FDA-Approved Mitapivat Transforms Thalassaemia Treatment: First Oral Drug Offers New Hope

• Mitapivat, the first FDA-approved oral drug for thalassaemia, targets red blood cell energy metabolism, offering a disease-modifying approach beyond traditional transfusions and iron chelation.
• Clinical trials (ENERGIZE, ENERGIZE-T) show mitapivat can significantly increase haemoglobin levels, reduce fatigue, and decrease transfusion dependence in adult alpha- and beta-thalassaemia patients.
• While promising, response rates vary, and side effects like headache, nausea, and potential liver risks require careful monitoring under a Risk Evaluation and Mitigation Strategy.
• For India, with its high thalassaemia burden, mitapivat could be transformative by reducing transfusion reliance, but regulatory approval, affordability, and access remain significant challenges.
• Mitapivat offers a scalable, less invasive alternative to complex gene-editing therapies, though it is non-curative and requires lifelong use, complementing other emerging treatments.

Why It Matters: Mitapivat marks a significant shift in thalassaemia care, offering the first oral, disease-modifying treatment.