BLA Accepted for Celgene/Acceleron’s Luspatercept

FDA has accepted for review a Celgene and Acceleron BLA for luspatercept, an investigational erythroid maturation agent for treating adult patients with very low to intermediate-risk myelodysplastic syndromes (MDS)-associated anemia who have ring sideroblasts and require red blood cell (RBC) transfusions, and for treating adult patients with beta-thalassemia-associated anemia who require RBC transfusions. The FDA has granted a priority review and set a 12/4 user fee review action target for the beta-thalassemia indication. The agency also set a 4/4/2020 user fee review action for the MDS indication.

The submission is based on safety and efficacy data from the pivotal Phase 3 studies MEDALIST and BELIEVE, which evaluated the ability of luspatercept to effectively treat the anemia associated with MDS and beta-thalassemia, respectively. Luspatercept is a first-in-class erythroid maturation agent that regulates late-stage red blood cell maturation.