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6d ago

FDA Approves Aqvesme for Thalassemia Anemia, First U.S. Therapy for Both Patient Groups

Approval reflects late-stage efficacy results with use restricted under a REMS requiring frequent liver tests.

Signage is seen outside of the Food and Drug Administration (FDA) headquarters in White Oak, Maryland, U.S., August 29, 2020. REUTERS/Andrew Kelly/File Photo
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Overview

  • The authorization covers adults with alpha- or beta-thalassemia in both transfusion-dependent and non-transfusion-dependent disease.
  • The ENERGIZE and ENERGIZE-T Phase 3 trials (about 452 participants) met primary and key secondary endpoints, improving hemoglobin, reducing transfusion burden, and lessening fatigue versus placebo.
  • Trial safety data showed five cases suggestive of hepatocellular injury among Aqvesme recipients, including two hospitalizations, with abnormalities emerging within six months and improving after the drug was stopped.
  • The FDA-approved Aqvesme REMS requires liver tests before the first dose, every four weeks for 24 weeks, and then as clinically indicated.
  • Agios expects U.S. availability in late January 2026 after REMS rollout, while mitapivat continues as Pyrukynd for pyruvate kinase deficiency.