FDA Misses PDUFA Date for BMS’ Reblozyl for Rare Blood Disorder

The FDA has extended the review of the supplemental biologics license application (sBLA) for Reblozyl (luspatercept-aamt) for the treatment of anemia in adults with non-transfusion-dependent (NTD) beta thalassemia to June 27, 2022.

The regulatory agency has extended the Prescription Drug User Fee Act (PDUFA) goal date by three months to provide time for a full review of the submission. The sBLA was based on safety and efficacy results from the pivotal phase 2 BEYOND study evaluating Reblozyl plus best supportive care in adults with NTD beta thalassemia.

Beta thalassemia is a blood disorder where low levels of hemoglobin lead to lack of oxygen throughout the body. It is caused by mutations in the HBB gene. It is relatively rare in the United States, but is one of the most common autosomal recessive disorders. The disorder is prevalent in the Mediterranean, Middle East, Africa, central Asia, the Indian subcontinent, and the Far East, according to the National Organization for Rare Disorders.

Reblozyl, is a first-in-class therapeutic that is currently approved in the United States and European Union to address transfusion-dependent anemia-associated beta thalassemia. It is being developed and commercialized through a collaboration between Bristol Myers Squibb and Merck, following Merck’s acquisition of Acceleron Pharma in November 2021.