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News  ›  GlobeNewswire Inc.

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

GlobeNewswire Inc. Logo GlobeNewswire Inc. • •By Agios Pharmaceuticals, Inc.
U.S. FDA Approves Agios’ AQVESME™ (mitapivat) for the Treatment of Anemia in Adults with Alpha- or Beta-Thalassemia

The FDA has approved Agios Pharmaceuticals' AQVESME (mitapivat), an oral pyruvate kinase activator, for treating anemia in adults with alpha- or beta-thalassemia. AQVESME is the only FDA-approved medicine for both non-transfusion-dependent and transfusion-dependent forms of the disease. The approval is based on Phase 3 ENERGIZE and ENERGIZE-T trials showing significant improvements in hemoglobin levels, fatigue, and transfusion burden. The drug will be available in late January 2026 under a REMS program due to hepatocellular injury risks observed in clinical trials.

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AMJB   positive

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AGIO   positive

FDA approval of AQVESME represents a significant milestone for the company, establishing it as the only approved treatment for both transfusion-dependent and non-transfusion-dependent thalassemia. The approval is based on successful Phase 3 trials meeting primary and secondary endpoints. However, the positive sentiment is tempered by the requirement for a restrictive REMS program due to hepatocellular injury risks observed in 5 patients during trials, which may impact market adoption and commercial success.

Keywords
#FDA approval #mitapivat #thalassemia #anemia treatment #pyruvate kinase activator #ENERGIZE trials #REMS program #hepatocellular injury #rare disease

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