The U.S. Food and Drug Administration (FDA) approved Celgene and Acceleron Pharma’s Reblozyl for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions. The approval marks the first approved treatment in the United States for this condition.
The approval also marks the first for Acceleron, which sent shares up nearly 7% in afternoon trading. Reblozyl, an erythroid maturation agent, was approved under Fast Track Designation and the drug received Orphan Drug designation.
With the approval, Reblozyl (luspatercept-aamt) becomes a new therapeutic class for beta thalassemia patients. Reblozyl works by regulating late-stage red blood cell maturation to help patients reduce their RBC transfusion burden. The drug is not indicated for use as a substitute for RBC transfusions in patients who require immediate correction of anemia, Celgene stressed in its Friday afternoon announcement.
