The U.S. FDA has expanded approval for Agios Pharmaceuticals’ oral pyruvate kinase activator, mitapivat, to include the treatment of anemia in adults with alpha- or beta-thalassemia.
Mitapivat was first approved in 2022 as a treatment for hemolytic anemia in adults with pyruvate kinase deficiency, a rare inherited form of anemia, under the brand name Pyrukynd. The expanded approval, under the brand name Aqvesme, makes the drug the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.
In the ENERGIZE and ENERGIZE-T phase 3 trials, five patients receiving Aqvesme experienced adverse reactions suggestive of hepatocellular injury (HCI), with two of these patients requiring hospitalization. To mitigate the risk of HCI, the Aqvesme REMS program requires liver tests prior to the first dose, every four weeks thereafter for 24 weeks, and then as clinically indicated.
The sNDA approval came after significant delays. The PDUFA goal date was first extended by three months, from September 7, 2025 to December 7, 2025, after Agios submitted a proposed REMS to mitigate the risk of hepatocellular injury described in the original sNDA, which constituted a major amendment to the application. Agios then revealed earlier this month that the sNDA was still under “active review” and that the agency would miss its December 7 PDUFA goal date.