The FDA approved mitapivat for thalassemia anemia in adults, improving hemoglobin and reducing transfusions.

The U.S. FDA has approved Agios Pharmaceuticals’ AQVESME (mitapivat) for treating anemia in adults with alpha- or beta-thalassemia, the first drug approved for both non-transfusion-dependent and transfusion-dependent forms. Based on Phase 3 trial results, the oral pyruvate kinase activator improved hemoglobin levels, reduced transfusion needs, and enhanced quality of life. Availability is expected in late January 2026, following implementation of a REMS program to monitor liver safety due to rare cases of hepatocellular injury.