Agios Pharmaceuticals Secures FDA Approval for Aqvesme (Mitapivat) in Thalassemia Treatment
The U.S. FDA approved Aqvesme (mitapivat) for treating anemia in adults with alpha- or beta-thalassemia12345.
Approval announced around December 2025 by Agios Pharmaceuticals145.
The treatment will be available to patients in late January 2026 after REMS implementation13.
Based on Phase 3 trials showing improved hemoglobin and reduced transfusion burden1.
Five patients in trials had adverse reactions suggesting hepatocellular injury1.
Sources:
1. https://www.investing.com/news/stock-market-news/agios-pharmaceuticals-secures-fda-approval-for-thalassemia-treatment-93CH-4422232
2. https://www.fiercepharma.com/pharma/vandas-motion-sickness-drug-and-agios-thalassemia-treatment-make-grade-fda
3. https://www.clinicaladvisor.com/news/aqvesme-approved-for-anemia-in-alpha-or-beta-thalassemia/
4. https://www.hcplive.com/view/fda-approves-mitapivat-aqvesme-for-non-transfusion-dependent-alpha--and-beta-thalassemia