Submission based on results from Phase 2 BEYOND study of Reblozyl plus best supportive care in adults with NTD beta thalassemia
PRINCETON, NJ, USA I December 03, 2021 I Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review the supplemental Biologics License Application (sBLA) for Reblozyl® (luspatercept-aamt), a first-in-class erythroid maturation agent, for the treatment of anemia in adults with non-transfusion dependent (NTD) beta thalassemia. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition, the European Medicines Agency has validated the Type II variation for Reblozyl in NTD beta thalassemia. Reblozyl is being co-developed and co-commercialized with Merck & Co., Inc., known as MSD outside the United States and Canada, following Merck’s recent acquisition of Acceleron Pharma, Inc.
These applications were based on safety and efficacy results from the pivotal Phase 2 BEYOND study evaluating Reblozyl plus best supportive care in patients with NTD beta thalassemia.
“Patients with non-transfusion dependent beta thalassemia may not require lifelong blood transfusions for survival, but their need for effective treatment options is significant as they face a range of clinical complications due to chronic anemia and iron overload,” said Noah Berkowitz, M.D., Ph.D., senior vice president, Hematology Development, Bristol Myers Squibb. “Reblozyl is an important therapy approved for anemia associated with beta thalassemia and lower-risk myelodysplastic syndromes in multiple countries, including the United States and within the European Union. Along with our partners at Merck, we are committed to continuing to advance our clinical program for Reblozyl and look forward to working with the FDA during its review of our application for this underserved patient population.”
Updated analyses from the BEYOND study will be presented at the 63rd American Society of Hematology Annual Meeting and Exposition from December 11-14.
About BEYOND
BEYOND (NCT03342404) is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept-aamt (ACE-536) versus placebo in adults with non-transfusion dependent beta thalassemia. The study is divided into the Screening Period, Double-blind Treatment Period (DBTP) and Post-Treatment Follow-up Period (PTFP) and randomized 145 subjects at a 2:1 ratio of Reblozyl versus placebo. The primary endpoint of the study is the proportion of subjects who have an increase from baseline ≥1.0 g/dL in mean of hemoglobin values over a continuous 12-week interval from Week 13 to Week 24 of treatment in the absence of transfusions. Key secondary endpoints include mean change in non-transfusion dependent beta thalassemia-patient reported outcome (NTDT-PRO) Tiredness and Weakness (TW) domain score and baseline hemoglobin (Hb).
About Beta Thalassemia
Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. It is one of the most common autosomal recessive disorders, and the total annual incidence of symptomatic individuals is estimated at 1 in 100,000 people globally.1 The disease is associated with ineffective erythropoiesis, which results in the production of fewer and less healthy red blood cells (RBCs), often leading to severe anemia—a condition that can be debilitating and can lead to other complications for patients—as 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.1 Non-transfusion dependent thalassemia is a term used to describe patients who do not require lifelong regular transfusions for survival, although they may experience a range of clinical complications and require occasional or even frequent transfusions, usually for defined periods of time.2
About Reblozyl®
Reblozyl, the first and only erythroid maturation agent, promotes late-stage red blood cell maturation in animal models.1 Bristol Myers Squibb and Merck & Co., Inc., through Merck’s acquisition of Acceleron, are jointly developing Reblozyl as part of a global collaboration. Reblozyl is currently approved in the U.S. for the treatment of:
- anemia in adult patients with beta thalassemia who require regular red blood cell transfusions, and
- anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndrome with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.
Please see full Prescribing Information for REBLOZYL
Bristol Myers Squibb: Creating a Better Future for People with Cancer
Bristol Myers Squibb is inspired by a single vision — transforming people’s lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to look at cancer from every angle. Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.
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.
Juno Therapeutics, Inc. is a wholly owned subsidiary 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.
References:
- Galanello R, Origa R. Beta thalassemia. Orphanet Journal of Rare Diseases. 2010;5(11). Available at: https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-5-11. Accessed November 2021.
- Musallam, K. M., Rivella, S., Vichinsky, E., & Rachmilewitz, E. A. (2013). Non-transfusion-dependent thalassemias. Haematologica, 98(6), 833–844. https://doi.org/10.3324/haematol.2012.066845. Accessed November 2021.
SOURCE: Bristol Myers Squibb
Post Views: 27
Submission based on results from Phase 2 BEYOND study of Reblozyl plus best supportive care in adults with NTD beta thalassemia
PRINCETON, NJ, USA I December 03, 2021 I Bristol Myers Squibb (NYSE: BMY) today announced that the U.S. Food and Drug Administration (FDA) has accepted for priority review the supplemental Biologics License Application (sBLA) for Reblozyl® (luspatercept-aamt), a first-in-class erythroid maturation agent, for the treatment of anemia in adults with non-transfusion dependent (NTD) beta thalassemia. The FDA has set a Prescription Drug User Fee Act (PDUFA) goal date of March 27, 2022. In addition, the European Medicines Agency has validated the Type II variation for Reblozyl in NTD beta thalassemia. Reblozyl is being co-developed and co-commercialized with Merck & Co., Inc., known as MSD outside the United States and Canada, following Merck’s recent acquisition of Acceleron Pharma, Inc.
These applications were based on safety and efficacy results from the pivotal Phase 2 BEYOND study evaluating Reblozyl plus best supportive care in patients with NTD beta thalassemia.
“Patients with non-transfusion dependent beta thalassemia may not require lifelong blood transfusions for survival, but their need for effective treatment options is significant as they face a range of clinical complications due to chronic anemia and iron overload,” said Noah Berkowitz, M.D., Ph.D., senior vice president, Hematology Development, Bristol Myers Squibb. “Reblozyl is an important therapy approved for anemia associated with beta thalassemia and lower-risk myelodysplastic syndromes in multiple countries, including the United States and within the European Union. Along with our partners at Merck, we are committed to continuing to advance our clinical program for Reblozyl and look forward to working with the FDA during its review of our application for this underserved patient population.”
Updated analyses from the BEYOND study will be presented at the 63rd American Society of Hematology Annual Meeting and Exposition from December 11-14.
About BEYOND
BEYOND (NCT03342404) is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept-aamt (ACE-536) versus placebo in adults with non-transfusion dependent beta thalassemia. The study is divided into the Screening Period, Double-blind Treatment Period (DBTP) and Post-Treatment Follow-up Period (PTFP) and randomized 145 subjects at a 2:1 ratio of Reblozyl versus placebo. The primary endpoint of the study is the proportion of subjects who have an increase from baseline ≥1.0 g/dL in mean of hemoglobin values over a continuous 12-week interval from Week 13 to Week 24 of treatment in the absence of transfusions. Key secondary endpoints include mean change in non-transfusion dependent beta thalassemia-patient reported outcome (NTDT-PRO) Tiredness and Weakness (TW) domain score and baseline hemoglobin (Hb).
About Beta Thalassemia
Beta thalassemia is an inherited blood disorder caused by a genetic defect in hemoglobin. It is one of the most common autosomal recessive disorders, and the total annual incidence of symptomatic individuals is estimated at 1 in 100,000 people globally.1 The disease is associated with ineffective erythropoiesis, which results in the production of fewer and less healthy red blood cells (RBCs), often leading to severe anemia—a condition that can be debilitating and can lead to other complications for patients—as 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.1 Non-transfusion dependent thalassemia is a term used to describe patients who do not require lifelong regular transfusions for survival, although they may experience a range of clinical complications and require occasional or even frequent transfusions, usually for defined periods of time.2
About Reblozyl®
Reblozyl, the first and only erythroid maturation agent, promotes late-stage red blood cell maturation in animal models.1 Bristol Myers Squibb and Merck & Co., Inc., through Merck’s acquisition of Acceleron, are jointly developing Reblozyl as part of a global collaboration. Reblozyl is currently approved in the U.S. for the treatment of:
- anemia in adult patients with beta thalassemia who require regular red blood cell transfusions, and
- anemia failing an erythropoiesis stimulating agent and requiring 2 or more red blood cell units over 8 weeks in adult patients with very low- to intermediate-risk myelodysplastic syndrome with ring sideroblasts (MDS-RS) or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis (MDS/MPN-RS-T).
Reblozyl is not indicated for use as a substitute for red blood cell transfusions in patients who require immediate correction of anemia.
Please see full Prescribing Information for REBLOZYL
Bristol Myers Squibb: Creating a Better Future for People with Cancer
Bristol Myers Squibb is inspired by a single vision — transforming people’s lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to look at cancer from every angle. Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.
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.
Juno Therapeutics, Inc. is a wholly owned subsidiary 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.
References:
- Galanello R, Origa R. Beta thalassemia. Orphanet Journal of Rare Diseases. 2010;5(11). Available at: https://ojrd.biomedcentral.com/articles/10.1186/1750-1172-5-11. Accessed November 2021.
- Musallam, K. M., Rivella, S., Vichinsky, E., & Rachmilewitz, E. A. (2013). Non-transfusion-dependent thalassemias. Haematologica, 98(6), 833–844. https://doi.org/10.3324/haematol.2012.066845. Accessed November 2021.
SOURCE: Bristol Myers Squibb
Post Views: 27
