Celgene and its developmental partner Acceleron Pharma are eying a potential U.S. Food and Drug Administration approval of a blood-disease treatment.
Celgene and its developmental partner Acceleron Pharma are eying a potential U.S. Food and Drug Administration (FDA) approval of a blood-disease treatment.
Late Friday, the two companies announced they had filed 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 companies filed the BLA based on the positive results from Phase III studies MEDALIST and BELIEVE.
When Bristol-Myers Squibb struck its $74 billion deal to acquire Celgene earlier this year, luspatercept was identified as one of the key assets that will drive future revenues for the combined companies.
In the Phase III MEDALIST trial, luspatercept achieved a highly statistically significant improvement in the primary endpoint of red blood cell (RBC) transfusion independence of at least eight consecutive weeks of treatment during the first 24 weeks of treatment compared to placebo. Not only did luspatercept hit its primary endpoint in the Phase III trial, but luspatercept also met the key secondary endpoint of demonstrating a highly statistically significant improvement in RBC transfusion independence of at least 12 consecutive weeks during the first 24 weeks.
In the BELIEVE trial, luspatercept and best supportive care also hit the mark. The data showed that luspatercept achieved a highly statistically significant improvement in the primary endpoint of erythroid response, which was defined as at least a 33 percent reduction from baseline in red blood cell transfusion burden with a reduction of at least two units during the protocol-defined period of 12 consecutive weeks, from week 13 to week 24, compared to placebo.
Jay Backstrom, Celgene’s chief medical officer, said there is a high unmet need for patients with MDS or beta-thalassemia who suffer from the effects of their disease-related anemia. He said new treatment options are needed for these patients.
“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,” Backstrom said in a statement. With this submission, we look forward to working with the agency to deliver luspatercept to patients with these serious blood diseases.”
Luspatercept is a first-in-class erythroid maturation agent (EMA) that regulates late-stage red blood cell maturation.
Habib Dable, President and chief executive officer of Cambridge, Mass.-based Acceleron, said called the BLA a key milestone for the company. Dable said they 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. Dable said that everyone involved with the research has worked diligently to develop luspatercept for patients with chronic anemias associated with these serious blood disorders.”
In addition to seeing regulatory approval in the U.S., the companies also plan to submit a marketing application to the European Medicines Agency in the second quarter of 2019.
