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U.S. FDA Approves Agios' AQVESME™ (mitapivat) for the Treatment of Anemia in Adults with Alpha- or Beta-Thalassemia

AGIO
  • The FDA approved AQVESME (mitapivat) for treating anemia in adults with non-transfusion-dependent or transfusion-dependent alpha- or beta-thalassemia.
  • AQVESME is the only FDA-approved medicine indicated for anemia in both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia.
  • The approval is based on results from the global Phase 3 ENERGIZE and ENERGIZE-T trials involving 452 patients.
  • Clinical trials demonstrated significant improvements in hemoglobin levels, reductions in transfusion burden, & decreased fatigue compared to placebo.
  • AQVESME carries a Boxed Warning for hepatocellular injury, with five patients in trials experiencing adverse reactions suggestive of liver injury.
  • Due to liver safety risks, AQVESME is available exclusively through the AQVESME REMS program requiring mandatory liver testing & provider/patient certification.
  • The AQVESME REMS program mandates liver tests prior to the first dose, every four weeks for 24 weeks, & then as clinically indicated.
  • The drug will be marketed under the brand name AQVESME in the U.S. for thalassemia indications, while PYRUKYND® remains the brand name for PK deficiency.
  • AQVESME is expected to be available in the U.S. in late January 2026 following implementation of the REMS program.
  • Agios Pharmaceuticals will host an investor conference call & webcast on December 24, 2025, at 8:00 a.m. ET to discuss the approval.
  • Common adverse reactions reported in trials include headache & insomnia occurring in ≥5% of patients.
  • Patients must avoid concomitant use of strong CYP3A inhibitors/inducers & moderate CYP3A inhibitors, & monitor interactions with sensitive CYP substrates.
  • Approximately 6,000 adult patients in the U.S. are diagnosed with thalassemia, a rare inherited blood disorder affecting hemoglobin production.