DetailsCategory: AntibodiesPublished on Sunday, 28 June 2020 14:40Hits: 307
Reblozyl regulates late-stage red blood cell (RBC) maturation to potentially reduce or eliminate the need for regular RBC transfusions
Reblozyl is the first and only erythroid maturation agent to be approved in the European Union, representing a new class of therapy
PRINCETON, NJ & CAMBRIDGE, MA, USA I June 26, 2020 IBristol Myers Squibb (NYSE: BMY) and Acceleron Pharma Inc. (NASDAQ: XLRN) today announced that the European Commission (EC) has approved Reblozyl (luspatercept) for the treatment of:
"Dependence on blood transfusions caused by anemia in hematologic malignancies like MDS can often mean frequent and lengthy hospital visits, which can pose additional health risks and affect patients quality of life, said Uwe Platzbecker, M.D., lead investigator of the MEDALIST study, Head of Clinic and Policlinic for Hematology and Cell Therapy, Leipzig University Hospital. Todays approval of Reblozyl provides healthcare professionals with a new therapy that has been shown to significantly reduce the number of red blood cell transfusions needed by MDS patients and, in some cases, helped them to achieve transfusion independence.
While beta thalassemia remains an orphan disease, the lifelong blood transfusions often needed by patients can have a significant impact on the limited blood supply in their communities, and there are few treatment alternatives, said Maria Domenica Cappellini, M.D., lead investigator of the BELIEVE study, Professor of Medicine, University of Milan, Fondazione IRCCS Ca Granda. The European Commissions approval of Reblozyl provides eligible adult patients with beta thalassemia a new, much needed treatment option for their anemia, and with it, the possibility of becoming less dependent on red blood cell transfusions.
Reblozyl is the first and only erythroid maturation agent approved in the European Union, representing a new class of therapy for eligible patients. This approval is based on data from the pivotal Phase 3 MEDALIST and BELIEVE studies, evaluating the ability of Reblozyl to effectively address anemia associated with MDS and beta thalassemia, respectively.
Across the EU, 25 million blood transfusions occur every year, some of which are needed by patients with anemia due to hematologic diseases like MDS and beta thalassemia, said Diane McDowell, M.D., vice president, Hematology Global Medical Affairs, Bristol Myers Squibb. Reblozyl has the potential to address the ineffective erythropoiesis associated with MDS and beta thalassemia, decrease patients dependence on red blood cell transfusions and impact the underlying consequences of the high burden of anemia for these patients. Alongside our partners at Acceleron, we recognize the continuing need in disease-related anemias and are committed to working collaboratively with European health authorities to make Reblozyl available to these patients as quickly as possible.
About MEDALIST
MEDALIST is a Phase 3, randomized, double-blind, placebo-controlled, multi-center study evaluating the safety and efficacy of Reblozyl plus best supportive care (BSC) versus placebo plus BSC in adults with IPSS-R-defined very low-, low- or intermediate-risk non-del(5q) MDS. All patients were RBC transfusion-dependent and were either refractory or intolerant to prior erythropoiesis stimulating agent (ESA) therapy, or were ESA nave and unlikely to respond due to endogenous serum erythropoietin levels of 200 U/L, and had no prior treatment with disease modifying agents.
The trial showed a statistically significant improvement in RBC transfusion burden with Reblozyl, the studys primary endpoint, with 37.9% of patients treated with Reblozyl achieving independence from RBC transfusions for at least eight weeks during the first 24 weeks of the trial compared to 13.2% of patients on placebo. The trial also met the secondary endpoint of transfusion independence for at least 12 weeks within the first 24 and 48 weeks of the study, which was achieved in a significantly greater proportion of patients receiving Reblozyl versus placebo.
The majority of treatment-emergent adverse events (TEAEs) were Grade 1-2. Grade 3 or 4 TEAEs were reported in 42.5% of patients who received Reblozyl and 44.7% of patients who received placebo. Discontinuation due to an adverse reaction (Grades 1-4) occurred in 4.5% of patients who received Reblozyl. The most common (>10%) all-grade adverse reactions included fatigue, musculoskeletal pain, dizziness, diarrhea, nausea, hypersensitivity reactions, hypertension, headache, upper respiratory tract infection, bronchitis and urinary tract infection.
Results of the MEDALIST trial were first presented during the Plenary Session of the American Society of Hematology (ASH) Annual Meeting in December 2018 (ASH Abstract #001) and were selected for the Best of ASH. The New England Journal of Medicine published the MEDALIST trial results in January 2020.
About MDS
MDS are a group of hematologic malignancies characterized by ineffective production of healthy red blood cells, white blood cells and platelets, which can lead to anemia and frequent or severe infections, and can progress to Acute Myeloid Leukemia (AML). People with MDS who develop anemia often require regular blood transfusions to increase the number of healthy red blood cells in circulation. Frequent transfusions are associated with an increased risk of transfusion reactions, infections and iron overload. There are approximately 50,000 patients with MDS in the EU5 countries (France, Germany, Italy, Spain and the United Kingdom).
About BELIEVE
BELIEVE is a Phase 3, randomized, double-blind, placebo-controlled multi-center study comparing Reblozyl plus BSC versus placebo plus BSC in adults who require regular RBC transfusions (6-20 RBC units per 24 weeks with no transfusion-free period greater than 35 days during that period) due to beta thalassemia.
The trial showed a statistically significant improvement in RBC transfusion burden during weeks 13 to 24 compared to the baseline 12-week interval prior to randomization (21.4% Reblozyl versus 4.5% placebo), meeting the studys primary endpoint. The trial also met the secondary endpoint of transfusion burden reduction of at least 33% (with a reduction of at least two units) during weeks 37 to 48, which was achieved in a significantly greater proportion of patients receiving Reblozyl versus placebo. The trial also met an exploratory endpoint, with 70.5% of patients treated with Reblozyl achieving at least a 33% reduction in RBC transfusion burden of at least two units for any 12 consecutive weeks compared to the 12-week interval prior to treatment, compared to 29.5% of patients on placebo.
The majority of TEAEs were Grade 1-2. Discontinuation due to an adverse reaction (Grades 1-4) occurred in 5.4% of patients who received Reblozyl. The most common adverse reactions (>10%) were headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea and dizziness.
Results of the BELIEVE trial were first presented at the ASH Annual Meeting in December 2018 and selected for the Best of ASH. The New England Journal of Medicine published the BELIEVE trial results in March 2020.
About Beta Thalassemia
Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. The disease is associated with ineffective erythropoiesis, which results in the production of fewer and less healthy RBCs, often leading to severe anemiaa condition that can be debilitating and can lead to other complications for patientsas well as other serious health issues. Treatment options for anemia associated with beta thalassemia are limited, consisting mainly of frequent RBC transfusions that have the potential to contribute to iron overload, which can cause serious complications such as organ damage. Across the United States, Germany, France, Italy, Spain and the United Kingdom, there are approximately 17,000 patients with beta thalassemia.
About Reblozyl
Reblozyl (luspatercept-aamt), a first-in-class erythroid maturation agent, promotes late-stage red blood cell maturation in animal models. Bristol Myers Squibb and Acceleron are jointly developing Reblozyl as part of a global collaboration. Reblozyl is currently approved in the U.S. for the treatment of:
Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.
Bristol Myers Squibb: Advancing Cancer Research
At Bristol Myers Squibb, patients are at the center of everything we do. The goal of our cancer research is to increase patients quality of life, long-term survival and make cure a possibility. We harness our deep scientific experience, cutting-edge technologies and discovery platforms to discover, develop and deliver novel treatments for patients.
Building upon our transformative work and legacy in hematology and Immuno-Oncology that has changed survival expectations for many cancers, our researchers are advancing a deep and diverse pipeline across multiple modalities. In the field of immune cell therapy, this includes registrational chimeric antigen receptor (CAR) T-cell agents for numerous diseases, and a growing early-stage pipeline that expands cell and gene therapy targets, and technologies. We are developing cancer treatments directed at key biological pathways using our protein homeostasis platform, a research capability that has been the basis of our approved therapies for multiple myeloma and several promising compounds in early to mid-stage development. Our scientists are targeting different immune system pathways to address interactions between tumors, the microenvironment and the immune system to further expand upon the progress we have made and help more patients respond to treatment. Combining these approaches is key to delivering potential new options for the treatment of cancer and addressing the growing issue of resistance to immunotherapy. We source innovation internally, and in collaboration with academia, government, advocacy groups and biotechnology companies, to help make the promise of transformational medicines a reality for patients.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Celgene and Juno Therapeutics are wholly owned subsidiaries of Bristol-Myers Squibb Company. In certain countries outside the U.S., due to local laws, Celgene and Juno Therapeutics are referred to as, Celgene, a Bristol Myers Squibb company and Juno Therapeutics, a Bristol Myers Squibb company.
About Acceleron
Acceleron is a biopharmaceutical company dedicated to the discovery, development, and commercialization of therapeutics to treat serious and rare diseases. Acceleron's leadership in the understanding of TGF-beta superfamily biology and protein engineering generates innovative compounds that engage the body's ability to regulate cellular growth and repair.
Acceleron focuses its commercialization, research, and development efforts in hematologic and pulmonary diseases. In hematology, Acceleron and its global collaboration partner, Bristol Myers Squibb, are co-promoting REBLOZYL (luspatercept-aamt), the first and only approved erythroid maturation agent, in the United States for the treatment of anemia in certain blood disorders. The Companies are also developing luspatercept for the treatment of chronic anemia in patient populations of MDS, beta-thalassemia, and myelofibrosis. In pulmonary, Acceleron is developing sotatercept for the treatment of pulmonary arterial hypertension, having recently reported positive topline results of the Phase 2 PULSAR trial.
For more information, please visit http://www.acceleronpharma.com. Follow Acceleron on Social Media: @AcceleronPharma and LinkedIn.
SOURCE: Bristol-Myers Squibb
Here is the original post:
European Commission Approves Reblozyl (luspatercept) for the Treatment of Transfusion-Dependent Anemia in Adult Patients with Myelodysplastic...
- Patient Dies of Acute Liver Failure After Treatment With Sareptas DMD Gene Therapy Elevidys - CGTLive - March 19th, 2025
- Patient dies following muscular dystrophy gene therapy, Sarepta reports - The Associated Press - March 19th, 2025
- Duchenne patient dies after receiving Sarepta gene therapy - March 19th, 2025
- Liver Failure-Associated Death Reported in Patient Treated With Sarepta Gene Therapy Elevidys - MedCity News - March 19th, 2025
- DoD grant funds Hollings researcher's idea to pursue gene therapy for cancer - Medical University of South Carolina - March 19th, 2025
- Recon: Sarepta reports death of teen who received Duchenne gene therapy; Novartis to slash 427 jobs while revamping cardiovascular business -... - March 19th, 2025
- Data Gaps Leave Long-Term Impact of Ex Vivo Gene Therapy in DMD Uncertain - AJMC.com Managed Markets Network - March 19th, 2025
- CHO Plus Obtains U.S. Patent for Improved Production of Viral Vectors for Gene Therapy - Business Wire - March 19th, 2025
- Sarepta Shares Fall on Report of Patient Death After Gene Therapy - Bloomberg - March 19th, 2025
- Hologen AI commits up to $430M to help take MeiraGTx's Parkinson's gene therapy through phase 3 and beyond - Fierce Biotech - March 19th, 2025
- Duchenne patient on Sareptas gene therapy dies - The Business Journals - March 19th, 2025
- Im Unstoppable: New gene therapy cures first New Yorker of sickle cell anemia - PIX11 New York News - March 19th, 2025
- Boost in cancer treatment: PGI working on lab for stem cell, gene therapies - The Times of India - March 19th, 2025
- Man Cured Of Sickle Cell Disease In New York Thanks To New Gene Therapy - Forbes - March 19th, 2025
- Sarepta says teen died after its gene therapy treatment By Reuters - Investing.com - March 19th, 2025
- Innovative Gene Therapy Approach Drives Buy Rating for Insmed in DMD Treatment - TipRanks - March 19th, 2025
- Sarepta says patient dies after treatment with gene therapy - TradingView - March 19th, 2025
- Sarepta tumbles after patient dies following gene therapy treatment - TradingView - March 19th, 2025
- MeiraGTx teams with cryptic AI startup, co-founded by Eric Schmidt, to advance Parkinson's gene therapy - Endpoints News - March 19th, 2025
- Sickle cell anemia patient reunites with Long Island doctors whose gene therapy treatments made him symptom-free - Newsday - March 19th, 2025
- Extracellular vesicles for the delivery of gene therapy - Nature.com - March 9th, 2025
- Around the Helix: Cell and Gene Therapy Company Updates March 5, 2025 - CGTLive - March 9th, 2025
- Inside the secret island where wealthy people go to alter their DNA - Daily Mail - March 9th, 2025
- Regenerons Gene Therapy DB-OTO Trial Shows Promising Hearing Improvement - The Hearing Review - March 9th, 2025
- Global Cell and Gene Therapy Manufacturing Market to Reach ~USD 10 Billion by 2032 | DelveInsight - GlobeNewswire - March 9th, 2025
- College Station gene therapy company partners with nonprofit to develop treatments for rare diseases - KBTX - March 9th, 2025
- World Hearing Day 2025: Looking Back at Progress in Gene Therapy - CGTLive - March 9th, 2025
- Reflecting on a milestone year for cell and gene therapies - Pharmaceutical Technology - March 9th, 2025
- Q&A: Whats Next for Hemophilia Gene Therapy? | Newswise - Newswise - March 9th, 2025
- 'Llife-changing' gene therapy in London partially restores CT child's sight - CT Insider - March 9th, 2025
- The Genesis of Cell Therapy: Bridging Traditional Pharmacology and Gene Therapy - Technology Networks - March 9th, 2025
- Regenxbio at TD Cowen Conference: Gene Therapy Advancements - Investing.com - March 9th, 2025
- Anova Announces First Patient Enrolled to Phase 1/2a Study of DB107 for the Treatment of High-Grade Gliomas - Business Wire - March 9th, 2025
- Apertura Gene Therapy Supports the Broad Institute in Development of Gene Therapy for Prion Disease Using Engineered AAV Capsid Targeting TfR1 for CNS... - March 9th, 2025
- Gene therapy research offers hope for people with chronic kidney disease - Medical Xpress - January 6th, 2025
- Sangamo Therapeutics to Regain Full Rights to Hemophilia A Gene Therapy Program Following Pfizers Decision to Cease Development of Giroctocogene... - January 6th, 2025
- JCR Pharmaceuticals and Modalis Therapeutics Announce Transition to the Next Phase of Joint Research Agreement for Development of Novel Gene Therapy -... - January 6th, 2025
- Gene therapy targets the retina to treat eye disease - Nature.com - January 6th, 2025
- Sangamos Stock Plummets as Pfizer Axes Hemophilia Gene Therapy Pact - BioSpace - January 6th, 2025
- How Increased Use of Gene Therapy Treatment for Sickle Cell Disease Could Affect the Federal Budget - Congressional Budget Office - January 6th, 2025
- The Future of Regulatory Processes in Cell and Gene Therapy - Pharmaceutical Executive - January 6th, 2025
- CGTLive's 2024 Pillars of Progress: Most-Watched Conference Interviews - CGTLive - January 6th, 2025
- Pfizer cuts losses on near-approval hemophilia gene therapy, adding to troubled Sangamo's woes - Fierce Biotech - January 6th, 2025
- JCR Pharmaceuticals and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- JCR and Modalis Advance Joint Gene Therapy Research - TipRanks - January 6th, 2025
- Novartis Gene Therapy Shows Promise in Treating SMA - Yahoo Finance - January 6th, 2025
- Gene Therapy Market to Hit Valuation of US$ 42.26 Billion By 2033 | Astute Analytica - Yahoo Finance - January 6th, 2025
- Novartis gene therapy helps children with rare muscle disorder in study - Reuters - January 6th, 2025
- Capricor Puts Rolling BLA for DMD Cardiomyopathy Cell Therapy Deramiocel in Front of the FDA - CGTLive - January 6th, 2025
- Positive data could expand use of Novartis gene therapy for SMA - Yahoo Finance - January 6th, 2025
- Sangamo spirals after Pfizer halts hemophilia A gene therapy partnership - MM+M Online - January 6th, 2025
- Cell Therapy and Gene Therapy CDMO Market to Reach USD 11.11 Billion by 2030 | Discover Growth Trends and Insights | Valuates Reports - PR Newswire - January 6th, 2025
- Struggling With Adoption, Sickle Cell Gene Therapy Manufacturers Embrace CMS Model - News & Insights - January 6th, 2025
- Sangamo Therapeutics to Regain Rights to Gene Therapy Program from Pfizer - Contract Pharma - January 6th, 2025
- Researchers Create Gene Therapy with Potential to Treat Peripheral Pain ... - December 28th, 2024
- How CRISPR Is Changing Cancer Research and Treatment - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - December 28th, 2024
- 100 cell and gene therapy leaders to watch in 2025 - December 28th, 2024
- Can a new gene therapy reverse heart failure? - Futurity - December 28th, 2024
- Sustained visual improvements in LHON patients treated with AAV gene therapy - Medical Xpress - December 28th, 2024
- Nebraska Medicine administers novel gene therapy to first hemophilia ... - December 28th, 2024
- Gene Therapy for Cardiomyopathies Presents Promising Alternative to Current Treatment - Managed Healthcare Executive - December 28th, 2024
- Stem Cell Transplantation Still the Main Treatment Option for Beta-Thalassemia - Medpage Today - December 28th, 2024
- Caribou Overhyped Gene-Therapy Testing, Investor Class Suit Says - Bloomberg Law - December 28th, 2024
- WuXi AppTec sells off cell and gene therapy operations in US, UK - FirstWord Pharma - December 28th, 2024
- Top 5 Print Publication Articles of 2024 - Managed Healthcare Executive - December 28th, 2024
- Gene Therapy Shows Long-Term Vision Benefits in Rare Eye Disease - Medpage Today - December 28th, 2024
- UPenn gene therapy pioneers biotech gets $34 million in funding - The Philadelphia Inquirer - December 28th, 2024
- PHC Corporation to present LiCellGrow at Advanced Therapies Week 2025 - Drug Target Review - December 28th, 2024
- The Evolution of Cell & Gene Therapy: Development and Manufacturing Insights and the Role of CDMOs - Pharmaceutical Technology Magazine - December 28th, 2024
- Pig kidney transplants, new schizophrenia drug: Here are 5 of the biggest medical breakthroughs in 2024 - ABC News - December 28th, 2024
- Cell Therapy Manufacturing Trends And Advancements Continuing In 2025 - BioProcess Online - December 28th, 2024
- Can Gene Therapy Treat Chronic Pain? - LabRoots - December 28th, 2024
- Driving innovation: India's foray into gene and cell therapies - The Economic Times - December 28th, 2024
- Governor Hochul Celebrates the Opening Of New York's First Cell and Gene Therapy Hub at Roswell Park Comprehensive Cancer Center in Buffalo - PR Web - December 19th, 2024
- GenSight Biologics Provides Update on Regulatory Discussions and Financial Situation - Business Wire - December 19th, 2024
- Atsena completes dosing in part A of X-linked retinoschisis gene therapy trial - Healio - December 19th, 2024
- Astellas and Sangamo Therapeutics Announce Capsid License Agreement to Deliver Genomic Medicines for Neurological Diseases - StreetInsider.com - December 19th, 2024
- Ring Therapeutics lays off just under half of staff in 2nd wave of cuts this year, CEO set to step down - Fierce Biotech - December 19th, 2024
- Gov. Hochul celebrates opening of first cell and gene therapy hub in NYS - WIVB.com - News 4 - December 19th, 2024