Celgene and Acceleron Announce FDA Accepts Luspatercept Biologics License Application in Myelodysplastic Syndromes and Beta-Thalassemia

Celgene Corporation and Acceleron Pharma Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted Celgene’s Biologics License Application (BLA) for luspatercept, an investigational 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 FDA has granted Priority Review to this BLA for the evaluation of the beta-thalassemia indication and set a Prescription Drug User Fee Act (PDUFA), or target action, date of December 4, 2019. The FDA has also set a PDUFA date of April 4, 2020 for the evaluation of the MDS indication.

“The acceptance of the luspatercept filings and granting of the U.S. priority review for beta-thalassemia represent another important step in delivering this novel therapy to patients in need,” said Jay Backstrom, M.D., Chief Medical Officer for Celgene. “We believe that luspatercept can play a critical role in treating the anemia associated with these serious blood diseases, and with these milestones achieved we look forward to working closely with the agency to move this therapy toward approval.”

The safety and efficacy results provided in the application are 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. MEDALIST and BELIEVE were both presented at the 2018 American Society of Hematology annual meeting, where MEDALIST was included in the plenary session.

The companies also announced that Celgene’s Marketing Authorization Application in the EU has been successfully validated and the review is now underway.

“The ongoing U.S. and European regulatory reviews of the luspatercept filings in MDS and beta-thalassemia strongly support our primary goal, which has always been to bring a potentially transformative new treatment to these patients with unmet clinical need as quickly as possible,” said Habib Dable, President and Chief Executive Officer of Acceleron. “At the same time, we continue to explore the ability of luspatercept to address anemia in additional settings, including patients with treatment-naïve MDS, non-transfusion-dependent beta-thalassemia, and myelofibrosis.”

Luspatercept is an investigational therapy that is not approved for any use in any country.