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FDA accepts for priority review sBLA for Reblozyl in adults with non-transfusion dependent (NTD) beta thalassemia. BMS + Merck Inc.,

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Last updated:20th Dec 2021
Published:5th Dec 2021
Bristol Myers Squibb announced that the 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., following Merck’s recent acquisition of Acceleron Pharma, Inc. These applications were based on safety and efficacy results from the pivotal Phase II BEYOND study evaluating Reblozyl plus best supportive care in patients with NTD beta thalassemia.

Reblozyl is currently approved in the U.S. for the treatment of: i. anemia in adult patients with beta thalassemia who require regular red blood cell transfusions, and 2. 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.

Condition: Beta-thalassemia
Type: drug

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