Luspatercept-aamt Approved to Treat Anemia in Beta Thalassemia

The FDA approved luspatercept-aamt for the treatment of anemia in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.

The approval decision was based on efficacy results from the multicenter, randomized, double-blind, placebo-controlled phase III BELIEVE trial, which enrolled 336 adults with transfusion-dependent beta thalassemia. Participants were randomized 2:1 to receive luspatercept-aamt (administered subcutaneously once every 3 weeks) or placebo.

After 24 weeks of treatment, 48 of 224 patients (21.4%) in the luspatercept-aamt arm achieved the primary endpoint of RBC transfusion reduction (defined as a ≥33% reduction in RBC transfusion burden, with a reduction of ≥2 RBC units from baseline, during weeks 13-24). In the placebo group, 4.5% of participants achieved the primary endpoint (risk difference = 17.0; 95% CI 10.4-23.6; p<0.0001).

The most common adverse events (AEs; occurring in >10% of patients) included headache, bone pain, arthralgia, fatigue, cough, abdominal pain, diarrhea, and dizziness.

Luspatercept-aamt was approved through the FDA’s fast-track program and with orphan-drug designation.

Source: FDA news release, November 8, 2019.