Celgene and Acceleron’s Reblozyl (luspatercept-aamt) earned FDA approval for treatment of anemia in adults with beta thalassemia who require regular red blood cell transfusions.
The agency granted the approval based on the results of a clinical trial of 336 patients with beta thalassemia who required transfusions. Twenty-one percent of the patients who received Reblozyl achieved at least a 33 percent reduction in transfusions compared to 4.5 percent of the patients who received a placebo.
Reblozyl is the first FDA-approved treatment for Cooley’s anemia. It was previously granted both fast track and orphan drug designations.