The Biologics License Application has been submitted to the U.S. FDA. The erythroid maturation agent, luspatercept is expected to be used for the treatment of adult patients with myelodysplastic syndromes and beta-thalassemia.
Massachusetts/USA – Celgene Corporation and Acceleron Pharma have recently announced that Celgene has submitted a Biologics License Application (BLA) for luspatercept, an erythroid maturation agent. It will be used for the treatment of adult patients with very low to intermediate risk myelodysplastic syndromes (MDS)-associated anemia that have ring sideroblasts and require red blood cell (RBC) transfusions. It will also be used for the treatment of adult patients with beta-thalassemia-associated anemia that 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.
“The BLA submission is a key milestone for Acceleron and a credit to our longstanding collaboration with Celgene,” said Habib Dable, President and Chief Executive Officer of Acceleron. “We believe luspatercept’s positive clinical trial results demonstrate its potential as a novel treatment for patients with lower-risk MDS as well as in beta-thalassemia. All involved have worked diligently to develop luspatercept for patients with chronic anemias associated with these serious blood disorders.”
Luspatercept is an investigational therapy that is not approved for any use in any country for any indication.