Celgene Corporation (NASDAQ: CELG) and Acceleron Pharma Inc. (NASDAQ: XLRN) announced on 4/5/19 that Celgene has submitted a Biologics License Application (BLA) for luspatercept, an erythroid maturation agent, for the treatment of 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 the treatment of adult patients with beta-thalassemia-associated anemia who require RBC transfusions.
The submission is based on the safety and efficacy results of the pivotal phase 3 studies MEDALIST and BELIEVE, both recently presented at the American Society of Hematology annual meeting, where MEDALIST was included in the plenary session.
“There remains a high unmet medical need for patients with MDS or beta-thalassemia who suffer from the effects of their disease-related anemia. The primary treatment option for these patients currently is chronic transfusion of red blood cells which can be associated with complications such as iron overload,” said Jay Backstrom, M.D., Chief Medical Officer for Celgene. “New treatment options are urgently needed for these patients. With this submission, we look forward to working with the Agency to deliver luspatercept to patients with these serious blood diseases.”
The companies also plan to submit a marketing application to the European Medicines Agency in the second quarter of 2019.
Luspatercept is a first-in-class erythroid maturation agent (EMA) that regulates late-stage red blood cell maturation. Acceleron and Celgene are jointly developing luspatercept as part of a global collaboration. A phase 3 trial (COMMANDS) in ESA-naïve, lower-risk MDS patients, the BEYOND phase 2 trial in non-transfusion-dependent beta-thalassemia, and a phase 2 trial in myelofibrosis are ongoing.