FDAnews Drug Daily Bulletin
Pharmaceuticals / Regulatory Affairs

Reblozyl Earns FDA’s First Approval for Rare Blood Disorder

Nov. 20, 2019

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.

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