FDA approves Agios Pharmaceuticals’ Aqvesme in anemia patients

Agios Pharmaceuticals (AGIO) “announced that the U.S. Food and Drug Administration has approved AQVESME, or mitapivat, an oral pyruvate kinase activator, for the treatment of anemia in adults with alpha- or beta-thalassemia. With this approved indication, AQVESME becomes the only FDA-approved medicine for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The FDA approval of AQVESME in thalassemia is based on results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively. A total of 452 patients who are representative of the real-world thalassemia population were enrolled in the trials. The ENERGIZE and ENERGIZE-T Phase 3 trials met all primary and key secondary efficacy endpoints, demonstrating that AQVESME improves hemolytic anemia and a key quality-of-life measure compared to placebo, including significant reductions in transfusion burden and significant improvements in hemoglobin and fatigue. Due to the AQVESME REMS program, mitapivat will be marketed under the brand name AQVESME in the U.S. for the thalassemia indication. Mitapivat will continue to be marketed as PYRUKYND(R) (mitapivat) in the U.S. for the PK deficiency indication, which does not require a REMS program. Outside the U.S., mitapivat will continue to be marketed as PYRUKYND for its PK deficiency and thalassemia indications in regions where it is approved, and will retain this name upon approval in regions currently under regulatory review. Agios expects AQVESME to be available in the U.S. in late January 2026, following implementation of the AQVESME REMS program.”