The Food and Drug Administration (FDA) has approved Reblozyl (luspatercept–aamt; Celgene and Acceleron) for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions.

Reblozyl, a recombinant fusion protein, is an erythroid maturation agent that promotes late-stage red blood cell (RBC) maturation to help reduce RBC transfusion burden. The approval was based on data from a phase 3 BELIEVE trial (N=336) that evaluated the safety and efficacy of luspatercept plus best supportive care (BSC; RBC transfusions, iron-chelating agents, use of antibiotic, antiviral, and antifungal therapy; and/or nutritional support as needed) vs placebo plus BSC in adults who require regular RBC transfusion (defined as 6 to 20 RBC units in the 24 weeks prior to randomization and no transfusion-free period for >35 days during that period) due to beta thalassemia. The primary end point was the proportion of patients with hematological improvement from Week 13 to 24 after randomization.

Results showed a clinically meaningful and statistically significant improvement with luspatercept, with 21.4% of luspatercept-treated patients achieving hematological improvement (defined as ≥33% reduction from baseline in RBC transfusion burden with a reduction of at least 2 units) compared with 4.5% of patients in the placebo group (risk difference: 17.0; 95% CI: 10.4, 23.6; P<.0001). As for safety, the most common treatment-emergent adverse reactions included headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea and dizziness.

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“When patients receive multiple blood transfusions, there is a risk for iron overload, which can affect many organs,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research. “Today’s approval provides patients with a therapy that, for the first time, will help decrease the number of blood transfusions.”

Reblozyl is supplied as 25mg and 75mg lyophilized powder in single-dose vials for reconstitution and subcutaneous administration. The product is expected to be available in 1 week.

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