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FDA Accepts for Priority Review Supplemental Application of Reblozyl


The FDA is reviewing Reblozy to treat anemia in patients with beta thalassemia. The PDUFA action date is March 27, 2022.

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 goal date of March 27, 2022.

In addition, the European Medicines Agency has validated the Type II variation for Reblozyl inNTD beta thalassemia. Reblozyl is being co-developed and co-commercialized by Bristol Myers Squibb and Merck, following Merck’s recent acquisition of Acceleron Pharma.

Noah Berkowitz, M.D., Ph.D.

Noah Berkowitz, M.D., Ph.D.

“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,” Noah Berkowitz, M.D., Ph.D., senior vice president, hematology development at Bristol Myers Squibb said in a statement.

The applications are based on the pivotal phase 2 BEYOND study. Updated analyses from the will be presented at the American Society of Hematology Annual Meeting from December 11-14.

Reblozyl is currently indicated to treat anemia in adult patients with beta thalassemia who require regular red blood cell transfusions, and anemia in those with myelodysplastic syndrome with ring sideroblasts or with myelodysplastic/myeloproliferative neoplasm with ring sideroblasts and thrombocytosis

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.

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