On November 8, the U.S. Food and Drug Administration (FDA) approved luspatercept-aamt (Reblozyl) for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell transfusions.
Beta thalassemia is a blood disorder that reduces the production of hemoglobin. In people with beta thalassemia, low levels of hemoglobin lead to a lack of oxygen in many parts of the body. Affected individuals also have anemia, which can cause pale skin, weakness, fatigue, and more serious complications. People with beta thalassemia are at an increased risk of developing abnormal blood clots.
Efficacy was evaluated in the BELIEVE trial, a multicenter, randomized, double-blind, placebo-controlled trial which enrolled 336 adult patients with beta thalassemia requiring regular red blood cell transfusions. Patients were randomized 2:1 to luspatercept-aamt (n = 224) or placebo (n = 112). Luspatercept-aamt was administered subcutaneously once every 3 weeks as long as a reduction in transfusion requirement was observed or until unacceptable toxicity. The primary efficacy outcome measure was the proportion of patients achieving regular red blood cell transfusion burden reduction from baseline of at least 33%, with a reduction of at least two units from week 13 to week 24.
Of the patients who received luspatercept-aamt, 21.4% achieved the primary endpoint compared with 4.5% of those who received placebo (risk difference = 17.0; 95% confidence interval = 10.4–23.6, P < .0001).
The most common adverse reactions (> 10%) were headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea, and dizziness.
The recommended starting dose is 1 mg/kg once every 3 weeks by subcutaneous injection. View the full prescribing information for luspatercept-aamt.