with severe asthma with no phenotype or biomarker limitation. With the approval of the Tezspire pre-filled pen, we can give patients in Europe greater flexibility and support physicians in treating a broad population of severe asthma patients.”
AstraZeneca anticipates a regulatory decision by the US Food and Drug Administration (FDA) on self-administration and the new pre-filled pen in the first half of 2023. Tezspire is currently approved for the treatment of severe asthma in the US, EU, Japan and other countries.4-6
Notes
Tezspire pre-filled pen
Tezspire will be available as a fixed-dose 210mg subcutaneous injection via a pre-filled, single-use auto-injector (the Tezspire pre-filled pen), in addition to the pre-filled, single-use syringe (Tezspire pre-filled syringe). Both are administered every four weeks.
The Tezspire pre-filled pen enables patients and caregivers to self-administer the medicine at home or in clinic via a simple process. The device is fitted with a safety guard and viewing window and has audible clicks at the start and end of the injection to guide patients.
Clinical trials
PATH-HOME was a Phase III multi-centre, open-label, parallel-group trial designed to assess patient, caregiver and healthcare provider-reported functionality and performance of a single-use, pre-filled syringe (PFS) or auto-injector (AI) with a fixed 210mg dose of Tezspire administered subcutaneously every four weeks in a clinic and in an at-home setting in 216 patients aged 12 years and older with severe asthma.2
The majority (92%) of healthcare providers, patients and caregivers were able to successfully administer the Tezspire pre-filled pen both in the clinic and at home throughout the trial.2 At-home administration of the Tezspire pre-filled pen at weeks 12 and 16 was successful in 97% of the patients or caregivers (102/105).2 The trial also demonstrated for the first time that adolescents can successfully administer Tezspire using the two devices.2 The very low proportion of device malfunctions (0.9% of PFS and 0.8% of AIs) provides support that the instructions for use provided to healthcare providers, patients and caregivers is adequate for successful subcutaneous administration of Tezspire both in the clinic and at home.2
PATH-BRIDGE was a single-centre, randomised, open-label, parallel-group Phase I trial in healthy people to compare the pharmacokinetic (PK) exposure following a single 210mg dose of Tezspire by using a vial-and-syringe (V-S), PFS or pre-filled AI device.1 Tezspire PK exposure was comparable following subcutaneous administration via V-S, PFS or AI.1 In addition, injection site-pain was low in severity and injection-site reactions were uncommon in all device groups.1
In addition to PATH-BRIDGE and PATH-HOME, the PATHFINDER clinical trial programme included the pivotal NAVIGATOR Phase III trial in which Tezspire demonstrated superiority across every primary and key secondary endpoint in patients with severe asthma, compared to placebo, when added to standard therapy.7
NAVIGATOR was the first Phase III trial to show benefit in severe asthma irrespective of eosinophils by targeting thymic stromal lymphopoietin (TSLP).7 These results support the FDA Breakthrough Therapy Designation granted to Tezspire in September 2018 for patients with severe asthma, without an eosinophilic phenotype. In July 2021, Tezspire was the first and only biologic to be granted Priority Review in the US for the treatment of asthma by the FDA.
Tezspire
Tezspire (tezepelumab) is being developed by AstraZeneca in collaboration with Amgen as a first-in-class human monoclonal antibody that inhibits the action of TSLP, a key epithelial cytokine that sits at the top of multiple inflammatory cascades and is critical in the initiation and persistence of allergic, eosinophilic and other types of airway inflammation associated with severe asthma, including airway hyperresponsiveness.8,9 TSLP is released in response to multiple triggers associated with asthma exacerbations, including allergens, viruses and other airborne particles.8,9 Expression of TSLP is increased in the airways of patients with asthma and has been correlated with disease severity.8,9 Blocking TSLP may prevent the release of pro-inflammatory cytokines by immune cells, resulting in the prevention of asthma exacerbations and improved asthma control.7,8,10 Tezspire acts at the top of the inflammation cascade and has the potential to help address a broad population of severe asthma patients irrespective of biomarker levels.7,8
Tezspire is approved in the US, EU, Japan and other countries for the treatment of severe asthma.4-6 Tezspire is also in development for other potential indications including chronic obstructive pulmonary disease (COPD), chronic rhinosinusitis with nasal polyps, chronic spontaneous urticaria and eosinophilic esophagitis (EoE). In October 2021, tezepelumab was granted Orphan Drug Designation by the FDA for the treatment of EoE.
Amgen collaboration
In 2020, Amgen and AstraZeneca updated a 2012 collaboration agreement for Tezspire. Both companies will continue to share costs and profits equally after payment by AstraZeneca of a mid single-digit inventor 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, Amgen and AstraZeneca will jointly commercialise Tezspire in North America. Amgen will record product sales in the US, with AZ recording its share of US profits as Collaboration Revenue. Outside of the US, AstraZeneca will record product sales, with Amgen recording profit share as Other/Collaboration revenue.
AstraZeneca in Respiratory & Immunology
Respiratory & Immunology, part of BioPharmaceuticals, is one of AstraZeneca’s main disease areas and is a key growth driver for the Company.
AstraZeneca is an established leader in respiratory care with a 50-year heritage. The Company aims to transform the treatment of asthma and COPD by focusing on earlier biology-led treatment, eliminating preventable asthma attacks, and removing COPD as a top-three leading cause of death. The Company’s early respiratory research is focused on emerging science involving immune mechanisms, lung damage and abnormal cell-repair processes in disease and neuronal dysfunction.
With common pathways and underlying disease drivers across respiratory and immunology, AstraZeneca is following the science from chronic lung diseases to immunology-driven disease areas. The Company’s growing presence in immunology is focused on five mid- to late-stage franchises with multi-disease potential, in areas including rheumatology (including systemic lupus erythematosus), dermatology, gastroenterology, and systemic eosinophilic-driven diseases. AstraZeneca’s ambition in Respiratory & Immunology is to achieve disease modification and durable remission for millions of patients worldwide.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
Amgen, AstraZeneca's asthma therapy Tezspire's self-administration option gets approval in EU
Jan. 13, 2023 4:33 AM ET
By: Ravikash, SA News Editor
A panel of the European Medicine Agency (EMA) issued a positive opinion on a self-administration option of Amgen (NASDAQ:AMGN) and AstraZeneca's (NASDAQ:AZN) asthma therapy Tezspire.
TUKYSA® (tucatinib) in Combination with Trastuzumab
Seagen Announces FDA Accelerated Approval of TUKYSA® (tucatinib) in Combination with Trastuzumab for People with Previously Treated RAS Wild-Type, HER2-Positive Metastatic Colorectal Cancer
01/19/2023
– First FDA-approved treatment in HER2-positive metastatic colorectal cancer –
– Combination regimen is approved for use in the second-line treatment setting and beyond –
BOTHELL, Wash.--(BUSINESS WIRE)-- Seagen Inc. (Nasdaq: SGEN) today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval to TUKYSA® (tucatinib) in combination with trastuzumab for adult patients with RASwild-type, HER2-positive unresectable or metastatic colorectal cancer that has progressed following treatment with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy. TUKYSA is approved under the FDA’s Accelerated Approval Program based on tumor response rate and durability of response from the phase 2 MOUNTAINEER clinical trial. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials. This is the first FDA-approved treatment in HER2-positive metastatic colorectal cancer. The FDA previously granted Breakthrough Therapy Designation and Priority Review for TUKYSA in this setting.
“Historically, patients with HER2-positive metastatic colorectal cancer who have progressed following frontline therapy have had poor outcomes,” said John Strickler, M.D., associate professor of medicine, Duke University Medical Center, and lead investigator for the MOUNTAINEER trial. “The FDA approval of a chemotherapy-free combination regimen that specifically targets HER2 is great news for these patients.”
“Biomarker testing is bringing new hope to people living with some types of colorectal cancer by opening the door to targeted treatments like TUKYSA for those who have RAS wild-type, HER2-positive disease,” said Michael Sapienza, CEO, Colorectal Cancer Alliance. “It is critical that physicians and patients understand the importance of comprehensive biomarker testing at diagnosis because it can inform treatment decisions and help improve outcomes.”
Results from the MOUNTAINEER trial showed a 38% overall response rate (ORR) (95% Confidence Interval [CI]: 28, 49) per blinded independent central review (BICR) in the patients who received TUKYSA in combination with trastuzumab (N=84 with a median age of 55.0 years [range: 24 to 77]). Complete responses were observed in 3.6% of patients (n=3), and partial responses were observed in 35% of patients (n=29). The median duration of response (DOR) per BICR was 12.4 months (95% CI: 8.5, 20.5). At study entry, 64% and 70% of these patients had liver or lung metastases, respectively.
The Prescribing Information for TUKYSA includes warnings and precautions for diarrhea, hepatotoxicity and embryo-fetal toxicity, some of which may be severe or fatal. In MOUNTAINEER, serious adverse reactions occurred in 22% of patients; the most common (in ≥2% of patients) were intestinal obstruction (7%), urinary tract infection (3.5%), pneumonia, abdominal pain and rectal perforation (2.3% each). The most common adverse reactions (≥20%) in patients treated with TUKYSA and trastuzumab were diarrhea, fatigue, rash, nausea, abdominal pain, infusion-related reactions and pyrexia. Adverse reactions leading to permanent discontinuation of TUKYSA occurred in 6% of patients; the most common (in ≥2% of patients) was increased alanine aminotransferase (ALT) (2.3%). Please see additional Important Safety Information below.
“The accelerated approval of TUKYSA for RAS wild-type, HER2-positive metastatic colorectal cancer expands TUKYSA-based therapy to patients across two distinct types of cancer,” said Marjorie Green, M.D., Senior Vice President and Head of Late-Stage Development, Seagen. “We believe the efficacy and safety profile of the TUKYSA and trastuzumab-based regimen further establishes its role as an important backbone of dual HER2 inhibition in the treatment of adult patients with certain HER2-expressing breast and colorectal cancers.”
The FDA’s Accelerated Approval Program allows for approval of a medicine based on a surrogate endpoint that is reasonably likely to predict clinical benefit if the medicine fills an unmet medical need for a serious condition. A global, randomized phase 3 clinical trial (MOUNTAINEER-03) is ongoing and will compare TUKYSA in combination with trastuzumab and mFOLFOX6 with standard of care, which is intended to serve as a confirmatory trial and potentially support future global regulatory submissions. Merck, known as MSD outside of the U.S. and Canada, is commercializing TUKYSA in regions outside of the U.S., Canada and Europe and plans to discuss results from the MOUNTAINEER trial with certain health authorities as it continues to accelerate the filing of TUKYSA in its territories.
About Colorectal Cancer
In the U.S., approximately 153,000 people will be diagnosed with colorectal cancer in 2023, and the rate of the disease is increasing in younger adults.1 Approximately 22% of U.S. patients with colorectal cancer are diagnosed at the advanced stage.2 Human epidermal growth factor receptor 2 (HER2) is overexpressed in 3-5% of patients with metastatic colorectal cancer and approximately 10% of patients with RASwild-type metastatic colorectal cancer.3 In 2023, colorectal cancer is anticipated to lead to about 52,550 deaths in the U.S., where it is the third-leading cause of cancer-related deaths.1
About MOUNTAINEER
MOUNTAINEER is a U.S. and European open-label, multicenter phase 2 clinical trial of TUKYSA in combination with trastuzumab that evaluated 84 patients with HER2-positive, RASwild-type, unresectable or metastatic colorectal cancer following previous standard-of-care therapies. Patients evaluated in MOUNTAINEER had not received prior anti-HER2 therapy. Patients received TUKYSA (300 mg) twice per day orally in combination with trastuzumab intravenously (8 mg/kg loading dose, then 6 mg/kg every three weeks thereafter) until disease progression or unacceptable toxicity. The major efficacy outcome measures were overall response rate (ORR) and duration of response (DOR) as assessed by blinded independent central review (BICR) according to RECIST (Response Evaluation Criteria in Solid Tumors) version 1.1.
About TUKYSA (tucatinib)
TUKYSA (tucatinib) tablets (50 | 150 mg tablets) is an oral medicine that is a tyrosine kinase inhibitor of the HER2 protein. In vitro (in lab studies), TUKYSA inhibited phosphorylation of HER2 and HER3, resulting in inhibition of downstream MAPK and AKT signaling and cell growth (proliferation), and showed anti-tumor activity in HER2-expressing tumor cells. In vivo (in living organisms), TUKYSA inhibited the growth of HER2-expressing tumors. The combination of TUKYSA and the anti-HER2 antibody trastuzumab showed increased anti-tumor activity in vitro and in vivo compared to either medicine alone.
TUKYSA was approved by the U.S. FDA in April 2020 in combination with trastuzumab and capecitabine for treatment of adult patients with advanced unresectable or metastatic HER2-positive breastcancer, including patients with brain metastases, who have received one or more prior anti-HER2-based regimens in the metastatic setting. In January 2023, TUKYSA received accelerated approval by the U.S. FDA in combination with trastuzumab for adult patients with RASwild-type, HER2-positive unresectable or metastatic colorectal cancer that has progressed following treatment with fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapy. Merck, known as MSD outside the U.S. and Canada, has exclusive rights to commercialize TUKYSA in Asia, the Middle East and Latin America and other regions outside of the U.S., Canada and Europe.
About Seagen
Seagen is a global biotechnology company that discovers, develops and commercializes transformative cancer medicines to make a meaningful difference in people’s lives. Seagen is headquartered in the Seattle, Washington area, and has locations in California, Canada, Switzerland and the European Union. For more information on the company’s marketed products and robust pipeline, visit www.seagen.com and follow @SeagenGlobal on Twitter.
Seagen wins FDA accelerated approval for colorectal cancer therapy
Jan. 19, 2023 2:37 PM ET
Seagen Inc. (SGEN), MRKRHHBY, RHHBF
By: Dulan Lokuwithana, SA News Editor1 Comment
- Seagen (NASDAQ:SGEN) announced Thursday that the FDA granted accelerated approval for its tyrosine kinase inhibitor Tukysa, in combination with Roche’s (OTCQX:RHHBY) (OTCQX:RHHBF) trastuzumab for the treatment of certain adults with colorectal cancer as a second-line option.
- https://seekingalpha.com/news/3926075-sgen-stock-gains-fda-clears-colorectal-cancer-therapy
- Seagen Inc. (SGEN), MRK
- https://www.herceptin.com/hcp.html
- https://www.tukysa.com/
- https://www.seagen.com/
Xofluza® (baloxavir marboxil)
Roche announces the European Commission approval of Xofluza for the treatment and prevention of influenza in children aged one year and above
January 12, 2023
- Xofluza is now approved in the EU for the treatment of uncomplicated influenza and for post-exposure prophylaxis of influenza in children aged one year and above, and in adolescents and adults
- Xofluza is the first influenza antiviral with a new mechanism of action in almost 20 years, stopping viral replication faster than oseltamivir
- Single-dose Xofluza helps reduce the societal burden of influenza by helping patients recover quickly and by preventing infection in individuals following contact with someone with the virus
Basel, 12 January 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the European Commission (EC) has approved Xofluza® (baloxavir marboxil) in children aged one year and above for the treatment of uncomplicated influenza and for post-exposure prophylaxis of influenza. Post-exposure prophylaxis aims to prevent influenza in individuals following contact with someone infected with the influenza virus. The Commission’s Decision is based on the results of the phase III miniSTONE-2 and BLOCKSTONE studies.1,2 This approval marks the first single-dose, oral influenza medicine approved in Europe for children, and now means that Xofluza can be used to treat and prevent uncomplicated influenza in children aged one year and above, and in adolescents and adults.3
“We are delighted that the European Commission has approved Xofluza for children aged one year and above, as influenza can be particularly dangerous for younger children,” said Levi Garraway, M.D., Ph.D., Roche’s Chief Medical Officer and Head of Global Product Development. “We are hopeful that Xofluza’s convenient single-dose oral regimen will help children recover quickly, as well as reduce the societal burden of influenza.”
Influenza is a serious infectious disease and represents a significant threat to public health.4-6 Rates of influenza in Europe are rising and it is expected that the virus will infect around one in four children each year.6,7 In addition to being at higher risk of infection, children also play a significant role in the spread of influenza from one person to another.6,8 In studies of influenza transmission in households, the risk of infection from others in the household was as high as 38%.9 Prophylactic treatment with Xofluza, which stops viral replication faster than oseltamivir, may help to limit the spread of influenza and therefore reduce the impact and burden of influenza on society.2,10,11
The approval is based on the results of the phase III miniSTONE-2 and BLOCKSTONE studies.1,2 The miniSTONE-2 study met its primary endpoint of safety and showed that Xofluza reduced the length of time that influenza was released from the body by more than two days compared with oseltamivir (viral replication; median time of 24.2 hours versus 75.8 hours, respectively). Xofluza was well tolerated with no new safety signals identified.1
In the BLOCKSTONE study Xofluza showed a statistically significant prophylactic effect after a single oral dose, by reducing the risk of people developing influenza after exposure to an infected household member by 86% versus placebo (1.9% in participants treated with Xofluza and 13.6% in the placebo group).2
Xofluza was first approved for use in Europe in 2021 for the treatment of uncomplicated influenza and for post-exposure prophylaxis of influenza in adults and adolescents aged 12 years and above.12 Xofluza is now approved in more than 70 countries for the treatment of influenza types A and B.13 Roche is continuing to investigate Xofluza in phase III trials in children under the age of one (NCT03653364), as well as to assess its potential to reduce transmission of influenza from an infected person to household members (NCT03969212).14,15
About miniSTONE-21
miniSTONE-2 was a phase III, multicentre, randomised, double-blind study that evaluated the safety, pharmacokinetics and efficacy of a single-dose of Xofluza® (baloxavir marboxil) compared with oseltamivir in otherwise healthy children aged one to less than 12 years with influenza infection and displaying influenza symptoms for no more than 48 hours (temperature of 38°C or over, and one or more respiratory symptoms).
Participants enrolled in the study were recruited in parallel into two cohorts: children aged five to less than 12 years and children aged one to less than five years. Participants were randomly assigned to receive a single-dose of Xofluza or oseltamivir twice a day over five days (dosing according to body weight).
Time to alleviation of influenza signs and symptoms were comparable between Xofluza and oseltamivir. The median time to alleviation of signs and symptoms in influenza-infected participants was 138 hours (95% CI: 117, 163) and 150 hours (95% CI: 115, 166) for those who received Xofluza or oseltamivir, respectively. Xofluza reduced the length of time that influenza was released from the body by more than two days compared with oseltamivir (viral replication; median time of 24.2 hours versus 75.8 hours, respectively). Xofluza was well tolerated with no new safety signals identified.
About BLOCKSTONE2
BLOCKSTONE was a phase III, double-blind, multicentre, randomised, placebo-controlled, post-exposure prophylaxis study that evaluated single-dose Xofluza® (baloxavir marboxil) compared with placebo in household members (adults and children), who were living with someone with influenza, confirmed by a rapid influenza diagnostic test (the ‘index patient’).
Participants enrolled in the study were household members of someone who had been diagnosed with influenza. The participants were randomised to receive a single-dose of Xofluza (dose according to body weight) or placebo as a preventive measure against developing influenza.
Xofluza showed a statistically significant prophylactic effect after a single oral dose, by reducing the risk of people developing influenza after exposure to an infected household member by 86% versus placebo. The proportion of households, who developed laboratory-confirmed clinical influenza was 1.9% in participants treated with Xofluza and 13.6% in the placebo-treated group.
Xofluza was well tolerated in this study and no new safety signals were identified. The study was conducted in Japan by Shionogi & Co., Ltd.
About Xofluza® (baloxavir marboxil)
Xofluza is a first-in-class, single-dose oral medicine with an innovative mechanism of action that has demonstrated efficacy in a wide range of influenza viruses, including in vitro activity against oseltamivir-resistant strains and avian strains (H7N9, H5N1) in non-clinical studies.10,16,17 Xofluza is the first in a class of antivirals designed to inhibit the cap-dependent endonuclease protein, which is essential for viral replication.10
Xofluza is approved in more than 70 countries for the treatment of influenza types A and B. In Europe, Xofluza is now approved for the treatment of uncomplicated influenza and for post-exposure prophylaxis of influenza in children aged one year and above, and in adolescents and adults. Xofluza is the first innovation in mechanism of action for an influenza antiviral approved by the European Commission in almost 20 years.18
Robust clinical evidence has demonstrated the benefit of Xofluza in several populations (otherwise-healthy, high-risk, children and post-exposure prophylaxis in individuals aged one year and above).1,2,10,19 Xofluza is being further studied in a phase III development programme, including in children under the age of one (NCT03653364) as well as to assess its potential to reduce transmission of influenza from an infected person to healthy people (NCT03969212).14,15
Xofluza was discovered by Shionogi & Co., Ltd. and is being further developed and commercialised globally in collaboration with the Roche Group (which includes Genentech in the US) and Shionogi & Co., Ltd. Under the terms of this agreement, Roche holds worldwide rights to Xofluza excluding Japan and Taiwan, which will be retained exclusively by Shionogi & Co., Ltd.
About Roche in influenza
Influenza is a serious infectious disease and represents a significant threat to public health. Seasonal epidemics result in three to five million cases of severe disease, millions of hospitalisations and up to 650,000 deaths globally every year.4 Roche has a long heritage in developing medicines that contribute to public health. We are committed to bringing innovation in the field of infectious diseases, including influenza. Tamiflu® (oseltamivir) has made a significant difference both to the treatment of seasonal influenza as well as in the management of recent pandemics, and we are proud to have brought this innovative medicine to patients. Although vaccines are an important first line of defence in preventing influenza, there is a need for new medical options for prevention (prophylaxis) and treatment. Roche is committed to addressing the unmet need in this area through its agreement with Shionogi & Co., Ltd. to develop and commercialise Xofluza® (baloxavir marboxil).
About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.
In recognising our endeavor to pursue a long-term perspective in all we do, Roche has been named one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices for the thirteenth consecutive year. This distinction also reflects our efforts to improve access to healthcare together with local partners in every country we work.
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.
Roche influenza therapy Xofluza gets approval in EU for use in children
Jan. 12, 2023 4:27 AM ET
Roche Holding AG (RHHBY), RHHBF
By: Ravikash, SA News Editor
- The European Commission (EC) approved Roche's (OTCQX:RHHBY) (OTCQX:RHHBF) influenza therapy Xofluza to include children aged one year and above.
- https://seekingalpha.com/news/3924038-roche-influenza-therapy-xofluza-gets-approval-in-eu-for-use-in-children
- Roche Holding AG (RHHBY), RHHBF
- https://www.xofluza.com/
Nirsevimab US regulatory submission accepted for the prevention of RSV lower respiratory tract disease in infants and children up to age 24 months
PUBLISHED5 January 2023 5 January 2023 07:00 GMT
If FDA approved, nirsevimab would be the first single-dose RSV preventative option for the broad newborn and infant population in the US
Submission supported by comprehensive clinical trial programme, which demonstrated protection against RSV disease through the RSV season
AstraZeneca’s Biologics License Application (BLA) for nirsevimab has been accepted for review by the US Food and Drug Administration (FDA) for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease in newborns and infants entering or during their first RSV season, and for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season.
Nirsevimab is being developed and commercialised by AstraZeneca in collaboration with Sanofi and is the first single-dose preventative option for the broad infant population, including those born healthy, at term or preterm, or with specific health conditions. The FDA has indicated it will work to expedite its review. The Prescription Drug User Fee Act date, the FDA target action date for its decision, is in the third quarter of 2023. If approved at that time, nirsevimab will be available in the US for the 2023/2024 RSV season.
RSV is a very contagious virus that can lead to serious respiratory illness, according to the Centers for Disease Control and Prevention.1 In the US, RSV is the leading cause of hospitalisation for babies under one.2 Any infant can be hospitalised in their first RSV season and about 75% of infants hospitalised for RSV in the US were born at term with no underlying conditions.3-5
Dr William Muller, Associate Professor, Pediatrics, Northwestern University Feinberg School of Medicine and Scientific Director, Clinical and Community Trials, Ann & Robert H. Lurie Children’s Hospital of Chicago, Illinois, US, said: “A substantial burden of disease from RSV affects infants, families, and healthcare providers every year. Effective interventions to prevent RSV are a critical need. This year in the US, we’ve seen first-hand how frightening the impact of this respiratory disease is on our patients and how stressful it is on the healthcare system, highlighting the urgency of addressing this problem.”
Iskra Reic, Executive Vice President, Vaccines and Immune Therapies, AstraZeneca, said: “This decision brings us a step closer to delivering a first-in-class preventative option for a broad infant population in the US. If approved, we believe nirsevimab may transform the medical community’s approach to respiratory syncytial virus prevention in infants and we are committed to working with the FDA to support completion of the review as quickly as possible.”
The BLA was based on results from the comprehensive nirsevimab clinical development programme, including the MELODY Phase III (primary cohort and all subjects), MEDLEY Phase II/III (first and second RSV season), and Phase IIb trials.6-11 Data from the MELODY trial was published in the New England Journal of Medicine in March 2022 and demonstrated a reduction in the incidence of medically-attended lower respiratory tract infections (LRTI) caused by RSV by 74.5% (95% CI 49.6, 87.1; p<0.001) vs. placebo through day 151 (a typical RSV season) with a single dose.6 Nirsevimab also demonstrated a comparable safety and tolerability profile to Synagis (palivizumab) in the MEDLEY Phase II/III trial, with occurrence of treatment emergent adverse events (TEAEs) or treatment emergent serious adverse events (TESAEs) similar between groups.10-11
In November 2022, nirsevimab was granted marketing authorisation in the European Union for the prevention of RSV lower respiratory tract disease in newborns and infants during their first RSV season, under the name Beyfortus.12 Additional global regulatory submissions are underway.
Notes
Nirsevimab
Nirsevimab is a single dose long-acting antibody, developed and commercialised in partnership by AstraZeneca and Sanofi using AstraZeneca’s YTE technology. It is designed to protect infants entering or during their first RSV season and for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season.
Nirsevimab has been developed to offer newborns and infants direct RSV protection via an antibody to help prevent LRTI caused by RSV. Monoclonal antibodies do not require the activation of the immune system to help offer timely, rapid and direct protection against disease.14
Nirsevimab has been granted regulatory and other designations to facilitate expedited development by several major regulatory agencies around the world. These include Breakthrough Therapy Designation by the China Center for Drug Evaluation under the National Medical Products Administration; Breakthrough Therapy Designation from the US Food and Drug Administration; access granted to the European Medicines Agency (EMA PRIority Medicines (PRIME) scheme; and named “a medicine for prioritized development” under the Project for Drug Selection to Promote New Drug Development in Pediatrics by the Japan Agency for Medical Research and Development (AMED). In November 2022, Beyfortus was approved by the European Commission and by the UK Medicines and Healthcare products Regulatory Agency (MHRA).12-13
Pivotal clinical trials
The Phase IIb study was a randomised, placebo-controlled trial designed to measure the efficacy of nirsevimab against medically attended LRTI through 150 days postdose. Healthy preterm infants of 29–35 weeks’ gestation were randomised (2:1) to receive a single 50mg intramuscular injection of nirsevimab or placebo.8-9
The dosing regimen was recommended based on further exploration of the Phase IIb data.8 The subsequent Phase III study, MELODY applied the recommended dosing regimen.6-7
The MELODY Phase III study was a randomised, placebo-controlled trial conducted across 21 countries designed to determine efficacy of nirsevimab against medically attended LRTI due to RSV confirmed by reverse transcriptase polymerase chain reaction testing through 150 days after dosing, versus placebo, in healthy late preterm and term infants (35 weeks gestational age or greater) entering their first RSV season.6-7
MEDLEY was a Phase II/III, randomised, double-blind, Synagis-controlled trial with the primary objective of assessing safety and tolerability for nirsevimab in preterm infants and infants with congenital heart disease (CHD) and/or chronic lung disease of prematurity (CLD) eligible to receive Synagis.10-11 Between July 2019 and May 2021 approximately 918 infants entering their first RSV season were randomised to receive a single 50mg (in infants weighing <5kg) or 100mg (in infants weighing ≥5kg) intramuscular injection of nirsevimab or Synagis. Safety was assessed by monitoring the occurrence of TEAEs and TESAEs through 360 days post-dose.10-11 Serum levels of nirsevimab following dosing (on day 151) in this trial were comparable with those observed in the MELODY Phase III trial, indicating similar protection in this population to that in the healthy term and late preterm infants is likely.11 Data was published in the New England Journal of Medicine (NEJM) in March 2022.
The safety profile of nirsevimab was similar to Synagis in the MEDLEY Phase II/III and consistent with the safety profile in term and preterm infants studied in the MELODY Phase III trial.6-7,10-11 While uncommon, the most reported adverse reactions were: rash 14 days post-dose, (the majority of which were mild to moderate); pyrexia (fever) within 7 days post-dose; non-serious injection site reactions within 7 days post-dose.
The results of MELODY, MEDLEY Phase II/III and the Phase IIb trials demonstrate that nirsevimab helps protect infants during their first RSV season against RSV disease with a single dose.6-11,15-16 This broad infant population includes preterm, healthy late preterm and term infants, as well as infants with specific conditions.
These trials formed the basis of regulatory submissions which began in 2022.
Results from the first year MELODY Phase III trial
The primary endpoint of the MELODY Phase III trial was met, reducing the incidence of medically attended LRTI, such as bronchiolitis or pneumonia, caused by RSV by 74.5% (95% CI 49.6, 87.1; P<0.001) compared to placebo. Infants were randomised (2:1) to receive a single 50mg (in infants weighing <5kg) or 100mg (in infants weighing ≥5kg) intramuscular injection of nirsevimab or placebo. Between July 2019 and March 2020, 1,490 infants were randomised to receive either nirsevimab or placebo at the RSV season start.6-7 Data was published in NEJM in March 2022.
Sanofi Alliance
In March 2017, AstraZeneca and Sanofi announced an agreement to develop and commercialise nirsevimab. Under the terms of the agreement, AstraZeneca leads all development and manufacturing activities, and Sanofi leads commercialisation activities and records revenue. Under the terms of the global agreement, Sanofi made an upfront payment of €120m, has paid a development milestone of €30m and will pay up to a further €465m upon achievement of certain development and sales-related milestones. The two companies share costs and profits. Revenue from the agreement is reported as Collaboration Revenue in the Company’s financial statements.
Sobi agreement
Related, in November 2018, AstraZeneca agreed to sell US commercial rights for Synagis (palivizumab) to Swedish Orphan Biovitrum AB (publ) (Sobi) in addition to the right to participate in payments that may be received by AstraZeneca from the US profits or losses for nirsevimab. Under the agreement AstraZeneca received upfront consideration and also received non-contingent payments for nirsevimab during 2019-2021. AstraZeneca is also entitled to receive certain milestone payments for nirsevimab, including a $175m cash payment following the date on which the Biologics License Application (BLA) for nirsevimab was accepted for filing by the FDA and a $90m cash payment following the date on which BLA approval in the US occurs. AstraZeneca will continue to manufacture and supply nirsevimab globally and is entitled to an additional royalty from Sobi if profits from nirsevimab in the US exceed a pre-specified level.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca, Sanofi BLA for nirsevimab for respiratory syncytial virus accepted by FDA
Jan. 05, 2023 2:40 PM ET
By: Jonathan Block, SA News Editor
- The US FDA has accepted a Biologics License Application from AstraZeneca (NASDAQ:AZN) and Sanofi (NASDAQ:SNY) for nirsevimab for the treatment of respiratory syncytial virus (RSV) in newborns, infants, and children up to 24 months old.
- https://seekingalpha.com/news/3922494-astrazeneca-sanofi-bla-for-nirsevimab-for-respiratory-syncytial-virus-accepted-by-fda
- Sanofi (SNY), AZN
TECENTRIQ® (atezolizumab) injection plus AVASTINâ (bevacizumab) injection
[Ad hoc announcement pursuant to Art. 53 LR] Roche’s Tecentriq plus Avastin is the first treatment combination to reduce the risk of cancer returning in people with certain types of early-stage liver cancer in a Phase III trial January 19, 2023
- Pivotal Phase III IMbrave050 study investigating Tecentriq plus Avastin in people with early-stage hepatocellular carcinoma (HCC) at high risk of recurrence following surgery met primary endpoint of recurrence-free survival
- New adjuvant treatments are urgently needed as an estimated 70-80% of people with early-stage HCC experience disease recurrence following surgery1
- Data will be discussed with health authorities globally, including the US Food and Drug Administration and the European Medicines Agency to inform the next regulatory steps, and presented at an upcoming medical meeting
Basel, 19 January 2023 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the Phase III IMbrave050 study met its primary endpoint of recurrence-free survival (RFS) at the prespecified interim analysis. The study is evaluating Tecentriq® (atezolizumab) in combination with Avastin® (bevacizumab) as adjuvant treatment following surgery for people with early-stage hepatocellular carcinoma (HCC) at high risk of disease recurrence. The Tecentriq combination showed a statistically significant improvement in RFS in the intention-to-treat population of HCC patients who have an increased risk of recurrence following resection or ablation with curative intent, compared with active surveillance.
Overall survival data were immature at the time of interim analysis and follow-up will continue to the next analysis. Safety for Tecentriq and Avastin was consistent with the known safety profile of each therapeutic agent and with the underlying disease. Results from the IMbrave050 study will be discussed with health authorities, including the US Food and Drug Administration and the European Medicines Agency, and presented at an upcoming medical meeting.
“Today, more than 70% of people with early-stage HCC may have their cancer return after surgery, which is associated with poorer prognosis and shorter survival. IMbrave050 is the first Phase III study to show that a cancer immunotherapy combination reduced the risk of disease returning in people with this type of HCC,” said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development. “We are excited by the clinical benefit that this adjuvant Tecentriq combination may bring to people with early liver cancer and look forward to seeing more mature data to further confirm the benefit.”
With liver cancer incidence increasing and mortality rates rising globally, effective treatments are urgently required.1-4 Roche is working in partnership with the community and leveraging its diagnostic and pharmaceutical expertise to develop solutions for patients that address unmet needs at each stage of liver cancer. IMbrave050 is part of Roche’s overall commitment to drive fundamental treatment change and improve outcomes for people living with liver cancer. In unresectable HCC (uHCC), for example, Tecentriq plus Avastin was the first treatment in over a decade to significantly improve overall survival over the existing standard of care, based on data from the IMbrave150 study. The Tecentriq combination quickly became a standard of care in uHCC and is clearly defined as a preferred front-line treatment in multiple international clinical guidelines.
Roche has an extensive development programme for Tecentriq, including multiple ongoing and planned Phase III studies across different lung, genitourinary, skin, breast, gastrointestinal, gynaecological, and head and neck cancers. This includes studies evaluating Tecentriq both alone and in combination with other medicines, as well as studies in metastatic, adjuvant and neoadjuvant settings across various tumour types.
About the IMbrave050 study
IMbrave050 is a Phase III global, multicentre, open-label, randomised study evaluating the efficacy and safety of adjuvant Tecentriq plus Avastin, compared with active surveillance, in people with HCC at high risk of recurrence (determined by the size and number of cancerous lesions and the histopathology results, if available) after surgical resection or ablation with curative intent.
The study randomised 662 people with a ratio of 1:1 to receive either Tecentriq (1,200 mg every three weeks) plus Avastin (15 mg/kg every three weeks) for a maximum of 12 months, or no intervention with active surveillance. The primary endpoint is independent review facility-assessed RFS. Key secondary endpoints include overall survival, RFS as determined by the investigator and RFS in patients with PD-L1-positive disease.
About Tecentriq
Tecentriq is a cancer immunotherapy approved for some of the most aggressive and difficult-to-treat forms of cancer. Tecentriq was the first cancer immunotherapy approved for the treatment of a certain type of early-stage non-small cell lung cancer (NSCLC), small cell lung cancer (SCLC) and HCC. Tecentriq is also approved in countries around the world, either alone or in combination with targeted therapies and/or chemotherapies, for various forms of metastatic NSCLC, certain types of metastatic urothelial cancer, PD-L1-positive metastatic triple-negative breast cancer and BRAF V600 mutation-positive advanced melanoma.
Tecentriq is a monoclonal antibody designed to bind with a protein called programmed death ligand-1 (PD-L1), which is expressed on tumour cells and tumour-infiltrating immune cells, blocking its interactions with both PD-1 and B7.1 receptors. By inhibiting PD-L1, Tecentriq may enable the activation of T-cells. Tecentriq is a cancer immunotherapy that has the potential to be used as a foundational combination partner with other immunotherapies, targeted medicines and various chemotherapies across a broad range of cancers. In addition to intravenous infusion, the formulation of Tecentriq is also being investigated as a subcutaneous injection to help address the growing burden of cancer treatment for patients and healthcare systems.
https://www.avastin.com/hcp.html
https://www.avastin.com/patient/mcrc.html
About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.
In recognising our endeavor to pursue a long-term perspective in all we do, Roche has been named one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices for the thirteenth consecutive year. This distinction also reflects our efforts to improve access to healthcare together with local partners in every country we work.
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.
Roche's Tecentriq, Avastin combo cuts risk of liver cancer returning, in phase 3 trial
Jan. 19, 2023 4:55 AM ET
Roche Holding AG (RHHBY), RHHBF
By: Ravikash, SA News Editor
Roche (OTCQX:RHHBY) (OTCQX:RHHBF) said Tecentriq in combination with Avastin met the main goal of recurrence-free survival (RFS) in a phase 3 trial in patients with a type of liver cancer.
Roche Holding AG (RHHBY), RHHBF
LEQEMBI™ (lecanemab-irmb)
FDA Approves LEQEMBI™ (lecanemab-irmb) Under the Accelerated Approval Pathway for the Treatment of Alzheimer's Disease
JANUARY 6, 2023 • NEWS RELEASE
Accelerated Approval is based on Phase 2 data showing a reduction in amyloid-beta plaques in early AD patients treated with LEQEMBI™
Treatment with LEQEMBI should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials
TOKYO and CAMBRIDGE, Mass., Jan. 6, 2023 /PRNewswire/ -- Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") and Biogen Inc. (Nasdaq: BIIB, Corporate headquarters: Cambridge, Massachusetts, CEO: Christopher A. Viehbacher, "Biogen") announced today that under the Accelerated Approval Pathway the U.S. Food and Drug Administration (FDA) has approved lecanemab-irmb (Brand Name in the U.S.: LEQEMBI™) 100 mg/mL injection for intravenous use, a humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble ("protofibril")* and insoluble forms of amyloid beta (Aβ) for the treatment of Alzheimer's disease (AD). The approval is based on Phase 2 data that demonstrated that LEQEMBI reduced the accumulation of Aβ plaque in the brain, a defining feature of AD. Using the recently published data from the large global confirmatory Phase 3 clinical trial, Clarity AD, Eisai will work quickly to file a Supplemental Biologics License Application (sBLA) to the FDA for approval under the traditional pathway.
INDICATION
LEQEMBI is indicated for the treatment of Alzheimer's disease. Treatment with LEQEMBI should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials. There are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied. This indication is approved under accelerated approval based on reduction in amyloid beta plaques observed in patients treated with LEQEMBI. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial.
INDICATION
LEQEMBI is indicated for the treatment of Alzheimer's disease. Treatment with LEQEMBI should be initiated in patients with mild cognitive impairment or mild dementia stage of disease, the population in which treatment was initiated in clinical trials. There are no safety or effectiveness data on initiating treatment at earlier or later stages of the disease than were studied. This indication is approved under accelerated approval based on reduction in amyloid beta plaques observed in patients treated with LEQEMBI. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial.
Please see full Prescribing Information.
1. About LEQEMBITM (lecanemab-irmb)
LEQEMBITM (lecanemab-irmb) is a humanized immunoglobulin gamma 1 (IgG1) monoclonal antibody directed against aggregated soluble (protofibril) and insoluble forms of amyloid-beta (Aβ). LEQEMBI is indicated for the treatment of Alzheimer's disease (AD) in the U.S. This indication is approved under accelerated approval based on reduction in Aβ plaques observed in patients treated with LEQEMBI. Continued approval for this indication may be contingent upon verification of clinical benefit in a confirmatory trial.
LEQEMBI is the result of a strategic research alliance between Eisai and BioArctic. Eisai has been initiated submission of data for BLA to the National Medical Products Administration (NMPA) of China in December 2022. Eisai plans to file for marketing authorization applications of lecanemab in Japan and Europe by the end of Eisai's FY2022.
Since July 2020 Eisai's Phase 3 clinical study (AHEAD 3-45) for individuals with preclinical AD, meaning they are clinically normal and have intermediate or elevated levels of amyloid in their brains, is ongoing. AHEAD 3-45 is conducted as a public-private partnership between the Alzheimer's Clinical Trial Consortium that provides the infrastructure for academic clinical trials in AD and related dementias in the U.S, funded by the National Institute on Aging, part of the National Institutes of Health, Eisai and Biogen.
Since January 2022, the Tau NexGen clinical study for Dominantly Inherited AD (DIAD), that is conducted by Dominantly Inherited Alzheimer Network Trials Unit (DIAN-TU), led by Washington University School of Medicine in St. Louis, is ongoing. Eisai has completed a LEQEMBI subcutaneous bioavailability study, and subcutaneous dosing is currently being evaluated in the Clarity AD (Study 301) OLE.
2. About Amyloid-Related Imaging Abnormalities (ARIA)
ARIA is an important adverse event of amyloid-lowering therapies that is critical to monitor and manage during treatment. ARIA is most commonly seen as temporary swelling/effusion (ARIA-E) in areas of the brain that usually resolves over time. Some people may also have small spots of bleeding in or on the surface of the brain (ARIA-H) with the swelling. Although most people with ARIA-E do not have symptoms, some people may have symptoms such as headache, confusion, dizziness, vision changes and nausea.
3. About the Collaboration between Eisai and Biogen for AD
Eisai and Biogen have been collaborating on the joint development and commercialization of AD treatments since 2014. Eisai serves as the lead of LEQEMBI development and regulatory submissions globally with both companies co-commercializing and co-promoting the product and Eisai having final decision-making authority.
4. About the Collaboration between Eisai and BioArctic for AD
Since 2005, Eisai and BioArctic have had a long-term collaboration regarding the development and commercialization of AD treatments. Eisai obtained the global rights to study, develop, manufacture and market LEQEMBI for the treatment of AD pursuant to an agreement with BioArctic in December 2007. The development and commercialization agreement on the antibody LEQEMBI back-up was signed in May 2015.
5. About Eisai Co., Ltd.
Eisai's Corporate Concept is "to give first thought to patients and people in the daily living domain, and to increase the benefits that health care provides." Under this Concept (also known as human health care (hhc) Concept), we aim to effectively achieve social good in the form of relieving anxiety over health and reducing health disparities. With a global network of R&D facilities, manufacturing sites and marketing subsidiaries, we strive to create and deliver innovative products to target diseases with high unmet medical needs, with a particular focus in our strategic areas of Neurology and Oncology.
In addition, we demonstrate our commitment to the elimination of neglected tropical diseases (NTDs), which is a target (3.3) of the United Nations Sustainable Development Goals (SDGs), by working on various activities together with global partners.
For more information about Eisai, please visit www.eisai.com (for global headquarters: Eisai Co., Ltd.), and connect with us on Twitter @Eisai_SDGs.
6. About Biogen
As pioneers in neuroscience, Biogen discovers, develops and delivers worldwide innovative therapies for people living with serious neurological diseases as well as related therapeutic adjacencies. One of the world's first global biotechnology companies, Biogen was founded in 1978 by Charles Weissmann, Heinz Schaller, Sir Kenneth Murray, and Nobel Prize winners Walter Gilbert and Phillip Sharp. Today, Biogen has a leading portfolio of medicines to treat multiple sclerosis, has introduced the first approved treatment for spinal muscular atrophy, and developed the first approved treatment to address a defining pathology of Alzheimer's disease. Biogen is also commercializing biosimilars and focusing on advancing one of the industry's most diversified pipelines in neuroscience that will transform the standard of care for patients in several areas of high unmet need.
The company routinely posts information that may be important to investors on its website at www.biogen.com. To learn more, please visit www.biogen.com and Follow Biogen on social media – Twitter, LinkedIn, Facebook, YouTube.
View original content to download multimedia:https://www.prnewswire.com/news-releases/fda-approves-leqembi-lecanemab-irmb-under-the-accelerated-approval-pathway-for-the-treatment-of-alzheimers-disease-301715691.html
SOURCE Eisai Inc.
Biogen, Eisai's Alzheimer's treatment lecanemab approved by the FDA
Jan. 06, 2023 2:17 PM ET
Eisai Co., Ltd. (ESALF), ESALY, BIIB
By: Jonathan Block, SA News Editor23 Comments
tang90246
NOTE: Updated with information about Leqembi availability and price.
The US FDA on Friday approved Biogen (NASDAQ:BIIB) and Eisai's (OTCPK:ESALY) Alzheimer's therapy lecanemab, which will be marketed as Leqembi.
Eisai Co., Ltd. (ESALF), ESALY, BIIB
Qulipta (atogepant)
QULIPTA™ Now Approved by Health Canada for the Preventive Treatment of Episodic Migraine in Adults
Jan. 09, 2023 7:01 AM ET
- QULIPTA is the first and only oral calcitonin gene-related peptide (CGRP) receptor antagonist (gepant) specifically developed for the preventive treatment of episodic migraine.
- AbbVie is the only pharmaceutical company to offer three treatments across the full spectrum of migraine to help patients living with this debilitating disease.
MONTRÉAL, Jan. 9, 2023 /CNW Telbec/ - AbbVie (NYSE: ABBV) announced today that Health Canada has approved QULIPTA (atogepant) for the prevention of episodic migraine (< 15 migraine days per month) in adults.1 QULIPTA, the first and only oral calcitonin gene-related peptide (CGRP) receptor antagonist (gepant) preventive treatment, marks the latest innovation in AbbVie's migraine portfolio to support Canadians impacted by migraine.
"Migraine is one of the leading causes of disability in Canada and impacts a person's ability to function and perform their daily routines," says Dr. Elizabeth Leroux, Founder and Chair, Migraine Canada. "People with migraine face incredible challenges managing a debilitating neurological condition that others cannot see or truly understand. Migraine Canada welcomes the approval of QULIPTA for Canadians who will now have an opportunity to experience a new treatment that will help prevent and manage episodic migraine appropriately and take control of their migraine attacks before they even start."
The approval is supported by data from a robust clinical program evaluating the efficacy, safety and tolerability of QULIPTA in nearly 2,000 patients who experienced 4 to 14 migraine days per month. QULIPTA demonstrated statistically significant, clinically meaningful, rapid and continuous reductions in mean monthly migraine days among adults with episodic migraine compared to placebo across the 12-week treatment period with significant reductions seen in weeks 1-4. 1
"AbbVie is committed to bringing new innovative treatments to address the needs of Canadians living with migraine," says Tracey Ramsay, Vice President and General Manager, AbbVie Canada. "With the approval of QULIPTA for the preventive treatment of episodic migraine, AbbVie has expanded on our support to those living with migraine given the legacy of BOTOX® for the treatment of chronic migraine, and recently approved UBRELVY® for acute treatment of migraine. AbbVie is proud to partner with the migraine community to advance treatment and care for people living with this neurological disease."
About Migraine
Migraine is a complex neurological disease with recurrent attacks that lasts 4-72 hours. It can be defined by symptoms such as moderate to severe pain intensity, nausea, vomiting, photophobia and phonophobia.2 An estimated 2.7 million Canadians are reported diagnosed with migraine, however the number of people living with migraine is much higher.3 Episodic migraine is characterized as having less than 15 headache days per month, while 15 headache days or more per month, is considered chronic. 4
About QULIPTA (atogepant)
QULIPTA (atogepant) is the first and only oral calcitonin gene-related peptide (CGRP) receptor antagonist (gepant) specifically developed for the preventive treatment of episodic migraine. QULIPTA is an orally administered, small molecule, selective calcitonin gene-related peptide (CGRP) receptor antagonist that blocks the binding of the CGRP to its receptor. CGRP is a neuropeptide that may play a role in migraine pathophysiology.1
For important safety information, please consult the QULIPTA™ Product Monograph at www.abbvie.ca.
About AbbVie
AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women's health and gastroenterology, in addition to products and services across its Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.ca. Follow AbbVie Canada on Twitter, on Instagram or find us on LinkedIn.
For more information on AbbVie's complete migraine portfolio, please visit www.abbvie.ca.
SOURCE AbbVie Canada
Trodelvy® (sacituzumab govitecan-hziy)
January 03, 2023
European Medicines Agency Validates Marketing Authorization Application For Trodelvy® (sacituzumab govitecan-hziy) For Pre-treated HR+/HER2- Metastatic Breast Cancer
– Application Based on Statistically Significant and Clinically Meaningful Overall Survival and Progression-Free Survival Results from the Phase 3 TROPiCS-02 Study –
– Supplemental Biologics License Application Already Under Priority Review by the U.S. FDA –
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced that the European Medicines Agency (EMA) has validated a Type II variation Marketing Authorization Application (MAA) for Trodelvy® (sacituzumab govitecan-hziy) for the treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative (IHC 0, IHC 1+ or IHC 2+/ISH–) breast cancer who have received endocrine-based therapy and at least two additional systemic therapies in the metastatic setting.
“At Gilead Oncology our ambition is to transform care for people with cancer,” said Bill Grossman, MD, PhD, Senior Vice President, Therapeutic Area Head, Gilead Oncology. “Trodelvy is already moving us towards this ambition and changing the standard of care in second-line metastatic triple-negative breast cancer across the EU. The validation of our Marketing Authorization Application in pre-treated HR+/HER2- metastatic breast cancer marks an important step forward to potentially making Trodelvy available to even more patients with severely limited treatment options.”
This Marketing Authorization Application is based on data from the registrational Phase 3 TROPiCS-02 study, which met its primary endpoint of progression-free survival (PFS) and key secondary endpoint of overall survival (OS) versus comparator chemotherapies (treatment of physician’s choice (TPC) of chemotherapy). PFS data were published in the Journal of Clinical Oncology,and OS data were recently presented at ESMO Congress 2022.
The safety profile for Trodelvy in TROPiCS-02 was consistent with prior studies, and no new safety signals were identified in this population.
In October 2022, the U.S. Food and Drug Administration (FDA) accepted for priority review the supplemental Biologics License Application (sBLA) for Trodelvy for the treatment of adult patients with unresectable locally advanced or metastatic HR+/HER2-negative (IHC 0, IHC 1+ or IHC 2+/ISH–) breast cancer who have received endocrine-based therapy and at least two additional systemic therapies in the metastatic setting. The Prescription Drug User Fee Act (PDUFA) target action date is currently set for February 2023.
Trodelvy has not been approved by any regulatory agency for the treatment of HR+/HER2- metastatic breast cancer. Its safety and efficacy have not been established for this indication.
Trodelvy has a Boxed Warning for severe or life-threatening neutropenia and severe diarrhea; please see below for additional Important Safety Information.
About the TROPiCS-02 Study
The TROPiCS-02 study is a global, multicenter, open-label, Phase 3 study, randomized 1:1 to evaluate Trodelvy versus physicians’ choice of chemotherapy (eribulin, capecitabine, gemcitabine, or vinorelbine) in 543 patients with HR+/HER2- metastatic breast cancer who were previously treated with endocrine therapy, CDK4/6 inhibitors and two to four lines of chemotherapy for metastatic disease. The primary endpoint is progression-free survival per Response Evaluation Criteria in Solid Tumors (RECIST 1.1) as assessed by blinded independent central review (BICR) for participants treated with Trodelvy compared to those treated with chemotherapy. Secondary endpoints include overall survival, overall response rate, clinical benefit rate and duration of response, as well as assessment of safety and tolerability and quality of life measures. In the study, HER2 negativity was defined per American Society of Clinical Oncology (ASCO) and the College of American Pathologists (CAP) criteria as immunohistochemistry (IHC) score of 0, IHC 1+ or IHC 2+ with a negative in-situ hybridization (ISH) test. More information about TROPiCS-02 is available at https://clinicaltrials.gov/ct2/show/NCT03901339.
About Trodelvy
Trodelvy® (sacituzumab govitecan-hziy) is a first-in-class Trop-2 directed antibody-drug conjugate. Trop-2 is a cell surface antigen highly expressed in multiple tumor types, including in more than 90% of breast and bladder cancers. Trodelvy is intentionally designed with a proprietary hydrolyzable linker attached to SN-38, a topoisomerase I inhibitor payload. This unique combination delivers potent activity to both Trop-2 expressing cells and the microenvironment.
Trodelvy is approved in more than 40 countries, with multiple additional regulatory reviews underway worldwide, for the treatment of adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) who have received two or more prior systemic therapies, at least one of them for metastatic disease. Trodelvy is also approved in the U.S. under the accelerated approval pathway for the treatment of adult patients with locally advanced or metastatic urothelial cancer (UC) who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor.
Trodelvy is also being developed for potential investigational use in other TNBC and metastatic UC populations, as well as a range of tumor types where Trop-2 is highly expressed, including HR+/HER2- metastatic breast cancer, metastatic non-small cell lung cancer (NSCLC), metastatic small cell lung cancer (SCLC), head and neck cancer, and endometrial cancer.
U.S. Indications for Trodelvy
In the United States, Trodelvy is indicated for the treatment of:
- Adult patients with unresectable locally advanced or metastatic TNBC who have received two or more prior systemic therapies, at least one of them for metastatic disease.
- Adult patients with locally advanced or metastatic UC who have previously received a platinum-containing chemotherapy and either programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor. 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 a confirmatory trial.
- https://www.trodelvy.com/bladder-cancer
- Please see full Prescribing Information , including BOXED WARNING.
View source version on businesswire.com: https://www.businesswire.com/news/home/20230102005076/en/
Source: Gilead Sciences, Inc.
EMA approves change to marketing authorization for Gilead's breast cancer drug Trodelvy
Jan. 03, 2023 10:38 AM ET
By: Anuron Mitra, SA News Editor
Gilead Sciences (NASDAQ:GILD) on Tuesday said Europe's drug regulator had approved a change to its marketing authorization application for breast cancer treatment Trodelvy.
https://www.trodelvy.com/bladder-cancer
CARVYKTI® (ciltacabtagene autoleucel)
Legend Biotech Announces Acceptance of Its New Drug Application for Ciltacabtagene Autoleucel (Cilta-Cel) in China
January 02, 2023 04:45 AM Eastern Standard Time
SOMERSET, N.J.--(BUSINESS WIRE)--Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global biotechnology company developing, manufacturing and commercializing novel therapies to treat life-threatening diseases, announced today that China’s National Medical Products Administration (NMPA) has formally accepted its New Drug Application (NDA) for ciltacabtagene autoleucel (cilta-cel).
This submission is based on data from the confirmatory Phase 2 clinical study CARTIFAN-1 (NCT03758417) conducted in China, which evaluated the efficacy and safety of cilta-cel in adult patients with relapsed or refractory multiple myeloma who have received 3 or more prior lines of therapy, including a proteasome inhibitor and immunomodulatory drug.
Saijuan Chen, M.D., hematologist and molecular geneticist, Academician of the Chinese Academy of Engineering, and principal investigator of the CARTIFAN-1 clinical trial, said: “Incidence and mortality rates of multiple myeloma have recently increased in China, and the disease remains incurable. As a result, there is a huge unmet medical need for new treatment options. The data from the CARTIFAN-1 study showed that cilta-cel provided deep and durable responses in patients with relapsed or refractory multiple myeloma. We hope this drug becomes available to eligible patients as soon as possible.”
Ying Huang, Ph.D., Chief Executive Officer of Legend Biotech, said: “Meeting medical needs and serving patients around the world has always been the goal of Legend Biotech’s innovative research and development. Cilta-cel has been approved for marketing in the United States and Japan and has received conditional marketing authorization in Europe. We look forward to the possibility of providing a new treatment option for appropriate patients with relapsed and refractory multiple myeloma in China.”
###
About Ciltacabtagene autoleucel (cilta-cel)
Ciltacabtagene autoleucel is a BCMA-directed, genetically modified autologous T-cell immunotherapy, which involves reprogramming a patient’s own T-cells with a transgene encoding a chimeric antigen receptor (CAR) that identifies and eliminates cells that express BCMA. BCMA is primarily expressed on the surface of malignant multiple myeloma B-lineage cells, as well as late-stage B-cells and plasma cells. The cilta-cel CAR protein features two BCMA-targeting single domain antibodies designed to confer high avidity against human BCMA. Upon binding to BCMA-expressing cells, the CAR promotes T-cell activation, expansion, and elimination of target cells.1
In December 2017, Legend Biotech Corporation entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel.
In February 2022, cilta-cel was approved by the U.S. Food and Drug Administration (FDA) under the brand name CARVYKTI® for the treatment of adults with relapsed or refractory multiple myeloma.2 In May 2022, the European Commission (EC) granted conditional marketing authorization of CARVYKTI® for the treatment of adults with relapsed and refractory multiple myeloma.3 In September 2022, Japan’s Ministry of Health, Labour and Welfare (MHLW) approved CARVYKTI®.4 Cilta-cel was granted Breakthrough Therapy Designation in the U.S. in December 2019 and in China in August 2020. In addition, cilta-cel received a PRIority MEdicines (PRIME) designation from the European Commission in April 2019. Cilta-cel also received Orphan Drug Designation from the U.S. FDA in February 2019, from the European Commission in February 2020, and from the Pharmaceuticals and Medicinal Devices Agency (PMDA) in Japan in June 2020. In March 2022, the European Medicines Agency’s Committee for Orphan Medicinal Products recommended by consensus that the orphan designation for cilta-cel be maintained on the basis of clinical data demonstrating improved and sustained complete response rates following treatment.5
About CARTIFAN-1
CARTIFAN-1 (NCT03758417)6 is a Phase 2 open-label, confirmatory trial evaluating the efficacy and safety of cilta-cel in Chinese patients with relapsed/refractory multiple myeloma who have received at least three prior lines of treatments including a proteasome inhibitor and immunomodulatory drug. The primary endpoint is overall response rate.
CARVYKTI® U.S. Important Safety Information
INDICATIONS AND USAGE
CARVYKTI® (ciltacabtagene autoleucel) is a B-cell maturation antigen (BCMA)-directed genetically modified autologous T cell immunotherapy indicated for the treatment of adult patients with relapsed or refractory multiple myeloma, after four or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.
Please read full Prescribing Information including Boxed Warning for CARVYKTI®.
About Legend Biotech
Legend Biotech is a global biotechnology company dedicated to treating, and one day curing, life-threatening diseases. Headquartered in Somerset, New Jersey, we are developing advanced cell therapies across a diverse array of technology platforms, including autologous and allogeneic chimeric antigen receptor T-cell and natural killer (NK) cell-based immunotherapy. From our three R&D sites around the world, we apply these innovative technologies to pursue the discovery of cutting-edge therapeutics for patients worldwide.
Learn more at www.legendbiotech.com and follow us on Twitter and LinkedIn.
J&J, Legend Biotech's Carvykti for multiple myeloma gets review in China
Jan. 03, 2023 4:24 AM ET
Legend Biotech Corporation (LEGN), JNJ
By: Ravikash, SA News Editor
- Legend Biotech (NASDAQ:LEGN) said on Monday that China's National Medical Products Administration (NMPA) accepted its new drug application (NDA) seeking approval for ciltacabtagene autoleucel (cilta-cel) to treat a type of blood cancer.
- https://seekingalpha.com/news/3921428-jj-legend-biotechs-carvykti-for-multiple-myeloma-gets-review-in-china
- Legend Biotech Corporation (LEGN), JNJ
- https://www.carvykti.com/
- https://legendbiotech.com/research-development/pipeline/
- https://legendbiotech.com/
Tislelizumab (BGB-A317)
BeiGene Announces Acceptance of 12th Regulatory Submission in China for PD-1 Inhibitor Tislelizumab
Dec 30, 2022 9:00 AM
Submission seeks marketing authorization for first-line treatment of unresectable or metastatic hepatocellular carcinoma
CAMBRIDGE, Mass. & BASEL, Switzerland & BEIJING--(BUSINESS WIRE)-- BeiGene (NASDAQ: BGNE; HKEX: 06160; SSE: 688235), a global biotechnology company 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 tislelizumab in patients with first-line unresectable or metastatic hepatocellular carcinoma (HCC).
Hepatocellular carcinoma is the most common type of primary liver cancer worldwide and is associated with a very poor prognosis.1 New cases and deaths due to HCC in China account for half of the global numbers and the 5-year survival rate for patients with HCC in China is only 14%.2
“While the incidence of HCC is increasing in China, the treatment landscape has not advanced accordingly; survival benefits with newer treatments are modest and multi-kinase inhibitors have sub-optimal tolerability,” said Lai Wang, Ph.D., Global Head of R&D at BeiGene. “We believe the evidence from our rigorously conducted global clinical development program for tislelizumab in HCC support the efficacy and favorable tolerability profile and look forward to working with NMPA on this submission and bringing a new treatment option to patients with HCC in China.”
The sBLA is supported by data from the RATIONALE 301 clinical trial (NCT03412773) that enrolled 674 patients from research centers across Asia, Europe, and the United States. RATIONALE 301 results were presented as a late-breaking oral presentation at the 2022 European Society for Medical Oncology (ESMO) Congress in Paris.
Tislelizumab was approved by the China NMPA as a treatment for nine indications, including conditional approval ‘for the treatment of patients with hepatocellular carcinoma (HCC) who have been previously treated with at least one systemic therapy’. Additional tislelizumab’s sBLAs under review at CDE include: combination with chemotherapy as a first-line treatment for patients with advanced or metastatic gastric or gastroesophageal junction adenocarcinoma whose tumors express PD-L1; combination with chemotherapy as first-line treatment in patients with unresectable locally advanced, recurrent or metastatic esophageal squamous cell carcinoma. Tislelizumab is not approved for use outside of China.
About Tislelizumab
Tislelizumab is a humanized IgG4 anti-PD-1 monoclonal antibody specifically designed to minimize binding to Fc-gamma (Fcγ) receptors on macrophages, helping to aid the body’s immune cells to detect and fight tumors. In pre-clinical studies, binding to Fcγ receptors 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 investigational medicine from BeiGene’s immuno-oncology biologics program and is being evaluated in solid tumor and hematologic malignancies, as monotherapy and in combination.
The global tislelizumab clinical development program includes more than 11,500 subjects enrolled to-date in 21 registration-enabling trials, from more than 30 countries and regions.
https://www.beigene.com/our-science-and-medicines/tislelizumab/
About RATIONALE 301
RATIONALE 301 (NCT03412773) is a global, Phase 3, randomized, open-label study of tislelizumab compared with sorafenib as a first-line treatment in adult patients with unresectable HCC. The primary endpoint of the study is non-inferiority of Overall Survival between the two treatment groups. The key secondary endpoint is Overall Response Rate, as assessed by Blinded Independent Review Committee (BIRC) per RECIST v1.1. Other secondary endpoints include other efficacy assessments such as Progression Free Survival, Duration of Response, and Time to Progression per BIRC, as well as measures of health-related quality of life, and safety and tolerability.
About BeiGene
BeiGene is a global biotechnology company that is developing and commercializing innovative and affordable oncology medicines to improve treatment outcomes and access for far more patients worldwide. With a broad portfolio, we are expediting development of our diverse pipeline of novel therapeutics through our internal capabilities and collaborations. We are committed to radically improving access to medicines for far more patients who need them. Our growing global team of more than 9,000 colleagues spans five continents, with administrative offices in Cambridge, U.S., & Basel, Switzerland & Beijing, China. To learn more about BeiGene, please visit www.beigene.com and follow us on Twitter at @BeiGeneGlobal.
View source version on businesswire.com: https://www.businesswire.com/news/home/20221230005150/en/
Source: BeiGene
China accepts BeiGene's supplemental license application for treatment of liver cancer
Dec. 30, 2022 9:37 AM ET
By: Anuron Mitra, SA News Editor
- BeiGene (NASDAQ:BGNE) on Friday said China's drug regulator had accepted its supplemental biologics license application (sBLA) for its inhibitor tislelizumab for the treatment of the most common type of liver cancer.
- https://seekingalpha.com/news/3921278-china-accepts-beigenes-supplemental-license-application-for-treatment-of-liver-cancer
- BeiGene, Ltd. (BGNE)
- https://www.beigene.com/our-science-and-medicines/tislelizumab/
- https://www.beigene.com/
biotecMAX™ 2022 Insight
Imfinzi (durvalumab) plus Imjudo (tremelimumab)
Imfinzi plus Imjudo approved in Japan for advanced liver and non-small cell lung cancers, and Imfinzi approved for unresectable biliary tract and liver cancers
PUBLISHED28 December 2022 28 December 2022 07:00 GMT
Approvals based on significant survival benefits in
HIMALAYA, POSEIDON and TOPAZ-1 Phase III trials
AstraZeneca’s immunotherapies Imfinzi (durvalumab) and Imjudo (tremelimumab) have been approved in Japan for the treatment of three cancer types: advanced liver, biliary tract and lung.
The approvals authorise Imfinzi in combination with Imjudo for the treatment of adult patients with unresectable hepatocellular carcinoma (HCC) and for the treatment of adult patients with unresectable, advanced or recurrent non-small cell lung cancer (NSCLC) in combination with chemotherapy. Imfinzi was also authorised for the treatment of adult patients with unresectable HCC as monotherapy and for the treatment of adult patients with curatively unresectable biliary tract cancer (BTC) in combination with chemotherapy (gemcitabine plus cisplatin).
The concurrent approvals by the Japanese Ministry of Health, Labour, and Welfare are based on positive results from the HIMALAYA and TOPAZ-1 Phase III trials, each published in the New England Journal of Medicine Evidence and the POSEIDON Phase III trial, published in the Journal of Clinical Oncology.
Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “Japan has one of the highest rates of diagnosis for liver and biliary tract cancers in the world, and lung cancer remains the country’s leading cause of cancer death. With these approvals for Imfinzi and Imjudo, patients in Japan can now be treated with novel immunotherapy-based treatment regimens that have demonstrated significant survival benefits across three complex cancers with poor prognoses.”
Imfinzi and Imjudo approved in liver cancer
The approval of Imfinzi in combination with Imjudo for the treatment of unresectable HCC brings the first dual immunotherapy treatment regimen to patients in Japan. The approval is based on results from the HIMALAYA Phase III trial, in which a single dose of the anti-CTLA-4 antibody Imjudo 300mg added to the anti-PD-L1 antibody Imfinzi 1500mg followed by Imfinzi every four weeks (STRIDE regimen: Single Tremelimumab Regular Interval Durvalumab) significantly reduced the risk of death versus sorafenib. The addition of Imjudo to Imfinzi did not increase severe liver toxicity, and no bleeding risk was observed.
HIMALAYA also served as the basis for the approval of Imfinzi monotherapy in the same disease setting. In HIMALAYA, Imfinzi demonstrated non-inferior overall survival (OS) compared to sorafenib, and an improved tolerability profile versus sorafenib.
The safety profiles of the combination of Imjudo added to Imfinzi and for Imfinzi alone were consistent with the known profiles of each medicine, and no new safety signals were identified.
Liver cancer is the fifth-leading cause of cancer death in Japan and the sixth most commonly diagnosed cancer worldwide.1,2 HCC is the most common type of liver cancer with 80% of cases occurring in the Asia-Pacific region.3,4 In Japan, approximately 45,000 people are diagnosed with HCC and 32,000 die of the disease each year.5,6
Imfinzi and Imjudo approved in NSCLC
The approval of Imfinzi and Imjudo plus chemotherapy for the treatment of unresectable, advanced or recurrent NSCLC is based on results from the POSEIDON Phase III trial, which showed a limited course of five cycles of the anti-CTLA-4 antibody Imjudo added to Imfinzi plus four cycles of platinum-based chemotherapy significantly reduced the risk of death versus a range of chemotherapy options.
The safety profile for Imjudo plus Imfinzi and chemotherapy was consistent with the known profiles of each medicine, and no new safety signals were identified.
In Japan, lung cancer is the most commonly diagnosed cancer, with more than 138,000 patients diagnosed in 2020.1 The prognosis for patients with metastatic NSCLC in Japan is particularly poor, as less than 20% will live beyond three years after diagnosis without treatment.7
Regulatory applications for Imfinzi and Imjudo are currently under review in the EU and several other countries based on the HIMALAYA, TOPAZ-1 and POSEIDON results.
Imfinzi plus chemotherapy approved in biliary tract cancer
The approval of Imfinzi plus chemotherapy brings the first immunotherapy regimen to patients with curatively unresectable BTC in Japan after more than a decade of limited innovation. The approval is based on results from an interim analysis of the TOPAZ-1 Phase III trial, which showed that Imfinzi plus chemotherapy significantly reduced the risk of death compared to chemotherapy alone. Imfinzi plus chemotherapy was generally well tolerated and did not increase the discontinuation rate due to adverse events compared to chemotherapy alone.
BTC is a group of rare and aggressive cancers that occur in the bile ducts and gallbladder.8,9 In 2021, approximately 23,300 people in Japan were diagnosed with BTC, which is the sixth-leading cause of cancer-related deaths for women and seventh-leading cause of cancer-related death for men in Japan.7 Patients face a poor prognosis, with approximately 19% to 31% of patients with BTC surviving five years.7
Notes
HIMALAYA
HIMALAYA was a randomised, open-label, multicentre, global Phase III trial of Imfinzi monotherapy and the STRIDE regimen comprising a single priming dose of Imjudo 300mg added to Imfinzi 1500mg followed by Imfinzi every four weeks versus sorafenib, a standard-of-care multi-kinase inhibitor.
The trial included a total of 1,324 patients with unresectable, advanced HCC who had not been treated with prior systemic therapy and were not eligible for locoregional therapy (treatment localised to the liver and surrounding tissue).
The trial was conducted in 181 centres across 16 countries, including in the US, Canada, Europe, South America and Asia. The primary endpoint was OS for the combination versus sorafenib and key secondary endpoints included OS for Imfinzi versus sorafenib, objective response rate (ORR) and progression-free survival (PFS) for the combination and for Imfinzi alone.
In HIMALAYA, the STRIDE regimen reduced the risk of death by 22% versus sorafenib (hazard ratio [HR] 0.78; 95% confidence interval [CI], 0.66-0.92; p= 0.0035). An estimated 31% of patients treated with the combination were still alive after three years, with 20% of patients treated with sorafenib still alive at the same duration of follow-up. The safety profiles of the combination of Imjudo added to Imfinzi and for Imfinzi alone were consistent with the known profiles of each medicine, and no new safety signals were identified.
POSEIDON
The POSEIDON trial was a randomised, open-label, multi-centre, global, Phase III trial of Imfinzi plus platinum-based chemotherapy, or Imfinzi, Imjudo and chemotherapy, versus chemotherapy alone in the 1st-line treatment of 1,013 patients with metastatic NSCLC. The trial population included patients with either non-squamous or squamous disease, and the full range of PD-L1 expression levels. POSEIDON excluded patients with certain epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) fusions.
In the experimental arms, patients were treated with a flat dose of either Imfinzi (1,500mg) or Imfinzi plus Imjudo (75mg) with up to four cycles of chemotherapy every three weeks before either Imfinzi maintenance once every four weeks or Imfinzi and a fifth dose of Imjudo given at week 16. In comparison, the control arm allowed up to six cycles of chemotherapy. Pemetrexed maintenance treatment was allowed in all arms in patients with non-squamous disease if given during the induction phase. Nearly all patients with non-squamous disease (95.5%) had pemetrexed and platinum, while the majority of patients with squamous disease receiving chemotherapy (88.3%) received gemcitabine and platinum.
Primary endpoints included PFS and OS for the Imfinzi plus chemotherapy arm. Key secondary endpoints included PFS and OS in the Imfinzi plus Imjudo and chemotherapy arm. As both PFS endpoints were met for Imfinzi plus chemotherapy and Imfinzi, Imjudo and chemotherapy, the prespecified statistical analysis plan allowed for testing OS in the Imfinzi plus Imjudo and chemotherapy arm. The trial was conducted in more than 150 centres across 18 countries, including the US, Europe, South America, Asia and South Africa.
Patients treated with a limited course of five cycles of the anti-CTLA-4 antibody Imjudo added to Imfinzi plus four cycles of platinum-based chemotherapy experienced a 23% reduction in the risk of death versus a range of chemotherapy options (HR 0.77; 95% CI, 0.65-0.92; p=0.00304). An estimated 33% of patients were alive at two years versus 22% for chemotherapy. This treatment combination also reduced the risk of disease progression or death by 28% compared to chemotherapy alone (HR 0.72; 95% CI, 0.60-0.86; p=0.00031).
Updated results after approximately four years of follow-up presented at the European Society for Medical Oncology Congress 2022 demonstrated sustained survival benefit, improving overall survival (OS) by 25% compared to chemotherapy alone (HR 0.75; 95% CI, 0.63-0.88). An estimated 25% of patients treated with the combination were alive at three years versus 13.6% for those treated with chemotherapy alone. The safety profile for Imjudo plus Imfinzi and chemotherapy was consistent with the known profiles of each medicine, and no new safety signals were identified.
TOPAZ-1
TOPAZ-1 was a randomised, double-blind, placebo controlled, multicentre, global Phase III trial of Imfinzi in combination with chemotherapy (gemcitabine plus cisplatin) versus placebo in combination with chemotherapy as a 1st-line treatment in 685 patients with unresectable advanced or metastatic BTC including intrahepatic and extrahepatic cholangiocarcinoma, and gallbladder cancer. Patients with ampullary carcinoma were excluded.
The primary endpoint was OS and key secondary endpoints included PFS, ORR and safety. The trial was conducted in 105 centres across 17 countries including in the US, Europe, South America and several countries in Asia including South Korea, Thailand, Japan and China.
At the interim analysis, Imfinzi plus chemotherapy reduced the risk of death by 20% versus chemotherapy alone (based on a hazard ratio [HR] of 0.80; 95% confidence interval [CI] 0.66-0.97; p=0.021). Updated results from TOPAZ-1 after an additional 6.5 months of follow-up showed a 24% reduction in the risk of death versus chemotherapy alone (HR 0.76; 95% CI, 0.64-0.91), with more than two times as many patients treated with Imfinzi plus chemotherapy estimated to be alive at two years versus chemotherapy alone (23.6% versus 11.5%). Updated median overall survival (OS) was 12.9 months versus 11.3 with chemotherapy. Imfinzi plus chemotherapy was generally well tolerated, with no new safety signals observed, and did not increase the discontinuation rate due to adverse events (AEs) compared to chemotherapy alone.
Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour's immune-evading tactics and releasing the inhibition of immune responses.
Imfinzi is the only approved immunotherapy in the curative-intent setting of unresectable, Stage III NSCLC in patients whose disease has not progressed after chemoradiotherapy and is the global standard of care in this setting based on the PACIFIC Phase III trial.
Imfinzi is also approved in the US, EU, Japan, China and many other countries around the world for the treatment of extensive-stage small cell lung cancer (ES-SCLC) based on the CASPIAN Phase III trial. In 2021, updated results from the CASPIAN trial showed Imfinzi plus chemotherapy tripled patient survival at three years versus chemotherapy alone.
Imfinzi is also approved in combination with Imjudo and chemotherapy in metastatic non-small cell lung cancer in the US and Japan; in combination with chemotherapy in locally advanced or metastatic BTC in the US, Japan and several other countries; in combination with Imjudo in unresectable HCC in the US and Japan; as monotherapy in unresectable HCC in Japan; and in previously treated patients with advanced bladder cancer in several countries.
Since the first approval in May 2017, more than 100,000 patients have been treated with Imfinzi.
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 SCLC, NSCLC, bladder cancer, several gastrointestinal (GI) cancers, ovarian cancer, endometrial cancer and other solid tumours.
Imjudo
Imjudo (tremelimumab) is a human monoclonal antibody that targets the activity of cytotoxic T-lymphocyte-associated protein 4 (CTLA-4). Imjudo blocks the activity of CTLA-4, contributing to T-cell activation, priming the immune response to cancer and fostering cancer cell death.
In addition to its approved indications in liver and lung cancers, Imjudo is being tested in combination with Imfinzi across multiple tumour types including locoregional HCC (EMERALD-3), SCLC (ADRIATIC) and bladder cancer (VOLGA and NILE).
AstraZeneca in GI cancers
AstraZeneca has a broad development programme for the treatment of GI cancers across several medicines and a variety of tumour types and stages of disease. In 2020, GI cancers collectively represented approximately 5.1 million new cancer cases leading to approximately 3.6 million deaths.10
Within this programme, the Company is committed to improving outcomes in gastric, liver, BTC, oesophageal, pancreatic, and colorectal cancers.
Imfinzi is approved in the US and several other countries in combination with chemotherapy (gemcitabine plus cisplatin) for advanced biliary tract cancer and in the US and Japan in combination with Imjudo in unresectable HCC. Imfinzi is also approved as a monotherapy in unresectable HCC in Japan. Imfinzi is being assessed in combinations, including with Imjudo, in liver, oesophageal and gastric cancers in an extensive development programme spanning early to late-stage disease across settings.
Enhertu (trastuzumab deruxtecan), a HER2-directed antibody drug conjugate, is approved in the US and several other countries for HER2-positive advanced gastric cancer and is being assessed in colorectal cancer. Enhertu is jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.
Lynparza (olaparib), a first-in-class PARP inhibitor, is approved in the US and several other countries for the treatment of BRCA-mutated metastatic pancreatic cancer. Lynparza is developed and commercialised in collaboration with MSD (Merck & Co., Inc. inside the US and Canada).
AstraZeneca in lung cancer
AstraZeneca is working to bring patients with lung cancer closer to cure through the detection and treatment of early-stage disease, while also pushing the boundaries of science to improve outcomes in the resistant and advanced settings. By defining new therapeutic targets and investigating innovative approaches, the Company aims to match medicines to the patients who can benefit most.
The Company's comprehensive portfolio includes leading lung cancer medicines and the next wave of innovations, including Tagrisso (osimertinib) and Iressa (gefitinib); Imfinzi and Imjudo; Enhertu and datopotamab deruxtecan in collaboration with Daiichi Sankyo; Orpathys (savolitinib) in collaboration with HUTCHMED; as well as a pipeline of potential new medicines and combinations across diverse mechanisms of action.
AstraZeneca is a founding member of the Lung Ambition Alliance, a global coalition working to accelerate innovation and deliver meaningful improvements for people with lung cancer, including and beyond treatment.
AstraZeneca in immuno-oncology (IO)
AstraZeneca is a pioneer in introducing the concept of immunotherapy into dedicated clinical areas of high unmet medical need. The Company has a comprehensive and diverse IO portfolio and pipeline anchored in immunotherapies designed to overcome evasion of the anti-tumour immune response and stimulate the body’s immune system to attack tumours.
AstraZeneca aims to reimagine cancer care and help transform outcomes for patients with Imfinzi as a single treatment and in combination with Imjudo as well as other novel immunotherapies and modalities. The Company is also exploring next-generation immunotherapies like bispecific antibodies and therapeutics that harness different aspects of immunity to target cancer.
AstraZeneca is boldly pursuing an innovative clinical strategy to bring IO-based therapies that deliver long-term survival to new settings across a wide range of cancer types. With an extensive clinical programme, the Company also champions the use of IO treatment in earlier disease stages, where there is the greatest potential for cure.
AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.
The Company's focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.
AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca's Imjudo/Imfinzi combo gets approval in Japan for liver, lung cancers
Dec. 28, 2022 4:40 AM ET
AstraZeneca PLC (AZN) By: Ravikash, SA News Editor
Japan's Ministry of Health, Labour, and Welfare approved AstraZeneca's (NASDAQ:AZN) immunotherapies Imfinzi and Imjudo to treat certain types of liver and lung cancers and Imfinzi to treat biliary tract and liver cancers.
Calquence (acalabrutinib)
Calquence approved in Japan for adults with treatment-naïve chronic lymphocytic leukaemia
PUBLISHED28 December 2022
28 December 2022 07:05 GMT
Calquence significantly increased the time patients lived without disease progression or death vs. chemoimmunotherapy
AstraZeneca’s Calquence (acalabrutinib), a selective Bruton’s tyrosine kinase (BTK) inhibitor, has been approved in Japan for the treatment of adult patients with treatment-naïve chronic lymphocytic leukaemia (CLL) (including small lymphocytic lymphoma [SLL]). Calquence was previously approved in Japan for the treatment of adults with relapsed or refractory CLL.
The approval by the Japanese Ministry of Health, Labour and Welfare (MHLW) was based on positive results from two clinical trials, including the ELEVATE-TN Phase III trial in adults with treatment-naïve CLL. This trial showed that Calquence combined with obinutuzumab or as monotherapy demonstrated a significantly improved progression-free survival (PFS) when compared with the chemotherapy-based combination of chlorambucil and obinutuzumab.1,2 Data from the interim analysis of ELEVATE-TN was published in The Lancet in 2020.2 Additionally, a Phase I trial in treatment-naïve Japanese patients with CLL was also submitted to MHLW supporting the approval, with the trial showing an overall response rate of 88.9% (95% CI: 63.2, 98.8%) for Calquence alone and 100% (95% CI: 66.4, 100%) for Calquence combined with obinutuzumab.
CLL is the most prevalent type of adult leukaemia across the globe but is considered a rare disease in Japan and East Asia, with fewer than one person newly diagnosed per 100,000 persons per year across Japan.3-5
Koji Izutsu, MD, PhD, Department Head, Department of Hematology, National Cancer Center Hospital, Tokyo, Japan, said: “Results from ELEVATE-TN and our local Japanese trial confirm that Calquence provides a significant improvement in progression-free survival compared with chemotherapy-based combination of chlorambucil and obinutuzumab for patients with treatment-naïve chronic lymphocytic leukaemia. Today’s approval marks great progress for physicians and patients in Japan, as they can now be treated with Calquence earlier in their treatment journey.”
Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “The approval of Calquence in Japan for those with treatment-naïve chronic lymphocytic leukaemia now offers more patients a next-generation Bruton’s tyrosine kinase inhibitor that has proven longer-term efficacy and tolerability compared to standards of care. With this approval, people living with chronic lymphocytic leukaemia in Japan can now potentially benefit from our medicine in an earlier setting.”
Updated results of the ELEVATE-TN Phase III trial after a median follow-up of approximately five years were presented earlier this year. These results showed that Calquence maintained a statistically significant PFS benefit versus chlorambucil plus obinutuzumab, and a safety and tolerability profile consistent with the known profile for Calquence. At a median follow-up of 58.2 months, Calquence plus obinutuzumab reduced the risk of disease progression or death by 89% (based on a hazard ratio [HR] of 0.11, 95% confidence interval [CI] 0.07-0.16) and as a monotherapy by 79% (based on a HR of 0.21, 95% CI 0.15-0.30), compared with chlorambucil plus obinutuzumab.1
Calquence is approved for the treatment of CLL and SLL in the US and is approved for the treatment of CLL in the EU and in several other countries worldwide in the treatment-naïve and relapsed or refractory settings. Calquence is also approved in the US and several other countries for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy. Calquence is not currently approved for the treatment of MCL in Japan or the EU.
Notes
CLL
CLL is the most prevalent type of leukaemia in adults, with over 100,000 new cases globally in 2019, but is considered a rare disease in Japan and East Asia, with fewer than one person newly diagnosed per 100,000 persons per year across Japan.3-5 Although some people with CLL may not experience any symptoms at diagnosis, others may experience symptoms, such as weakness, fatigue, weight loss, chills, fever, night sweats, swollen lymph nodes and abdominal pain.6
In CLL, there is an accumulation of abnormal lymphocytes within the bone marrow. As the number of abnormal cells increases, there is less room within the marrow for the production of normal white blood cells, red blood cells and platelets.7 This could result in anaemia, infection and bleeding. B-cell receptor signalling through BTK is one of the essential growth pathways for CLL.
ELEVATE-TN
ELEVATE-TN (ACE-CL-007) is a randomised, multicentre, open-label Phase III trial evaluating the safety and efficacy of Calquence alone or in combination with obinutuzumab versus chlorambucil in combination with obinutuzumab in previously untreated patients with CLL. In the trial, 535 patients were randomised (1:1:1) into three arms. Patients in the first arm received chlorambucil in combination with obinutuzumab. Patients in the second arm received Calquence (100mg twice daily until disease progression) in combination with obinutuzumab. Patients in the third arm received Calquence monotherapy (100mg twice daily until disease progression).8
The primary endpoint was PFS in the Calquence and obinutuzumab arm compared to the chlorambucil and obinutuzumab arm, assessed by an independent review committee (IRC), and a key secondary endpoint was IRC-assessed PFS in the Calquence monotherapy arm compared to the chlorambucil and obinutuzumab arm. Other secondary endpoints included overall response rate, time to next treatment, overall survival and investigator assessed PFS. After interim analysis, assessments were by investigator only.8
Initial results from the ELEVATE-TN Phase III trial were presented in December 2019 at the American Society of Hematology Annual Meeting and Exhibition. The trial met its primary endpoint (IRC-assessed PFS with Calquence plus obinutuzumab versus chlorambucil plus obinutuzumab) at the data cut-off for the interim analysis after a median follow-up of 28.3 months. The findings, along with previously reported data from the ASCEND Phase III trial in relapsed or refractory CLL, supported the approvals of Calquence by the US FDA and the Australian Therapeutic Goods Administration for the treatment of adult patients with CLL or SLL and by the European Medicines Agency (EMA) and Health Canada for CLL.
Calquence
Calquence (acalabrutinib) is a next-generation, selective inhibitor of BTK. Calquence binds covalently to BTK, thereby inhibiting its activity.9 In B cells, BTK signalling results in activation of pathways necessary for B-cell proliferation, trafficking, chemotaxis and adhesion.
Calquence is approved for the treatment of CLL and SLL in the US, approved for CLL in the EU and many other countries worldwide and approved in Japan for relapsed or refractory CLL and SLL.
In the US and several other countries, Calquence is also approved for the treatment of adult patients with MCL who have received at least one prior therapy. The US MCL 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. Calquence is not currently approved for the treatment of MCL in Europe or Japan.
As part of an extensive clinical development programme, AstraZeneca and Acerta Pharma are currently evaluating Calquence in more than 20 company-sponsored clinical trials. Calquence is being evaluated for the treatment of multiple B-cell blood cancers, including CLL, MCL, diffuse large B-cell lymphoma, Waldenström’s macroglobulinaemia, marginal zone lymphoma and other haematologic malignancies.
https://www.calquencehcp.com/mcl.html
AstraZeneca in haematology
AstraZeneca is pushing the boundaries of science to redefine care in haematology. We have expanded our commitment to patients with haematologic conditions, not only in oncology but also in rare diseases with the acquisition of Alexion, allowing us to reach more patients with high unmet needs. By applying our deep understanding of blood cancers, leveraging our strength in solid tumour oncology and delivering on Alexion’s pioneering legacy in complement science to provide innovative medicines for rare diseases, we are pursuing the end-to-end development of novel therapies designed to target underlying drivers of disease.
By targeting haematologic conditions with high unmet medical needs, we aim to deliver innovative medicines and approaches to improve patient outcomes. Our goal is to help transform the lives of patients living with malignant, rare and other related haematologic diseases, shaped by insights from patients, caregivers and physicians to have the most meaningful impact.
AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.
The Company’s focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.
AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
Japan approves AstraZeneca's blood cancer drug Calquence
Dec. 28, 2022 4:57 AM ET
By: Ravikash, SA News Editor
- The Japanese Ministry of Health, Labour and Welfare (MHLW) approved AstraZeneca's (NASDAQ:AZN) Calquence (acalabrutinib) to treat adult patients with treatment-naïve chronic lymphocytic leukemia (CLL), including small lymphocytic lymphoma (SLL).
- https://seekingalpha.com/news/3920862-japan-approves-astrazenecas-blood-cancer-drug-calquence
- AstraZeneca PLC (AZN)
- https://www.calquence.com/
- https://www.calquencehcp.com/mcl.html
- https://www.astrazeneca.com/
Imfinzi (durvalumab) plus chemotherapy
Imfinzi plus chemotherapy approved in the EU as first immunotherapy regimen for patients with advanced biliary tract cancer
PUBLISHED21 December 2022 21 December 2022 07:00 GMT
Approval based on TOPAZ-1 updated survival results showing Imfinzi
combination reduced risk of death by 24% vs. chemotherapy alone
AstraZeneca’s Imfinzi (durvalumab) has been approved in the European Union (EU) for the 1st-line treatment of adult patients with unresectable or metastatic biliary tract cancer (BTC) in combination with chemotherapy (gemcitabine plus cisplatin).
The approval by the European Commission was based on the primary results from the TOPAZ-1 Phase III trial published in the New England Journal of Medicine Evidence, and on the updated results presented at the European Society for Medical Oncology Congress 2022. The approval follows the recommendation by The Committee for Medicinal Products for Human Use of the European Medicines Agency in November 2022.
At the interim analysis, Imfinzi plus chemotherapy reduced the risk of death by 20% versus chemotherapy alone (based on a hazard ratio [HR] of 0.80; 95% confidence interval [CI] 0.66-0.97; p=0.021). Updated results from TOPAZ-1 after an additional 6.5 months of follow-up showed a 24% reduction in the risk of death versus chemotherapy alone (HR 0.76; 95% CI, 0.64-0.91), with more than two times as many patients treated with Imfinzi plus chemotherapy estimated to be alive at two years versus chemotherapy alone (23.6% versus 11.5%). Updated median overall survival (OS) was 12.9 months versus 11.3 with chemotherapy.
BTC is a group of rare and aggressive cancers that occur in the bile ducts (cholangiocarcinoma) and gallbladder.1,2 There are approximately 211,000 new patients diagnosed with gallbladder and biliary tract cancer each year, and about 40,000 of these occur across Europe.3 These patients have a poor prognosis, with approximately 5% to 15% of patients with BTC surviving five years.4
Juan W. Valle, MD, Professor of Medical Oncology at the University of Manchester and The Christie NHS Foundation Trust, UK, and a lead investigator in the TOPAZ-1 Phase III trial, said: “Today’s approval marks an important shift in the treatment of this aggressive and often overlooked disease and a significant improvement compared to standard of care for these patients. After waiting over a decade for new therapeutic options, biliary tract cancer patients in the EU will now have the opportunity to benefit from an immunotherapy-based treatment for the first time.”
Dave Fredrickson, Executive Vice President, Oncology Business Unit, AstraZeneca, said: “With this approval, Imfinzi plus chemotherapy becomes the only immunotherapy-based treatment option available to patients in the EU with advanced biliary tract cancer. This approval underscores our commitment to transform survival outcomes while addressing the high unmet need for new and improved treatments for patients with hepatobiliary cancers.”
Imfinzi plus chemotherapy was generally well tolerated, with no new safety signals observed, and did not increase the discontinuation rate due to adverse events (AEs) compared to chemotherapy alone. Grade 3 or 4 treatment-related AEs were experienced by 60.9% of patients treated with Imfinzi plus chemotherapy, and by 63.5% of patients treated with chemotherapy alone.
Imfinzi plus chemotherapy is approved in the US and other countries for the treatment of adults with locally advanced or metastatic BTC. Regulatory applications are also currently under review in Japan and several other countries based on the TOPAZ-1 results.
TOPAZ-1
TOPAZ-1 was a randomised, double-blind, placebo controlled, multicentre, global Phase III trial of Imfinzi in combination with chemotherapy (gemcitabine plus cisplatin) versus placebo in combination with chemotherapy as a 1st-line treatment in 685 patients with unresectable advanced or metastatic BTC including intrahepatic and extrahepatic cholangiocarcinoma, and gallbladder cancer. Patients with ampullary carcinoma were excluded.
The primary endpoint was overall survival and key secondary endpoints included progression-free survival, objective response rate and safety. The trial was conducted in 105 centres across 17 countries including in the US, Europe, South America and several countries in Asia including South Korea, Thailand, Japan and China.
Imfinzi
Imfinzi (durvalumab) is a human monoclonal antibody that binds to the PD-L1 protein and blocks the interaction of PD-L1 with the PD-1 and CD80 proteins, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.
Imfinzi is the only approved immunotherapy in unresectable or metastatic BTC, unresectable hepatocellular carcinoma [in combination with Imjudo (tremelimumab)], and the curative-intent setting of unresectable, Stage III non-small cell lung cancer (NSCLC) in patients whose disease has not progressed after chemoradiotherapy. It is the global standard of care in this setting based on the PACIFIC Phase III trial.
Imfinzi is also approved in the US, EU, Japan, China and many other countries around the world for the treatment of extensive-stage small cell lung cancer (ES-SCLC) based on the CASPIAN Phase III trial. In 2021, updated results from the CASPIAN trial showed Imfinzi plus chemotherapy tripled patient survival at three years versus chemotherapy alone.
Imfinzi is also approved in the US for the treatment of adult patients with Stage IV (metastatic) NSCLC in combination with Imjudo and chemotherapy.
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 SCLC, NSCLC, bladder cancer, several GI cancers, ovarian cancer, endometrial cancer, and other solid tumours.
AstraZeneca in GI cancers
AstraZeneca has a broad development programme for the treatment of GI cancers across several medicines and a variety of tumour types and stages of disease. In 2020, GI cancers collectively represented approximately 5.1 million new cancer cases leading to approximately 3.6 million deaths.7
Within this programme, the Company is committed to improving outcomes in gastric, liver, biliary tract, oesophageal, pancreatic, and colorectal cancers.
Imfinzi is approved in the US in combination with chemotherapy (gemcitabine plus cisplatin) for advanced BTC and in combination with Imjudo in unresectable hepatocellular carcinoma. Imfinzi is being assessed in combinations, including with Imjudo in liver, oesophageal and gastric cancers in an extensive development programme spanning early to late-stage disease across settings.
Enhertu (trastuzumab deruxtecan), a HER2-directed antibody drug conjugate, is approved in HER2-positive advanced gastric cancer and is being assessed in colorectal cancer. Enhertu is jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.
Lynparza (olaparib), a first-in-class PARP inhibitor, is approved in BRCA-mutated metastatic pancreatic cancer. Lynparza is developed and commercialised in collaboration with MSD (Merck & Co., Inc. inside the US and Canada).
AstraZeneca in immuno-oncology (IO)
AstraZeneca is a pioneer in introducing the concept of immunotherapy into dedicated clinical areas of high unmet medical need. The Company has a comprehensive and diverse IO portfolio and pipeline anchored in immunotherapies designed to overcome evasion of the anti-tumour immune response and stimulate the body’s immune system to attack tumours.
AstraZeneca aims to reimagine cancer care and help transform outcomes for patients with Imfinzi as a single treatment and in combination with Imjudo as well as other novel immunotherapies and modalities. The Company is also exploring next-generation immunotherapies like bispecific antibodies and therapeutics that harness different aspects of immunity to target cancer.
AstraZeneca is boldly pursuing an innovative clinical strategy to bring IO-based therapies that deliver long-term survival to new settings across a wide range of cancer types. With an extensive clinical programme, the Company also champions the use of IO treatment in earlier disease stages, where there is the greatest potential for cure.
AstraZeneca in oncology
AstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.
The Company's focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.
AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca Imfinzi combo for biliary tract cancer gets approval in EU
Dec. 21, 2022 4:50 AM ET
By: Ravikash, SA News Editor
- The European Commission (EC) approved AstraZeneca's (NASDAQ:AZN) Imfinzi in combination with chemotherapies gemcitabine and cisplatin, as a first‑line treatment for adults with unresectable or metastatic biliary tract cancer (BTC).
- https://seekingalpha.com/news/3919300-astrazenecas-imfinzi-combo-for-biliary-tract-cancer-gets-approval-in-eu
- AstraZeneca PLC (AZN)
- https://www.imfinzi.com/
- https://www.astrazeneca.com/
YESCARTA® (axicabtagene ciloleucel)
December 22, 2022
Yescarta® Now Approved in Japan for Initial Treatment of Relapsed/Refractory Large B-Cell Lymphoma
– Based on Landmark ZUMA-7 Study, Patients with LBCL Treated with Yescarta in Second-Line Achieved Four-Fold Greater Improvement in Event-Free Survival of 8.3 Months vs. Two Months for Standard of Care (SOC)
– In ZUMA-7, Patients Treated with Yescarta Were 2.5 Times More Likely than SOC to be Alive at Two Years Without Disease Progression or Need for Additional Treatments –
SANTA MONICA, Calif. & TOKYO--(BUSINESS WIRE)-- Kite Pharma, Inc., a Gilead Company, (hereafter, Kite) (NASDAQ: GILD) and Daiichi Sankyo Co., Ltd. (hereafter, Daiichi Sankyo) (TSE: 4568) today jointly announced that the Japan Ministry of Health, Labour and Welfare (MHLW) has approved Yescarta (axicabtagene ciloleucel), a chimeric antigen receptor (CAR) T-cell therapy, for the initial treatment of patients with relapsed/refractory large B-cell lymphoma ( R/R LBCL): diffuse large B-cell lymphoma, primary mediastinal large B-cell lymphoma, transformed follicular lymphoma, and high-grade B-cell lymphoma. Yescarta should be used only in patients who have not received prior transfusion of CAR T-cells targeted at CD19 antigen.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20221222005384/en/
The Standard of Care (SOC) to treat LBCL patients has historically been a multi-step process that starts with chemoimmunotherapy, followed by high-dose chemotherapy (HDT) and then ends with a stem cell transplant (ASCT). Although approximately 60% of newly diagnosed LBCL patients will respond to the initial treatment with chemotherapy, 40% will relapse or will not respond and need second-line treatment. Yescarta is now approved for the initial treatment of R/R LBCL patients in Japan.
“We are very proud of this additional Yescarta approval in Japan,” said Christi Shaw, CEO of Kite. “As Japan has the second-largest number of people diagnosed with non-Hodgkin lymphoma globally, this approval marks an important step in bringing this innovative therapy earlier to more patients.”
“We are glad to have achieved this important milestone in order to provide an innovative treatment option to more patients with large B-cell lymphoma in Japan,” said Wataru Takasaki, PhD, Executive Officer, Head of R&D Division in Japan, Daiichi Sankyo. “It also further demonstrates the expertise of our Daiichi Sankyo R&D organization as we had successfully expanded the indication for this CAR-T modality.”
The approval of Yescarta for the initial treatment of R/R LBCL patients in Japan is based on clinical data, including the results of the global pivotal trial conducted by Kite (ZUMA-7). ZUMA-7 is the largest and longest trial of a CAR T-cell therapy versus SOC in this patient population. Results from the study demonstrated that at a median follow-up of two years, Yescarta-treated patients had a four-fold greater improvement in the primary endpoint of event-free survival (EFS; hazard ratio 0.40; 95% CI: 0.31-0.51, P<0.0001) over the current SOC (8.3 months vs 2.0 months). Additionally, Yescarta demonstrated a 2.5 fold increase in patients who were alive at two years without disease progression or need for additional cancer treatment vs SOC (41% v 16%).
This indication was first approved by the U.S. FDA in April 2022 followed by the European Commission in October 2022.
On December 7, 2022, Daiichi Sankyo and Kite jointly announced that the Marketing Authorization for Yescarta will be transferred to Gilead Sciences K.K., the Japan subsidiary of Gilead Sciences, Inc., in 2023. The Kite Cell Therapy Business Unit at Gilead Sciences K.K. will manage the sales and promotion activities of the product in Japan after the Marketing Authorization transfer.
Kite’s manufacturing facility in El Segundo, California, U.S., has been approved by Japanese regulatory authorities to commence manufacturing Yescarta for the Japan market in 2023.
About Zuma-7
ZUMA-7 is an ongoing, randomized, open-label, global, multicenter (US, Australia, Canada, Europe, Israel) Phase 3 study of 359 patients at 77 centers, evaluating the safety and efficacy of a single-infusion of Yescarta versus current SOC for second-line therapy (platinum-based salvage combination chemotherapy regimen followed by high-dose chemotherapy and autologous stem cell transplant in those who respond to salvage chemotherapy) in adult patients with relapsed or refractory LBCL within 12 months of first-line therapy. The primary endpoint is event free survival (EFS). Key secondary endpoints include objective response rate (ORR) and overall survival (OS). Additional secondary endpoints include patient reported outcomes and safety.
About YESCARTA®
Yescarta (axicabtagene ciloleucel) is a CAR T-cell therapy directed against CD19 (a cell membrane protein), which harnesses a patient’s own immune system to fight cancer. Axicabtagene ciloleucel is made by removing a patient’s T cells from their blood and engineering them in the lab to express chimeric antigen receptors so that they can recognize and destroy cancer cells when they are infused back to the patient’s body. The CAR T-cell therapy is manufactured specifically for each patient and administered only once. Axicabtagene ciloleucel received Orphan Drug Designation from the Japan MHLW in 2018 for the treatment of diffuse large B-cell lymphoma, primary mediastinal B-cell lymphoma, transformed follicular lymphoma and high-grade B-cell lymphoma. Yescarta was approved in Japan for the treatment of patients with relapsed or refractory large B-cell lymphomas, a type of non-Hodgkin lymphoma, in January 2021.
https://www.yescarta.com/lbcl/
Please see full U.S. Prescribing Information, including BOXED WARNING and Medication Guide.
Yescarta® is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:
- Adult patients with large B-cell lymphoma that is refractory to first-line chemoimmunotherapy or that relapses within 12 months of first-line chemoimmunotherapy. (1.1)
- Adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma.
Limitations of Use: Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma. (1.1)
- Adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy. This indication is approved under accelerated approval based on response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s). (1.2)
For the full European Prescribing Information, please visit: : https://www.ema.europa.eu/en/medicines/human/EPAR/yescarta.
About Daiichi Sankyo
Daiichi Sankyo is dedicated to creating new modalities and innovative medicines by leveraging our world-class science and technology for our purpose “to contribute to the enrichment of quality of life around the world.” In addition to our current portfolio of medicines for cancer and cardiovascular disease, Daiichi Sankyo is primarily focused on developing novel therapies for people with cancer as well as other diseases with high unmet medical needs. With more than 100 years of scientific expertise and a presence in more than 20 countries, Daiichi Sankyo and its 16,000 employees around the world draw upon a rich legacy of innovation to realize our 2030 Vision to become an “Innovative Global Healthcare Company Contributing to the Sustainable Development of Society.” For more information, please visit: www.daiichisankyo.com.
About Kite
Kite, a Gilead Company, is a global biopharmaceutical company based in Santa Monica, California, focused on cell therapy to treat and potentially cure cancer. As the global cell therapy leader, Kite has treated more patients with CAR T-cell therapy than any other company. Kite has the largest in-house cell therapy manufacturing network in the world, spanning process development, vector manufacturing, clinical trial production and commercial product manufacturing. For more information on Kite, please visit www.kitepharma.com.
About Gilead Sciences
Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California. Gilead Sciences acquired Kite in 2017.
U.S. Prescribing Information for Yescarta including BOXED WARNING, is available at www.kitepharma.com and www.gilead.com .
Kite, the Kite logo, Yescarta, and GILEAD are trademarks of Gilead Sciences, Inc. or its related companies.
View source version on businesswire.com: https://www.businesswire.com/news/home/20221222005384/en/
Source: Gilead Sciences, Inc.
Gilead's Yescarta approved in Japan for initial treatment of B-cell lymphomas
Dec. 22, 2022 4:59 PM ET
Gilead Sciences, Inc. (GILD), DSKYF, DSNKY
By: Anuron Mitra, SA News Editor
- Gilead Sciences' (NASDAQ:GILD) Kite Pharma and Japanese pharmaceutical giant Daiichi Sankyo (OTCPK:DSKYF) (OTCPK:DSNKY) on Thursday said that cancer drug Yescarta had received approval from the country's health ministry for initial treatment of various types of B-cell lymphoma.
- https://seekingalpha.com/news/3920108-gileads-yescarta-approved-in-japan-for-initial-treatment-of-b-cell-lymphomas
- Gilead Sciences, Inc. (GILD), DSKYF, DSNKY
- https://www.yescarta.com/lbcl/
- https://www.gilead.com/
LIBTAYO® (CEMIPLIMAB)
December 23, 2022 at 8:30 AM EST Back
LIBTAYO® (CEMIPLIMAB) APPROVED IN JAPAN FOR ADVANCED OR RECURRENT CERVICAL CANCER
TARRYTOWN, N.Y., Dec. 23, 2022 /PRNewswire/ -- Regeneron Pharmaceuticals (NASDAQ: REGN) today announced that the Ministry of Health, Labor and Welfare (MHLW) in Japan has granted marketing and manufacturing authorization for Libtayo® (cemiplimab) as monotherapy to treat patients with advanced or recurrent cervical cancer whose disease progressed after chemotherapy.
"In recent years, the incidence of cervical cancer has increased in Japan, with the prognosis for advanced stage disease remaining poor and treatment options limited," said Israel Lowy, M.D., Ph.D., Senior Vice President, Translational and Clinical Sciences, Oncology at Regeneron. "With this approval, Libtayo becomes the first single-agent immunotherapy approved in Japan for the treatment of advanced cervical cancer."
The MHLW approval is based on positive data from the international, multicenter Phase 3 EMPOWER-Cervical 1 trial, conducted in collaboration with NRG Oncology-Japan, the GOG Foundation, Inc., and the European Network for Gynaecological Oncological Trial (ENGOT) groups. The trial evaluated Libtayo compared to an investigator's choice of chemotherapy and enrolled 608 patients across 14 countries, including Japan, irrespective of PD-L1 expression status or histology. In March 2021, the trial was stopped early based on the highly significant effect of Libtayo on overall survival among squamous cell carcinoma patients following a unanimous recommendation by the Independent Data Monitoring Committee.
"With Libtayo, Japan now has an approved treatment option that has demonstrated significant survival benefits in this cervical cancer patient population compared to chemotherapy irrespective of PD-L1 expression – a milestone no other PD-1 inhibitor has achieved in a Phase 3 trial," said Prof. Kosei Hasegawa, Gynecologic Oncology, Saitama Medical University International medical Center, a trial investigator and a member of NRG Oncology-Japan. "We are proud to have contributed to bringing this new treatment option to women living with this difficult-to-treat cancer in Japan.
Cervical cancer is the fourth leading cause of cancer death in women worldwide and is most frequently diagnosed in women between the ages of 35 and 44. Almost all cases of cervical cancer are caused by human papillomavirus (HPV) infection. It is estimated that approximately 600,000 new cases of cervical cancer are diagnosed and 350,000 deaths from cervical cancer occur worldwide each year.
In November 2022, Libtayo was approved by the European Commission for the treatment of adult patients with recurrent or metastatic cervical cancer and disease progression on or after platinum-based chemotherapy.
About Libtayo
Libtayo is a fully human monoclonal antibody targeting the immune checkpoint receptor PD-1 on T cells and was invented using Regeneron's proprietary VelocImmune® technology. By binding to PD-1, Libtayo has been shown to block cancer cells from using the PD-1 pathway to suppress T-cell activation. In the U.S. and other countries Libtayo is indicated in certain patients with advanced basal cell carcinoma (BCC), advanced CSCC and advanced non-small cell lung cancer (NSCLC), as well as in advanced cervical cancer in Japan, the European Union, Canada and Brazil.
Libtayo was jointly developed by Sanofi and Regeneron under a global collaboration agreement. As of July 1, 2022, Regeneron is responsible for the development and marketing of Libtayo globally. In Japan, Libtayo is currently marketed by Sanofi on Regeneron's behalf over the course of a defined transition period.
The extensive clinical program for Libtayo is focused on difficult-to-treat cancers. Libtayo is currently being investigated in trials as a monotherapy, as well as in combination with either conventional or novel therapeutic approaches for other solid tumors and blood cancers. These potential uses are investigational, and their safety and efficacy have not been evaluated by any regulatory authority.
U.S. FDA-approved Indications
Libtayo is a prescription medicine used to treat:
-- People with a type of skin cancer called CSCC that has spread or cannot be cured by surgery or radiation.
-- People with a type of skin cancer called BCC:
- That cannot be removed by surgery (locally advanced BCC) and have received treatment with a hedgehog pathway inhibitor (HHI), or cannot receive treatment with an HHI.
- That has spread (metastatic BCC) and have received treatment with an HHI, or cannot receive treatment with an HHI. This use is approved based on how many patients responded to treatment and how long they responded. Studies are ongoing to provide additional information about clinical benefit.
-- Adults with a type of lung cancer called NSCLC:
- Libtayo may be used in combination with chemotherapy that contains a platinum medicine as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, OR your lung cancer has spread to other areas of your body (metastatic lung cancer), AND your tumor does not have an abnormal "EGFR", "ALK" or "ROS1" gene.
- Libtayo may be used alone as your first treatment when your lung cancer has not spread outside your chest (locally advanced lung cancer) and you cannot have surgery or chemotherapy with radiation, OR your lung cancer has spread to other areas of your body (metastatic lung cancer), AND your tumor tests positive for high "PD-L1", AND your tumor does not have an abnormal "EGFR", "ALK "or "ROS1" gene.
It is not known if Libtayo is safe and effective in children.
Please see full Prescribing Information, including Medication Guide.
About Regeneron in Oncology
At Regeneron, we're applying more than three decades of scientific innovation with the goal of developing paradigm-changing therapies for patients with cancer. Our oncology portfolio is built around two foundational approaches – our approved PD-1 inhibitor Libtayo and investigational bispecific antibodies – which are being evaluated both as monotherapies and in combination with emerging therapeutic modalities. Together, they provide us with unique combinatorial flexibility to develop potentially synergistic treatments for a wide range of solid tumors and blood cancers.
If you are interested in learning more about our clinical trials, please contact us (clinicaltrials@regeneron.com or 844-734-6643) or visit our clinical trials website.
About Regeneron's VelocImmune Technology
Regeneron's VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron's co-Founder, President and Chief Scientific Officer George D. Yancopoulos was a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create approximately one in five of all original, FDA-approved or authorized fully human monoclonal antibodies. This includes REGEN-COV® (casirivimab and imdevimab), Dupixent® (dupilumab), Libtayo, Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb) and Inmazeb™ (atoltivimab, maftivimab and odesivimab-ebgn).
About Regeneron
Regeneron is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led for nearly 35 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to nine FDA-approved treatments and numerous product candidates in development, almost all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, pain, hematologic conditions, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug development process through our proprietary VelociSuite technologies, such as VelocImmune, which uses unique genetically humanized mice to produce optimized fully human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center®, which is conducting one of the largest genetics sequencing efforts in the world.
For more information, please visit www.Regeneron.com or follow @Regeneron on Twitter.
View original content:https://www.prnewswire.com/news-releases/libtayo-cemiplimab-approved-in-japan-for-advanced-or-recurrent-cervical-cancer-301709636.html
SOURCE Regeneron Pharmaceuticals, Inc.
Regeneron's Libtayo approved in Japan for cervical cancer
Dec. 23, 2022 9:01 AM ET
Regeneron Pharmaceuticals, Inc. (REGN)
By: Jonathan Block, SA News Editor
- Japanese regulators have granted marketing authorization to Regeneron's (NASDAQ:REGN) Libtayo (cemiplimab) for advanced or recurrent cervical cancer.
- https://seekingalpha.com/news/3920272-regeneron-libtayo-approved-in-japan-for-cervical-cancer
- Regeneron Pharmaceuticals, Inc. (REGN)
- https://www.libtayo.com/
- https://www.regeneron.com/
Sunlenca® (Lenacapavir)
Sunlenca® (Lenacapavir) Receives FDA Approval As A First-In-Class, Twice-Yearly Treatment Option For People Living With Multi-Drug Resistant HIV
12/22/2022
– Sunlenca is the First and Only Approved Capsid Inhibitor-Based HIV Treatment Option –
– New Drug Application Approval Based on High Rates of Sustained Virologic Suppression in the CAPELLA Trial –
FOSTER CITY, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) today announced that Sunlenca® (lenacapavir), in combination with other antiretroviral(s) (ARV), has been granted approval by the U.S. Food and Drug Administration (FDA) for the treatment of HIV-1 infection in heavily treatment-experienced (HTE) adults with multi-drug resistant (MDR) HIV-1 infection. Sunlenca has a multi-stage mechanism of action distinguishable from other currently approved classes of antiviral agents and no known cross resistance exhibited in vitro to other existing drug classes. Sunlenca offers a new, twice-yearly treatment option for adults with HIV that is not adequately controlled by their current treatment regimen.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20221221005541/en/
Sunlenca Packaging (Photo: Business Wire)
“An effective antiretroviral regimen can be devised for most people living with the virus; however, some people living with HIV no longer have durable viral suppression due to resistance to multiple classes of antiretroviral therapies,” said Sorana Segal-Maurer, MD, Director of the Dr. James J. Rahal Jr. Division of Infectious Diseases at NewYork-Presbyterian Queens, Professor of Clinical Medicine at Weill Cornell Medicine and the Site Principal Investigator for the CAPELLA trial. “The availability of new classes of antiretroviral drugs is critical for heavily treatment-experienced people with multi-drug resistant HIV. Following today’s decision from the FDA, lenacapavir helps to fill a critical unmet need for people with complex prior treatment histories and offers physicians a long-awaited twice-yearly option for these patients who otherwise have limited therapy choices."
Despite the significant advances in ARV therapy, there remain numerous critical and pressing unmet needs for people living with HIV. This is particularly true for individuals who are heavily treatment experienced – which account for an estimated 2% of adults living with HIV who are on treatment globally –- and are unable to maintain virologic suppression due to resistance, intolerance or safety considerations. This type of complexity further increases the chance of treatment failure, underscoring the need for new treatment options that are active against resistant variants of the virus with a novel mechanism of action.
Lenacapavir is a breakthrough innovation with the potential to be a preferred and versatile foundational long-acting agent due to its therapeutic potency and a range of dosing frequencies and routes of administration. Lenacapavir is being developed as a foundation for Gilead’s future HIV therapies with the goal of offering several long-acting options that help address individual needs and preferences that may help optimize outcomes and reduce burden of care. Lenacapavir is being studied in multiple ongoing early and late-stage development programs and has the potential to offer a diverse set of person-centric options for treatment and prevention that could uniquely fit into the lives of people living with HIV and people who would benefit from pre-exposure prophylaxis (PrEP). The use of lenacapavir for HIV prevention is investigational and the safety and efficacy of lenacapavir for this use have not been established.
“This news is an important milestone in the work to help end the HIV epidemic as Sunlenca is now the only FDA-approved twice-yearly treatment for people with multi-drug resistant HIV,” said Daniel O’Day, Chairman and Chief Executive Officer, Gilead Sciences. “Gilead scientists have developed a unique and potent antiretroviral medicine in Sunlenca with the potential for flexible dosing options. Our goal is to deliver multiple long-acting options for treatment and prevention that are tailored to the needs of people living with HIV and people who could benefit from PrEP medicines.”
The FDA approval for Sunlenca is supported by data from the Phase 2/3 CAPELLA trial, which evaluated lenacapavir in combination with an optimized background regimen in people with multi-drug resistant HIV-1 who are heavily treatment experienced. CAPELLA participants had undergone previous treatment with a median of nine antiretroviral medications. In this patient population with significant unmet medical need, 83% (n=30/36) of participants randomly allocated to receive lenacapavir in addition to an optimized background regimen achieved an undetectable viral load (<50 copies/mL) at Week 52. Additionally, these participants achieved a mean increase in CD4 count of 82 cells/µL. These data were presented at the 29th Conference on Retroviruses and Opportunistic Infections (virtual CROI 2022).
Sunlenca was reviewed and approved as a medication with FDA Breakthrough Therapy Designation, which is intended to expedite the development and review of new drugs which may demonstrate substantial improvement over available therapy. In May 2019, the FDA granted Breakthrough Therapy Designation for the development of lenacapavir for the treatment of HIV-1 infection in heavily treatment-experienced patients with multi-drug resistance in combination with other antiretroviral drugs.
Lenacapavir, alone or in combination, is not approved by any regulatory authority outside of the United States, United Kingdom, Canada or the European Union for any use. The European Marketing Authorization applies to all 27 member states of the European Union, as well as Norway, Iceland and Liechtenstein.
Additional regulatory filings and decisions by regulatory authorities are anticipated to continue in 2023.
The use of lenacapavir for HIV prevention is investigational and the safety and efficacy of lenacapavir for this use have not been established. Gilead is studying the safety and efficacy of lenacapavir for HIV prevention in multiple ongoing clinical studies.
Please see below for the U.S. Indication and Important Safety Information for Sunlenca.
There is no cure for HIV or AIDS.
Sorana Segal-Maurer, MD, is a paid consultant for Gilead Sciences, Inc.
About CAPELLA (NCT04150068)
CAPELLA is a Phase 2/3, double-blinded, placebo-controlled global multicenter study designed to evaluate the antiviral activity of lenacapavir administered every six months as a subcutaneous injection in heavily treatment-experienced people with multi-drug resistant HIV-1 infection. CAPELLA includes men and women with HIV-1 and is being conducted at research centers in North America, Europe and Asia.
In CAPELLA, 36 participants with multi-class HIV-1 drug resistance and a detectable viral load while on a failing regimen were randomly allocated to receive oral lenacapavir or placebo in a 2:1 ratio for 14 days, in addition to continuing their failing regimen (functional monotherapy). An additional 36 participants were enrolled in a separate treatment cohort. Both cohorts are part of the ongoing maintenance period of the study evaluating the safety and efficacy of subcutaneous lenacapavir administered every six months in combination with an optimized background regimen. The primary endpoint was the proportion of participants randomly allocated to receive lenacapavir or placebo for 14 days, in addition to continuing their failing regimen, achieving ≥0.5 log10 copies/mL reduction from baseline in HIV-1 RNA at the end of the functional monotherapy period. The study found that 88% of participants receiving lenacapavir (n=21/24) experienced at least a 0.5 log10 reduction in HIV-1 viral load by the end of 14 days of functional monotherapy as compared with 17% of those receiving placebo (n=2/12).
Following the 14-day functional monotherapy period, participants randomly allocated to receive lenacapavir or placebo, in addition to continuing their failing regimen, started open-label lenacapavir and an optimized background regimen, while those enrolled in a separate treatment cohort received open-label lenacapavir and an optimized background regimen on Day 1. This ongoing maintenance period is evaluating the additional study endpoints of safety and efficacy of subcutaneous lenacapavir administered every six months in combination with an optimized background regimen.
The New England Journal of Medicine published the primary outcome results of the CAPELLA study in its May 11, 2022 issue - Capsid Inhibition with Lenacapavir in Multidrug-Resistant HIV-1 Infection.
For further information, please see https://clinicaltrials.gov/ct2/show/NCT04150068.
About Sunlenca ®
Sunlenca (300 mg tablet and 463.5 mg/1.5 mL injection) is a first-in-class, long-acting HIV capsid inhibitor approved in the United States, the United Kingdom, Canada and the European Union, for the treatment of HIV infection, in combination with other antiretroviral(s), in people with multi-drug resistant HIV who are heavily treatment-experienced. Sunlenca tablets are approved for oral loading during initiation of Sunlenca treatment, prior to or at the time of the first long-acting lenacapavir injection depending on initiation option. The multi-stage mechanism of action of Sunlenca is distinguishable from other currently approved classes of antiviral agents and is designed to provide a new avenue for the development of a long-acting treatment option for individuals with multi-drug resistant HIV whose virus no longer effectively responds to therapy. While most antivirals act on just one stage of viral replication, Sunlenca is designed to inhibit HIV at multiple stages of its lifecycle and has no known cross resistance exhibited in vitro to other existing drug classes. Sunlenca is the only HIV treatment option administered twice-yearly.
U.S. Indication for Sunlenca
Sunlenca, a human immunodeficiency virus type 1 (HIV-1) capsid inhibitor, in combination with other antiretroviral(s), is indicated for the treatment of HIV-1 infection in heavily treatment-experienced adults with multidrug resistant HIV-1 infection failing their current antiretroviral regimen due to resistance, intolerance, or safety considerations.
About Gilead Sciences
Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer.
For 35 years, Gilead has been a leading innovator in the field of HIV, driving advances in treatment, prevention and cure research. Gilead researchers have developed 12 HIV medications, including the first single-tablet regimen to treat HIV, the first antiretroviral for pre-exposure prophylaxis (PrEP) to reduce the risk of acquiring HIV infection, and the first, long-acting injectable HIV treatment medication administered twice-yearly. Our advances in medical research have helped to transform HIV into a preventable, chronic condition for millions of people.
Gilead is committed to continued scientific innovation to provide solutions for the evolving needs of people affected by HIV around the world. Through partnerships and collaborations, the company also aims to improve education, expand access and address barriers to care, with the goal of ending the HIV epidemic for everyone, everywhere. Gilead was recognized as the number one philanthropic funder of HIV-related programs in a report released by Funders Concerned About AIDS.
Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.
U.S. Prescribing Information for Sunlenca is available at www.gilead.com .
Sunlenca, Gilead and the Gilead logo are registered trademarks of Gilead Sciences, Inc., or its related companies.
For more information about Gilead, please visit the company’s website at www.gilead.com , follow Gilead on Twitter ( @Gilead Sciences ) or call Gilead Public Affairs at 1-800-GILEAD-5 or 1-650-574-3000.
View source version on businesswire.com: https://www.businesswire.com/news/home/20221221005541/en/
Source: Gilead Sciences, Inc.
Gilead wins U.S. approval for twice yearly HIV med lenacapavir
Dec. 22, 2022 11:05 AM ET
By: Dulan Lokuwithana, SA News Editor
- Gilead Sciences (NASDAQ:GILD) announced Thursday that the FDA approved its HIV capsid inhibitor, Sunlenca, in combination with other antiretrovirals for the treatment of adults with multi-drug resistant (MDR) HIV-1 infection.
- https://seekingalpha.com/news/3919859-gilead-wins-fda-approval-twice-yearly-hiv-drug
- Gilead Sciences, Inc. (GILD)
- https://www.gilead.com/news-and-press/press-room/press-releases/2022/12/sunlenca-lenacapavir-receives-fda-approval-as-a-firstinclass-twiceyearly-treatment-option-for-people-living-with-multidrug-resistant-hiv
LYNPARZA® (olaparib) in Combination With Abiraterone and Prednisone or Prednisolone
LYNPARZA® (olaparib) in Combination With Abiraterone and Prednisone or Prednisolone Approved in the EU as Treatment for Certain Patients With Metastatic Castration-Resistant Prostate Cancer
December 21, 2022 6:45 am ET
First PARP inhibitor and new hormonal agent combination approved for these patients in Europe
RAHWAY, N.J.--(BUSINESS WIRE)-- AstraZeneca and Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced that LYNPARZA has been approved in the European Union (EU) in combination with abiraterone and prednisone or prednisolone for the treatment of adult patients with metastatic castration-resistant prostate cancer (mCRPC) in whom chemotherapy is not clinically indicated.
This approval by the European Commission follows the positive recommendation from the Committee for Medicinal Products for Human Use received in November this year and was based on the Phase 3 PROpel trial, results of which were published in NEJM Evidence in June 2022.
In PROpel, LYNPARZA in combination with abiraterone and prednisone or prednisolone reduced the risk of disease progression or death by 34% (HR=0.66 [95% CI, 0.54-0.81]; p<0.0001) versus placebo plus abiraterone and prednisone or prednisolone, based on investigator assessment. Median radiographic progression-free survival (rPFS) was 24.8 months for the LYNPARZA plus abiraterone arm (95% CI, 20.5, 27.6) versus 16.6 months for the placebo plus abiraterone arm (95% CI, 13.9, 19.2). A planned sensitivity analysis by blinded independent central review was consistent with the investigator-based analysis, with a median rPFS of 27.6 months for the LYNPARZA plus abiraterone arm compared to 16.4 months for the placebo plus abiraterone arm.
Interim results for the key secondary efficacy endpoint of overall survival (OS) did not reach statistical significance, with an event rate of 37.1% in the LYNPARZA plus abiraterone arm versus 43.1% in the placebo plus abiraterone arm at the time of the analysis (HR=0.83 [95% CI, 0.66-1.03]).
The safety and tolerability of LYNPARZA in combination with abiraterone and prednisone or prednisolone was generally consistent with that of the individual medicines. Approximately 16% of patients who received LYNPARZA in combination with abiraterone and prednisone or prednisolone discontinued treatment due to an adverse event (AE). As previously reported, based on an analysis from the PROpel trial presented earlier this year at the American Society of Clinical Oncology Genitourinary Cancers Symposium, the most common AEs (≥20%) were anemia (46%), fatigue (37%) and nausea (28%). Grade ≥3 AEs were anemia (15%), hypertension (4%), urinary tract infection (2%), fatigue (2%), decreased appetite (1%), vomiting (1%), back pain (1%), diarrhea (1%) and nausea (0.3%).
Prostate cancer is the most commonly diagnosed cancer in men in Europe, with an estimated 473,000 cases and 108,000 deaths in 2020. Approximately 10-20% of patients with prostate cancer are estimated to develop castration-resistant prostate cancer (CRPC) within five years, with at least 84% of these patients presenting with metastases at the time of CRPC diagnosis. Patients diagnosed with advanced prostate cancer have a particularly poor prognosis, with a five-year relative survival rate of about 30%, compared to patients diagnosed with earlier stages of the disease, with a five-year relative survival rate of more than 99%.
Noel Clarke, urological surgeon and professor of urological oncology at Manchester’s Christie/Salford Royal Hospitals and Manchester University, a senior investigator of the PROpel trial, said, “The results of the PROpel Phase 3 trial of olaparib in combination with abiraterone as a first-line treatment show that this therapeutic combination can provide significant clinical benefit to patients with mCRPC. Patients with this condition in the EU will now, for the first time, have the opportunity to benefit from this new treatment combination.”
Dave Fredrickson, executive vice president, oncology business unit, AstraZeneca, said, “Many patients with mCRPC are only able to receive one line of active therapy, as the disease can progress quickly. LYNPARZA in combination with abiraterone has been shown to reduce the risk of disease progression by 34% versus the standard of care treatment in the PROpel trial. Moreover, the combination of LYNPARZA with abiraterone as a first-line treatment expands the use of LYNPARZA to a broader group of mCRPC patients than those treated with LYNPARZA alone in the second-line setting in the PROfound trial. Today’s approval marks a significant advance toward addressing the unmet need of patients with mCRPC in the EU.”
Dr. Eliav Barr, senior vice president, head of global clinical development and chief medical officer, Merck Research Laboratories, said, “Merck is committed to developing new treatment options for patients with mCRPC, a complex disease that urgently needs more therapies. This approval by the European Commission marks another step toward delivering on that commitment, and we look forward to extending the benefits of LYNPARZA to more patients with mCRPC in the EU.”
Use of LYNPARZAin combination with abiraterone and prednisone or prednisolone is currently under review by the U.S. FDA for the treatment of adult patients with mCRPC. LYNPARZA is approved in the U.S. as monotherapy for patients with homologous recombination repair (HRR) gene-mutated mCRPC (BRCA-mutated and other HRR gene mutations) who have progressed following prior treatment with enzalutamide or abiraterone and in the EU, Japan and China for patients with BRCA-mutated mCRPC who have progressed following prior therapy that included a new hormonal agent (NHA). These approvals were based on the data from the Phase 3 PROfound trial.
About PROpel
PROpel is a randomized, double-blind Phase 3 trial testing the efficacy, safety and tolerability of LYNPARZA versus placebo when given in addition to abiraterone and prednisone or prednisolone in patients with mCRPC who had not received prior chemotherapy or NHAs in the mCRPC setting. The major efficacy outcome was rPFS as assessed by investigator per RECIST v1.1 and Prostate Cancer Working Group (bone) criteria. OS was an additional efficacy outcome measure.
INDICATIONS
LYNPARZA is a poly (ADP-ribose) polymerase (PARP) inhibitor indicated:
First-Line Maintenance BRCAm Advanced Ovarian Cancer
For the maintenance treatment of adult patients with deleterious or suspected deleterious germline or somatic BRCA-mutated (gBRCAm or sBRCAm) advanced epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.
First-Line Maintenance HRD-Positive Advanced Ovarian Cancer in Combination with Bevacizumab
In combination with bevacizumab for the maintenance treatment of adult patients with advanced epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemotherapy and whose cancer is associated with homologous recombination deficiency (HRD)-positive status defined by either:
- a deleterious or suspected deleterious BRCA mutation, and/or
- genomic instability
Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.
Maintenance Recurrent Ovarian Cancer
For the maintenance treatment of adult patients with recurrent epithelial ovarian, fallopian tube, or primary peritoneal cancer, who are in complete or partial response to platinum-based chemotherapy.
Adjuvant Treatment of gBRCAm, HER2-Negative, High-Risk Early Breast Cancer
For the adjuvant treatment of adult patients with deleterious or suspected deleterious gBRCAm, human epidermal growth factor receptor 2 (HER2)-negative high-risk early breast cancer who have been treated with neoadjuvant or adjuvant chemotherapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.
gBRCAm, HER2-Negative Metastatic Breast Cancer
For the treatment of adult patients with deleterious or suspected deleterious gBRCAm, human epidermal growth factor receptor 2 (HER2)-negative metastatic breast cancer who have been treated with chemotherapy in the neoadjuvant, adjuvant, or metastatic setting. Patients with hormone receptor (HR)-positive breast cancer should have been treated with a prior endocrine therapy or be considered inappropriate for endocrine therapy. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.
First-Line Maintenance gBRCAm Metastatic Pancreatic Cancer
For the maintenance treatment of adult patients with deleterious or suspected deleterious gBRCAm metastatic pancreatic adenocarcinoma whose disease has not progressed on at least 16 weeks of a first-line platinum-based chemotherapy regimen. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.
HRR Gene-mutated Metastatic Castration-Resistant Prostate Cancer
For the treatment of adult patients with deleterious or suspected deleterious germline or somatic homologous recombination repair (HRR) gene-mutated metastatic castration-resistant prostate cancer (mCRPC) who have progressed following prior treatment with enzalutamide or abiraterone. Select patients for therapy based on an FDA-approved companion diagnostic for LYNPARZA.
Please see complete Prescribing Information , including Medication Guide .
Financial considerations
Under the oncology collaboration with AstraZeneca and following this new approval for LYNPARZA, AstraZeneca will receive a $105 million payment from Merck.
About LYNPARZA® (olaparib)
LYNPARZAis a first-in-class PARP inhibitor and the first targeted treatment to potentially exploit DNA damage response (DDR) pathway deficiencies, such as BRCA mutations, to preferentially kill cancer cells. Inhibition of PARP with LYNPARZA leads to the trapping of PARP bound to DNA single-strand breaks, stalling of replication forks, their collapse and the generation of DNA double-strand breaks and cancer cell death. LYNPARZA is being tested in a range of tumor types with defects and dependencies in the DDR.
LYNPARZA, which is being jointly developed and commercialized by AstraZeneca and Merck, has a broad clinical trial development program, and AstraZeneca and Merck are working together to understand how it may affect multiple PARP-dependent tumors as a monotherapy and in combination across multiple cancer types.
About the AstraZeneca and Merck strategic oncology collaboration
In July 2017, AstraZeneca and Merck, known as MSD outside of the United States and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialize certain oncology products including LYNPARZA, the world’s first PARP inhibitor, for multiple cancer types. Working together, the companies will develop these products in combination with other potential new medicines and as monotherapies. Independently, the companies will develop these oncology productsin combination with their respective PD-L1 and PD-1 medicines.
Merck’s focus on cancer
Our goal is to translate breakthrough science into innovative oncology medicines to help people with cancer worldwide. At Merck, the potential to bring new hope to people with cancer drives our purpose and supporting accessibility to our cancer medicines is our commitment. As part of our focus on cancer, Merck is committed to exploring the potential of immuno-oncology with one of the largest development programs in the industry across more than 30 tumor types. We also continue to strengthen our portfolio through strategic acquisitions and are prioritizing the development of several promising oncology candidates with the potential to improve the treatment of advanced cancers. For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials.
About Merck
At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
Source: Merck & Co., Inc.
AstraZeneca, Merck's Lynparza gets approval in EU for expanded use in prostate cancer
Dec. 21, 2022 4:29 AM ET
By: Ravikash, SA News Editor
- The European Commission (EC) approved AstraZeneca (NASDAQ:AZN) and Merck's (NYSE:MRK) Lynparza in combination with abiraterone and prednisone or prednisolone, to treat adult men with metastatic castration-resistant prostate cancer for whom chemotherapy is not clinically indicated.
- https://seekingalpha.com/news/3919299-astrazeneca-mercks-lynparza-gets-approval-in-eu-for-expanded-use-in-prostate-cancer
- AstraZeneca PLC (AZN), MRK
- https://www.lynparza.com/
- https://www.merck.com/
- https://www.astrazeneca.com/
KRAZATI™ (adagrasib)
Mirati Therapeutics Announces U.S. FDA Accelerated Approval of KRAZATI™ (adagrasib) as a Targeted Treatment Option for Patients with Locally Advanced or Metastatic Non-Small Cell Lung Cancer (NSCLC) with a KRASG12C Mutation
12/12/2022
SAN DIEGO, Dec. 12, 2022 /PRNewswire/ -- Mirati Therapeutics, Inc. (NASDAQ: MRTX), a targeted oncology company, today announced that the U.S. Food and Drug Administration (FDA) has granted accelerated approval for KRAZATI™ (adagrasib), a targeted treatment option for adult patients with KRASG12C-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.
Experience the full interactive Multichannel News Release here: https://www.multivu.com/players/English/8999051-mirati-therapeutics-fda-accelerated-approval-of-krazati-adagrasib/
This indication is approved under accelerated approval based on objective response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of a clinical benefit in a confirmatory trial(s).
To view the multimedia assets associated with this release, please visit: Mirati.com/approval
"The FDA approval of KRAZATI is a positive development for thousands of patients with KRASG12C mutations, including the approximately 14% of patients with NSCLC adenocarcinomas histology that harbor a KRASG12C mutation.1 Mirati is thrilled to make KRAZATI available in a tablet formulation to patients in the U.S. with advanced NSCLC who have progressed beyond a first-line treatment for the historically difficult-to-treat KRAS mutation," David Meek, chief executive officer, Mirati Therapeutics, Inc., continued, "We look forward to continuing to advance our KRAZATI development program including several monotherapy and combination studies in KRASG12C-mutated solid tumors."
KRAZATI has demonstrated a positive benefit-risk profile with accelerated approval based on the Phase 2 registration-enabling cohort of the KRYSTAL-1 study, evaluating KRAZATI 600 mg capsules administered orally twice daily in 116 patients with KRASG12C-mutated advanced NSCLC who previously received treatment with a platinum-based regimen and an immune checkpoint inhibitor. The primary efficacy endpoints were confirmed ORR and DOR as evaluated by blinded independent central review (BICR) according to response evaluation criteria in solid tumors (RECIST v1.1).
The trial demonstrated an ORR of 43% (95% CI: 34-53) with 80% (95% CI: 71-87) of patients achieving disease control. The median DOR was 8.5 months (95% CI: 6.2-13.8).
In a pooled efficacy analysis (n=132) including Phase 1/1b NSCLC and registrational Phase 2 NSCLC cohorts from the KRYSTAL-1 study evaluating adagrasib as a single agent at 600 mg capsules orally twice daily, adagrasib showed an ORR of 44% and a disease control rate of 81% based on BICR, a median DOR of 12.5 months (95% CI, 7.3-NE) and median overall survival of 14.1 months (94% CI, 9.2-19.2).
The safety profile of KRAZATI was evaluated in a pooled patient population with NSCLC and other solid tumors as a single agent at 600 mg orally twice daily in 366 patients enrolled in KRYSTAL-1 and KRYSTAL-12. The most common (≥ 25%) adverse reactions were nausea, diarrhea, vomiting, fatigue, musculoskeletal pain, hepatotoxicity, renal impairment, edema, dyspnea and decreased appetite. Permanent discontinuation of KRAZATI due to an adverse reaction occurred in 13% of patients.
Although KRASG12C is the most common KRAS mutation in NSCLC, patients have had limited options for the treatment of this debilitating and difficult-to-treat condition.2,3
"The approval of KRAZATI offers an effective therapy for patients with advanced NSCLC harboring the KRASG12C mutation. The positive ORR and DOR results, as observed in previously treated patients with NSCLC harboring the KRASG12C mutation, demonstrate the effectiveness of KRAZATI as an option for these difficult-to-treat patients," said Shirish M. Gadgeel, MD, chief of the Division of Hematology and Oncology, Department of Internal Medicine, Henry Ford Cancer Institute/Henry Ford Health System.
"KRASG12C in NSCLC is an area of high unmet need and new treatment options offer patients and our community new hope for survivorship," said Bonnie J. Addario, co-founder and board chair of the GO2 Foundation for Lung Cancer. "I'm pleased that patients have options, there's more awareness of this disease and we are all focused on improving the journeys of people living with KRASG12C-mutated NSCLC."
The Company partnered with Agilent and QIAGEN to develop blood- and tissue-based companion diagnostics (CDx), respectively, for KRAZATI that are now available. With tissue and blood modalities for companion diagnostics, patients have more flexibility, and clinicians have greater options for biomarker testing. These solutions help to personalize a patient's treatment path.
Mirati Therapeutics is launching Mirati & Me, a comprehensive program dedicated to supporting patients, caregivers and the oncology community including coverage and access, financial, educational and emotional support services. Learn more by visiting the Mirati & Me website or 1-844-647-2842.
For more information, visit KRAZATI.com.
About KRAZATI (adagrasib)
Mirati has risen to meet one of the most challenging mutations in cancer research by developing KRAZATI, a highly selective and potent oral small-molecule inhibitor of KRASG12C.
Intentionally designed to meet the challenge of KRASG12C, adagrasib is optimized to sustain target inhibition, an attribute that could be important to treat KRASG12C-mutated cancers, as the KRASG12C protein regenerates every 24−48 hours.4 Adagrasib has shown clinically to be a CNS penetrant, which may be important given that CNS metastases frequently occur in NSCLC and lead to poor prognosis.5,6,7
In the U.S., KRAZATI was reviewed by the FDA for Accelerated Approval (Subpart H), which allows for the approval of drugs that treat serious conditions, and that fill an unmet medical need based on surrogate endpoints. KRAZATI was reviewed under the FDA Real-Time Oncology Review (RTOR) pilot program, which aims to explore a more efficient review process that ensures safe and effective treatments are made available to patients as early as possible. Mirati submitted a Marketing Authorization Application (MAA) in the EU in May 2022. In 2021, adagrasib achieved Breakthrough Therapy Designation in the U.S. as a potential treatment for patients with NSCLC harboring the KRASG12C mutation who have received at least one prior systemic therapy.
Adagrasib continues to be evaluated as monotherapy and in combination with other anti-cancer therapies in patients with advanced KRASG12C-mutated solid tumors, including NSCLC, colorectal cancer, and pancreatic cancer. For more information, visit Mirati.com/science.
Mirati has an Expanded Access Program (EAP) for adagrasib for the treatment of eligible patients with KRASG12C-mutated cancers, regardless of tumor type, including patients with treated or untreated CNS metastases, in the U.S. Learn more about the EAP at Mirati.com/expanded-access-policy.
KRAZATI (adagrasib) U.S. Indication
KRAZATI is indicated for the treatment of adult patients with KRASG12C-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 objective response rate (ORR) and duration of response (DOR). Continued approval for this indication may be contingent upon verification and description of a clinical benefit in a confirmatory trial(s).
Please see Full Prescribing Information .
About the KRYSTAL-1 Study
KRYSTAL-1 is an open-label Phase 1/2 multiple-expansion cohort trial evaluating adagrasib as monotherapy and in combination with other anti-cancer therapies in patients with advanced solid tumors harboring the KRASG12C mutation.
About KRASG12C in NSCLC
Lung cancer is one of the most common cancers worldwide, accounting for 2.21 million new cases and 1.8 million deaths worldwide in 2020.8 Lung cancer consists of NSCLC in approximately 85% of cases and small cell lung cancer (SCLC) in approximately 15% of cases.9 KRASG12C is the most common KRAS mutation in NSCLC, present in approximately 14% of patients with lung adenocarcinoma, and is a biomarker mutation of poor prognosis.1,3
About Mirati Therapeutics, Inc.
Mirati Therapeutics, Inc. is a commercial-stage biotechnology company whose mission is to discover, design and deliver breakthrough therapies to transform the lives of patients with cancer and their loved ones. The company is relentlessly focused on bringing forward therapies that address areas of high unmet need, including lung cancer, and advancing a pipeline of novel therapeutics targeting the genetic and immunological drivers of cancer. Unified for patients, Mirati's vision is to unlock the science behind the promise of a life beyond cancer.
For more information about Mirati, visit us at Mirati.com or follow us on Twitter, LinkedIn and Facebook.
SOURCE Mirati Therapeutics, Inc.
Mirati up 4% as Krazati granted Breakthough Therapy designation for colorectal cancer
Dec. 21, 2022 5:39 PM ET Mirati Therapeutics, Inc. (MRTX)
By: Jonathan Block, SA News Editor
- Mirati Therapeutics' (NASDAQ:MRTX) Krazati (adagrasib), which recently won US FDA approval for non-small cell lung cancer, was granted Breakthrough Therapy designation by the agency for a type of colorectal cancer ("CRC").
- https://seekingalpha.com/news/3919658-mirati-up-4-krazati-granted-breakthrough-therapy-designation-for-colorectal-cancer
- Mirati Therapeutics, Inc. (MRTX)
- https://www.krazati.com/
Actemra®/RoActemra® (tocilizumab) injection
FDA approves Roche’s Actemra for the treatment of COVID-19 in hospitalised adults December 21, 2022
- Actemra is the first FDA-approved monoclonal antibody to treat COVID-19
- Since the beginning of the pandemic, more than one million people hospitalised with COVID-19 have been treated with Actemra worldwide
- Actemra is approved for this use in more than 30 countries for the treatment of COVID-19
Basel, 21 December 2022 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the U.S. Food and Drug Administration (FDA) has approved Actemra® (tocilizumab) intravenous (IV) for the treatment of COVID-19 in hospitalised adult patients who are receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO). Actemra is the first FDA-approved monoclonal antibody to treat COVID-19 and is recommended for use as a single 60-minute IV infusion.
“With new variants emerging, FDA-approved treatments including Actemra remain essential to the continued fight against COVID-19,” said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development. “Actemra is the first FDA-approved monoclonal antibody for treating patients with severe COVID-19, providing an important option for hospitalised patients and their healthcare providers who continue to be on the frontlines treating COVID-19.”
Four randomised, controlled studies evaluated Actemra for the treatment of COVID-19 in more than 5,500 hospitalised patients. Altogether, the results of these four studies (the University of Oxford-led RECOVERY trial, along with the Roche-sponsored global trials, EMPACTA, COVACTA and REMDACTA) showed that Actemra may improve outcomes in patients receiving corticosteroids and requiring supplemental oxygen or breathing support. The FDA approval is based on the results from the RECOVERY trial, as well as the EMPACTA trial, the first global, Phase III study in COVID-19 to focus on patients from underrepresented racial and ethnic groups. No new warnings and precautions related to Actemra in COVID-19 studies have been identified.
The FDA approval follows the FDA’s Emergency Use Authorization (EUA) for Actemra in hospitalised adults and children (ages 2 and older) with COVID-19, which was granted in June 2021. The use of Actemra to treat hospitalised people ages 2 to less than 18 years old is not FDA approved, however the EUA for this age group currently remains in place after the FDA approval for hospitalised adult patients.
More than one million people hospitalised with COVID-19 have been treated with Actemra worldwide, since the beginning of the pandemic. Around the world, Actemra is approved for use in more than 30 countries for patients hospitalised with severe COVID-19. In the United States, this is the seventh FDA approved indication for Actemra since the medicine was launched in 2010.
Roche stands together with society, governments, healthcare providers and all those working towards the common goal of overcoming the COVID-19 pandemic.
About Actemra/RoActemra in COVID-19
Actemra®/RoActemra® is approved for use in multiple territories including the United States, European Union, Japan, the United Kingdom, New Zealand, Russia and Brazil, provisionally approved in Australia, and authorised for emergency use in Ghana, Mexico and Korea for defined patients hospitalised with severe or critical COVID-19. It has also been recommended and prequalified by the World Health Organization.
About Actemra/Roactemra
Actemra®/RoActemra® was the first humanised interleukin-6 (IL-6) receptor antagonist approved for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA) who have used one or more disease-modifying antirheumatic drugs (DMARDs), such as methotrexate (MTX), that did not provide enough relief. The extensive Actemra RA IV clinical development program included five Phase III clinical studies and enrolled more than 4,000 people with RA in 41 countries. The Actemra RA subcutaneous clinical development program included two Phase III clinical studies and enrolled more than 1,800 people with RA in 33 countries. Actemra subcutaneous injection is also approved for the treatment of adult patients with giant cell arteritis (GCA), for the treatment of patients two years of age and older with active polyarticular juvenile idiopathic arthritis (PJIA) or active systemic juvenile idiopathic arthritis (SJIA), and for slowing the rate of decline in pulmonary function in adult patients with systemic sclerosis-associated interstitial lung disease (SSc-ILD). In addition, Actemra is also approved in the IV formulation for patients two years of age and older with active PJIA, SJIA, GCA or CAR T cell-induced cytokine release syndrome (CRS). Actemra is not approved for subcutaneous use in people with CRS. Actemra IV is approved for the treatment of COVID-19 in hospitalised adult patients who are receiving systemic corticosteroids and require supplemental oxygen, non-invasive or invasive mechanical ventilation, or extracorporeal membrane oxygenation (ECMO). It is not known if Actemra is safe and effective in children with PJIA, SJIA or CRS under two years of age or in children with conditions other than PJIA, SJIA or CRS.
About Roche’s response to the COVID-19 pandemic
As a leading healthcare company, we are doing all we can to support countries in their fight against COVID-19 and minimising its impact. That is why we are working with governments, policy makers, healthcare professionals and others to help contain the COVID-19 pandemic and make sure patients continue to receive the tests, treatment and care they need.
The pandemic has profoundly raised awareness of the role diagnostics play in COVID-19 diagnosis, treatment development and disease management. Roche has developed and launched more than 20 COVID-19 diagnostics solutions, including polymerase chain reaction (PCR) and rapid antigen and antibody tests. Our solutions serve the entire diagnostic continuum, from high-throughput laboratories to point-of-care and home self-testing, and cover all currently known variants. To help meet global demand, we have supplied more than 1.5 billion tests for COVID-19 since March 2020.
Roche continues to evaluate its existing therapeutic portfolio and is researching future options to help benefit patients with COVID-19. Our IL-6 inhibitor Actemra®/RoActemra® (tocilizumab) has been approved for patients hospitalised with severe COVID-19 in more than 30 countries including the European Union and the United States. The World Health Organization has prequalified Actemra for use in patients with severe COVID-19, facilitating its availability in low- and middle-income countries. In addition, we have been improving access to Actemra by introducing an international differentiated pricing strategy, providing the medicine at cost for use in low- and middle-income countries and non asserting patents in these regions during the pandemic.
We have also been partnering with Regeneron to jointly develop the antibody combination Ronapreve™ (casirivimab and imdevimab, known as REGEN-COV™ in the US). It has been approved in multiple territories including the European Union, Japan, and Switzerland and authorised for emergency or temporary pandemic use in many countries including the US. We are constantly monitoring and assessing Ronapreve’s neutralising activity against emerging variants of concern and entered a dialogue with regulators and industry representatives to discuss the efficacy of monoclonal antibodies in the context of rapidly evolving SARS-CoV-2 variants.
Our utmost goal remains to be a trusted partner who acts with urgency to save and improve the lives of patients with COVID-19 and to reduce its burden on society. For more information please visit our COVID-19 response page.
About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.
In recognising our endeavor to pursue a long-term perspective in all we do, Roche has been named one of the most sustainable companies in the pharmaceuticals industry by the Dow Jones Sustainability Indices for the thirteenth consecutive year. This distinction also reflects our efforts to improve access to healthcare together with local partners in every country we work.
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.
Roche's Actemra granted FDA approval for COVID-19
Dec. 21, 2022 6:00 PM ET
Roche Holding AG (RHHBF), RHHBY
By: Jonathan Block, SA News Editor
- Roche's (OTCQX:RHHBF) Actemra (tocilizumab) has become the first monoclonal antibody approved by the US FDA for COVID-19.
- https://seekingalpha.com/news/3919661-roche-actemra-granted-fda-approval-for-covid-19
- Roche Holding AG (RHHBF), RHHBY
- https://www.actemra.com/
- https://www.roche.com/
- https://www.gene.com/
PADCEV® (enfortumab vedotin-ejfv) with KEYTRUDA® (pembrolizumab)
Seagen, Astellas and Merck Announce FDA Acceptance of sBLAs for PADCEV® (enfortumab vedotin-ejfv) with KEYTRUDA® (pembrolizumab) for the First-Line Treatment of Certain Patients With Locally Advanced or Metastatic Urothelial Cancer
12/20/2022
– This combination has the potential to be the first treatment option combining an antibody-drug conjugate plus an immunotherapy in this treatment setting –
– FDA granted the applications Priority Review with a PDUFA date of April 21, 2023 –
BOTHELL, Wash. & TOKYO & RAHWAY, N.J.--(BUSINESS WIRE)--
Seagen Inc. (Nasdaq: SGEN),Astellas Pharma Inc. (TSE:4503) and Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced the U.S. Food and Drug Administration (FDA) has accepted for Priority Review supplemental Biologics License Applications (sBLAs) for PADCEV® (enfortumab vedotin-ejfv) and KEYTRUDA® (pembrolizumab) for use of these two agents in combination for the treatment of patients with locally advanced or metastatic urothelial cancer (la/mUC) who are not eligible to receive cisplatin-containing chemotherapy. The respective applications are intended to expand both labels for PADCEV and KEYTRUDA. The agency set a Prescription Drug User Fee Act (PDUFA) goal date for each application of April 21, 2023.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20221220005198/en/
“We look forward to working closely with the FDA as we seek potential accelerated approval for this combination in the hopes that it can be another treatment option for patients with locally advanced or metastatic urothelial cancer who are not eligible for cisplatin-containing chemotherapy,” said Ahsan Arozullah, M.D., M.P.H., Senior Vice President and Head of Development Therapeutic Areas, Astellas.
The combination therapy was granted Breakthrough Therapy designation by the FDA in February 2020. The respective sBLAs are supported by efficacy and safety data from the phase 1b/2 EV-103 trial (NCT03288545, also known as KEYNOTE-869) Dose Escalation/Cohort A and Cohort K. Results from Dose Escalation/Cohort A were published in the Journal of Clinical Oncology.1 Results from Cohort K were presented in a late-breaking session at the 2022 European Society for Medical Oncology (ESMO) Congress.2
“Urothelial cancer, the most common type of bladder cancer, is associated with poor survival in the advanced stage,” said Marjorie Green, M.D., Senior Vice President and Head of Late-Stage Development, Seagen. “The investigational results from our clinical development program support the combination of PADCEV and KEYTRUDA as a potential treatment for this patient population.”
Please see Important Safety Information at the end of this press release for both drugs, including a warning and precaution for immune-mediated adverse reactions for pembrolizumab and BOXED WARNING for PADCEV (enfortumab vedotin-ejfv) for serious skin reactions.
“Despite advancements in treatment options, approximately half of advanced bladder cancer patients in the U.S. are ineligible for cisplatin-based chemotherapy, and these patients need new options. We are encouraged by the investigational results of the combination of PADCEV and KEYTRUDA for this patient population and are fully committed to work to bring this new approach forward to patients,” said Dr. Eliav Barr, Senior Vice President, Head of Global Clinical Development and Chief Medical Officer, Merck Research Laboratories.
Seagen, Astellas and Merck are further investigating enfortumab vedotin plus pembrolizumab in the ongoing phase 3 EV-302 study (NCT04223856, also known as KEYNOTE-A39), evaluating the clinical benefit for the investigational treatment combination in patients with previously untreated advanced urothelial cancer. The trial is intended to serve as the confirmatory trial for the potential accelerated approval in the U.S. and serve as the basis for global registration.
The studies are part of an extensive program evaluating this combination in multiple stages of urothelial cancer, including two phase 3 clinical trials in muscle-invasive bladder cancer in EV-304 (NCT04700124, also known as KEYNOTE-B15) and EV-303 (NCT03924895, also known as KEYNOTE-905).
About the EV-103/KEYNOTE-869 Trial
The EV-103 trial (NCT03288545) is an ongoing, multi-cohort, open-label, multicenter phase 1b/2 study of enfortumab vedotin alone or in combination with pembrolizumab and/or chemotherapy in first- or second-line settings in patients with locally advanced or metastatic urothelial cancer (la/mUC) and in patients with muscle-invasive bladder cancer.
About PADCEV
PADCEV (enfortumab vedotin-ejfv) is a first-in-class antibody-drug conjugate (ADC) that is directed against Nectin-4, a protein located on the surface of cells and highly expressed in bladder cancer.7 Nonclinical data suggest the anticancer activity of PADCEV is due to its binding to Nectin-4-expressing cells followed by the internalization and release of the anti-tumor agent monomethyl auristatin E (MMAE) into the cell, which result in the cell not reproducing (cell cycle arrest) and in programmed cell death (apoptosis).8
Indication
PADCEV® is indicated for the treatment of adult patients with locally advanced or metastatic urothelial cancer (mUC) who:
- have previously received a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-L1) inhibitor and platinum-containing chemotherapy, or
- are ineligible for cisplatin-containing chemotherapy and have previously received one or more prior lines of therapy.8
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.
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 Seagen
Seagen Inc. is a global biotechnology company that discovers, develops and commercializes transformative cancer medicines to make a meaningful difference in people’s lives. Seagen is headquartered in the Seattle, Washington area, and has locations in California, Canada, Switzerland and the European Union. For more information on the company’s marketed products and robust pipeline, visit www.seagen.com and follow @SeagenGlobal on Twitter.
About Astellas
Astellas Pharma Inc. is a pharmaceutical company conducting business in more than 70 countries around the world. We are promoting the Focus Area Approach that is designed to identify opportunities for the continuous creation of new drugs to address diseases with high unmet medical needs by focusing on Biology and Modality. Furthermore, we are also looking beyond our foundational Rx focus to create Rx+® healthcare solutions that combine our expertise and knowledge with cutting-edge technology in different fields of external partners. Through these efforts, Astellas stands on the forefront of healthcare change to turn innovative science into VALUE for patients. For more information, please visit our website at https://www.astellas.com/en.
Merck’s focus on cancer
Our goal is to translate breakthrough science into innovative oncology medicines to help people with cancer worldwide. At Merck, the potential to bring new hope to people with cancer drives our purpose and supporting accessibility to our cancer medicines is our commitment. As part of our focus on cancer, Merck is committed to exploring the potential of immuno-oncology with one of the largest development programs in the industry across more than 30 tumor types. We also continue to strengthen our portfolio through strategic acquisitions and are prioritizing the development of several promising oncology candidates with the potential to improve the treatment of advanced cancers. For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials.
About Merck
At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
About the Seagen, Astellas and Merck Collaboration
Seagen and Astellas entered a clinical collaboration agreement with Merck to evaluate the combination of Seagen’s and Astellas’ PADCEV® (enfortumab vedotin-ejfv) and Merck’s KEYTRUDA® (pembrolizumab) in patients with previously untreated metastatic urothelial cancer. KEYTRUDA is a registered trademark of Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.
View source version on businesswire.com: https://www.businesswire.com/news/home/20221220005198/en/
Source: Seagen Inc.
Merck, Seagen, Astellas combo bladder cancer treatment sBLAs accepted by FDA
Dec. 20, 2022 8:41 AM ET
Astellas Pharma Inc. (ALPMF), ALPMY, SGEN, MRK
By: Jonathan Block, SA News Editor
- The US FDA has accepted supplemental Biologics License Applications from Merck (NYSE:MRK), Seagen (NASDAQ:SGEN), and Astellas (OTCPK:ALPMF)(OTCPK:ALPMY) for a combination of Keytruda (pembrolizumab) and Padcev (enfortumab vedotin) for urothelial cancer.
- https://seekingalpha.com/news/3918891-merck-seagen-astellas-combo-bladder-cancer-treatment-sblas-accepted-by-fda
- Astellas Pharma Inc. (ALPMF), ALPMY, SGEN, MRK
- https://www.padcev.com/
- https://www.keytruda.com/
DANYELZA® (naxitamab-gqgk)
Y-mAbs Announces Partnership Regarding Early Access Program for DANYELZA (naxitamab-gqgk) in Europe
Dec 21, 2022PDF Version
NEW YORK, Dec. 21, 2022 (GLOBE NEWSWIRE) -- Y-mAbs Therapeutics, Inc. (the “Company” or “Y-mAbs”) (Nasdaq: YMAB) a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, today announced that it has entered into a distribution agreement with WEP Clinical Ltd. (“WEP”) in connection with an early access program for DANYELZA (naxitamab-gqgk) 40mg/10mL Injection in Europe.
DANYELZA is a humanized, monoclonal antibody that binds to the glycolipid GD2. GD2 is a disialoganglioside that is overexpressed on neuroblastoma cells and other cells of neuroectodermal origin, including the central nervous system and peripheral nerves. DANYELZA is administered on days 1, 3, and 5 of each treatment cycle as an intravenous infusion after dilution. Treatment cycles are repeated every 4 weeks until complete response or partial response, followed by 5 additional cycles every 4 weeks.
In the U.S., DANYELZA is approved by the FDA and indicated, in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy.
“We are excited to be able to give European patients access to DANYELZA through this early access program with WEP,” said Thomas Gad, founder, President and Interim CEO. “Initially, DANYELZA will be available through such program in Spain and France, and we hope to add additional territories to the agreement.”
Researchers at Memorial Sloan Kettering Cancer Center (“MSK”) developed DANYELZA, which is exclusively licensed by MSK to Y-mAbs. MSK has institutional financial interests related to the compound and Y-mAbs.
About DANYELZA® (naxitamab-gqgk)
DANYELZA® (naxitamab-gqgk) is indicated, in combination with granulocyte-macrophage colony-stimulating factor (“GM-CSF”), for the treatment of pediatric patients 1 year of age and older and adult patients with relapsed or refractory high-risk neuroblastoma in the bone or bone marrow who have demonstrated a partial response, minor response, or stable disease to prior therapy. This indication was approved in the United States by the FDA under accelerated approval based on overall response rate and duration of response. Continued approval for this indication is contingent upon verification and description of clinical benefits in a confirmatory trial. DANYELZA® includes a Boxed Warning for serious infusion-related reactions, such as cardiac arrest and anaphylaxis, and neurotoxicity, such as severe neuropathic pain and transverse myelitis. See full Prescribing Information (https://labeling.ymabs.com/danyelza) for complete Boxed Warning and other important safety information.
About Y-mAbs
Y-mAbs is a commercial-stage biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic cancer products. In addition to conventional antibodies, the Company’s technologies include bispecific antibodies generated using the Y-BiClone platform and the SADA platform. The Company’s product pipeline includes one FDA-approved product, DANYELZA® (naxitamab-gqgk), which targets tumors that express GD2, and one product candidate at the registration-stage, OMBLASTYS® (omburtamab), which targets tumors that express B7-H3.
DANYELZA®, OMBLASTYS® and Y-mAbs® are registered trademarks of Y-mAbs Therapeutics, Inc.
Source: Y-mAbs Therapeutics, Inc
Y-mAbs inks contract with WEP for early access for cancer drug Danyelza in Europe
Dec. 21, 2022 9:49 AM ET
Y-mAbs Therapeutics, Inc. (YMAB)
By: Ravikash, SA News Editor
- Y-mAbs Therapeutics (NASDAQ:YMAB) said it signed a distribution agreement with WEP Clinical for an early access program for its neuroblastoma drug Danyelza (naxitamab-gqgk) 40mg/10mL Injection in Europe.
- https://seekingalpha.com/news/3919426-y-mabs-inks-contract-with-wep-for-early-access-of-cancer-drug-danyelza-in-europe
- Y-mAbs Therapeutics, Inc. (YMAB)
- https://danyelza.com/
- https://ymabs.com/
UPLIZNA® (inebilizumab-cdon)
UPLIZNA® (inebilizumab-cdon) Approved in Brazil for the Treatment of Neuromyelitis Optica Spectrum Disorder (NMOSD)
12/20/22PDF Version
-- First and only anti-CD19 B-cell-depleting monotherapy for the treatment of adult patients with NMOSD who are anti-aquaporin-4 immunoglobulin G seropositive (AQP4-IgG+) --
-- NMOSD is a devastating rare autoimmune disease that can cause vision loss and paralysis --
DUBLIN--(BUSINESS WIRE)--Dec. 20, 2022-- Horizon Therapeutics plc (Nasdaq: HZNP) today announced the Brazilian Health Regulatory Agency (ANVISA) has approved UPLIZNA as a monotherapy for the treatment of adult patients with NMOSD who are AQP4-IgG+. This rare autoimmune disease is caused by inflammation in the central nervous system, resulting in severe and recurrent attacks that can lead to permanent disability, such as paralysis and vision loss.1 An estimated 10,000 people in Brazil live with the disease.2
“This approval is an important part of Horizon’s commitment to helping people worldwide who are living with challenging rare diseases,” said Vikram Karnani, executive vice president, international and global medical affairs, Horizon. “The NMOSD community in Brazil now has an approved medicine that has been proven in clinical studies to provide sustained protection against NMOSD attacks.”
Unlike other autoimmune diseases, patients with NMOSD typically do not fully recover from attacks.3 It is estimated that 69% of patients have severe vision loss in at least one eye within three years of disease onset and 34% may develop permanent motor impairment.4,5,6 A primary goal of treatment is to help prevent cumulative damage and permanent disability that can be caused by subsequent attacks.
In the N-MOmentum pivotal clinical trial, UPLIZNA demonstrated a significant reduction in the risk of an NMOSD attack with only two infusions per year, following the initial loading doses. Additionally, 89% of patients in the AQP4-IgG+ group remained attack free during the six-month period post-treatment and more than 83% of patients on treatment remained attack free for at least four years.7
“NMOSD is an extremely debilitating and life-changing disease that can impact patients physically, socially and economically,” said Douglas Sato, M.D. Ph.D., neurologist, full professor of medicine, Pontifical Catholic University of Rio Grande do Sul (PUCRS). “UPLIZNA is unique in that it prevents attacks by depleting a broad range of B cells, including plasmablasts and plasma cells, and has a very good safety profile in NMOSD patients. This approval represents a significant advancement in the treatment of NMOSD and can provide hope for those who have been struggling with this disease.”
UPLIZNA was approved by the U.S. Food and Drug Administration (FDA) in June 2020, by the Japanese Ministry of Health, Labor and Welfare in March 2021 and by the European Commission (EC) in April 2022.
About Neuromyelitis Optica Spectrum Disorder (NMOSD)
NMOSD is a unifying term for neuromyelitis optica (NMO) and related syndromes. NMOSD is a rare, severe, relapsing, neuroinflammatory autoimmune disease that attacks the optic nerve, spinal cord, brain and brain stem.1,8 Approximately 80% of all patients with NMOSD test positive for anti-AQP4 antibodies.9 AQP4-IgG binds primarily to astrocytes in the central nervous system and triggers an escalating immune response that results in lesion formation and astrocyte death.10
Anti-AQP4 autoantibodies are produced by plasmablasts and plasma cells. These B-cell populations are central to NMOSD disease pathogenesis, and a large proportion of these cells express CD19.11 Depletion of these CD19+ B cells is thought to remove an important contributor to inflammation, lesion formation and astrocyte damage. Clinically, this damage presents as an NMOSD attack, which can involve the optic nerve, spinal cord and brain.10-12 Loss of vision, paralysis, loss of sensation, bladder and bowel dysfunction, nerve pain and respiratory failure can all be manifestations of the disease.13 Each NMOSD attack can lead to further cumulative damage and disability.14,15 NMOSD occurs more commonly in women and may be more common in individuals of African and Asian descent.16,17
About UPLIZNA (inebilizumab-cdon)
INDICATION
UPLIZNA is indicated for the treatment of neuromyelitis optica spectrum disorder (NMOSD) in adult patients who are anti-aquaporin-4 (AQP4) antibody positive.
About Horizon
Horizon is a global biotechnology company focused on the discovery, development and commercialization of medicines that address critical needs for people impacted by rare, autoimmune and severe inflammatory diseases. Our pipeline is purposeful: We apply scientific expertise and courage to bring clinically meaningful therapies to patients. We believe science and compassion must work together to transform lives. For more information on how we go to incredible lengths to impact lives, visit www.horizontherapeutics.com and follow us on Twitter, LinkedIn, Instagram and Facebook.
View source version on businesswire.com: https://www.businesswire.com/news/home/20221220005190/en/
Source: Horizon Therapeutics plc
Brazil approves Horizon Therapeutics' Uplizna to treat autoimmune disease NMOSD
Dec. 20, 2022 1:35 PM ET
Horizon Therapeutics Public Limited Company (HZNP)
By: Anuron Mitra, SA News Editor
- Horizon Therapeutics (NASDAQ:HZNP) on Tuesday said its Uplizna drug had received Brazilian regulatory approval for the treatment of adults with a rare autoimmune disease.
- https://seekingalpha.com/news/3919070-brazil-approves-horizon-therapeutics-uplizna-to-treat-autoimmune-disease-nmosd
- Horizon Therapeutics Public Limited Company (HZNP)
- https://www.uplizna.com/
- https://www.horizontherapeutics.com/
biotecMAX™ 2022 Insight
ENHERTU® (fam-trastuzumab deruxtecan-nxki) for injection
Enhertu recommended for approval in the EU by CHMP for patients with HER2-low metastatic breast cancer
PUBLISHED19 December 2022 19 December 2022 07:20 GMT
AstraZeneca and Daiichi Sankyo’s Enhertu is the first HER2-directed therapy to
demonstrate a significant survival benefit vs. chemotherapy in this patient population
AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) has been recommended for approval in the European Union (EU) as monotherapy for the treatment of adult patients with unresectable or metastatic HER2-low (IHC 1+ or IHC 2+/ISH-) breast cancer who have received prior chemotherapy in the metastatic setting or developed disease recurrence during or within six months of completing adjuvant chemotherapy.
Enhertu is a specifically engineered HER2-directed antibody drug conjugate (ADC) being jointly developed and commercialised by AstraZeneca and Daiichi Sankyo.
The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency based its positive opinion on results from the DESTINY-Breast04 Phase III trial, which were presented at the American Society of Clinical Oncology 2022 Annual Meeting and simultaneously published in The New England Journal of Medicine.1
In the trial, Enhertu reduced the risk of disease progression or death by 50% versus physician’s choice of chemotherapy (based on a hazard ratio [HR] of 0.50; 95% confidence interval [CI]: 0.40-0.63; p<0.001) in patients with HER2-low metastatic breast cancer with HR-positive or HR-negative disease. A median progression-free survival (PFS) of 9.9 months was seen with Enhertu versus 5.1 months in those treated with chemotherapy, as assessed by blinded independent central review (BICR). A 36% reduction in the risk of death (HR 0.64; 95% CI: 0.49-0.84; p=0.001) was seen with Enhertu compared to chemotherapy with a median overall survival (OS) of 23.4 months versus 16.8 months.
Susan Galbraith, Executive Vice President, Oncology R&D, AstraZeneca, said: “Enhertu is the first-ever HER2-directed medicine to show a survival benefit in patients with HER2-low metastatic breast cancer, confirming the importance of targeting lower levels of HER2 expression in patients previously classified as HER2-negative. The CHMP’s recommendation is encouraging and supports our ambition to evolve the way breast cancer is classified and treated to ultimately improve patient outcomes.”
Ken Takeshita, Global Head, R&D Daiichi Sankyo, said: “This positive CHMP opinion recognises the unmet need in the European Union for patients with HER2-low metastatic breast cancer. Currently, once patients with HR-positive disease progress on hormone therapy there are limited effective treatments, and few targeted options are available for patients with HR-negative disease. We look forward to the European Commission decision and aim to bring Enhertu to eligible patients as soon as possible.”
The safety profile observed in patients treated with Enhertu in the DESTINY-Breast04 trial was consistent with that seen in other trials of Enhertu in breast cancer with no new safety signals identified.
Notes
Breast cancer and HER2 expression
Breast cancer is the most common cancer and is one of the leading causes of cancer-related deaths worldwide.2 More than two million patients with breast cancer were diagnosed in 2020 with nearly 685,000 deaths globally.2 In Europe, approximately 531,000 breast cancer patients are diagnosed annually with nearly 141,000 deaths.3
HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumours including breast, gastric, lung and colorectal cancers, and is one of many biomarkers expressed in breast cancer tumours.4
HER2 expression is currently determined by an immunohistochemistry (IHC) test which estimates the amount of HER2 protein on a cancer cell, and/or an in situ hybridisation (ISH) test, which counts the copies of the HER2 gene in cancer cells.4,5 HER2 tests provide IHC and ISH scores across the full HER2 spectrum and are routinely used to determine appropriate treatment options for patients with metastatic breast cancer.
HER2-positive cancers are currently defined as HER2 expression measured as IHC 3+ or IHC 2+/ISH+, and HER2-negative cancers are defined as HER2 expression measured as IHC 0, IHC 1+ or IHC 2+/ISH-.4 However, approximately half of all breast cancers are HER2-low, defined as a HER2 score of IHC1+ or IHC 2+/ISH-.6-8 HER2-low occurs in both HR-positive and HR-negative disease.9
Currently, patients with HR-positive metastatic breast cancer and HER2-low disease have limited effective treatment options following progression on endocrine (hormone) therapy.10 Additionally, few targeted options are available for those with HR-negative disease.11
DESTINY-Breast04
DESTINY-Breast04 is a global, randomised, open-label, Phase III trial evaluating the efficacy and safety of Enhertu (5.4mg/kg) versus physician’s choice of chemotherapy (capecitabine, eribulin, gemcitabine, paclitaxel or nab-paclitaxel) in patients with HR-positive or HR-negative, HER2-low unresectable and/or metastatic breast cancer previously treated with one or two prior lines of chemotherapy. Patients were randomised 2:1 to receive either Enhertu or chemotherapy.
The primary endpoint of DESTINY-Breast04 is PFS in patients with HR-positive disease based on BICR. Key secondary endpoints include PFS based on BICR in all randomised patients (HR-positive and HR-negative disease), OS in patients with HR-positive disease and OS in all randomised patients (HR-positive and HR-negative disease). Other secondary endpoints include PFS based on investigator assessment, objective response rate based on BICR and on investigator assessment, duration of response based on BICR and safety.
DESTINY-Breast04 enrolled 557 patients at multiple sites in Asia, Europe and North America. For more information about the trial, visit ClinicalTrials.gov.