Mitapivat (Aqvesme) for Thalassemia
Mitapivat (Aqvesme) for Thalassemia
August 3, 2026 (Issue: 1760)
Mitapivat (Aqvesme – Agios), an oral pyruvate kinase
activator, has been approved by the FDA for treatment
of anemia in adults with alpha or beta thalassemia. It is
the first product to be approved in the US for treatment
of alpha thalassemia...more
- A Martin and AA Thompson. Thalassemias. Pediatr Clin North Am 2013; 60:1383. doi:10.1016/j.pcl.2013.08.008
- A Amid et al. Guidelines for the management of α-thalassaemia. Thalassaemia International Federation; 2023. Available at: https://bit.ly/42Vr09V. Accessed July 15, 2026.
- AT Taher et al. Guidelines for the management of transfusion-dependent β-thalassaemia (TDT), 5th edition. Thalassaemia International Federation; 2025. Available at: https://bit. ly/4vaYNrs. Accessed July 15, 2026.
- AT Taher et al. Guidelines for the management of non-transfusion-dependent β-thalassaemia, 3rd edition. Thalassaemia International Federation; 2023. Available at: https://bit.ly/4nTxfER. Accessed July 15, 2026.
- In brief: Casgevy for beta thalassemia. Med Lett Drugs Ther 2024; 66:79.
- Betibeglogene autotemcel (Zynteglo) for beta thalassemia. Med Lett Drugs Ther 2024; 66:e167.
- AT Taher et al. Mitapivat in adults with non-transfusion-dependent α-thalassaemia or β-thalassaemia (ENERGIZE): a phase 3, international, randomised, double-blind, placebo-controlled trial. Lancet 2025; 406:33. doi:10.1016/s0140-6736(25)00635-x
- Inhibitors and inducers of CYP enzymes, P-glycoprotein, and other transporters. Med Lett Drugs Ther 2023 January 25 (epub). Available at: www.medicalletter.org/downloads/CYP_PGP_Tables.pdf.
- Approximate WAC. WAC = wholesaler acquisition cost or manufacturer's published price to wholesalers; WAC represents a published catalogue or list price and may not represent an actual transactional price. Source: AnalySource® Monthly. July 5, 2026. Reprinted with permission by First Databank, Inc. All rights reserved. ©2026. www.fdbhealth.com/drug-pricing-policy.
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