Agios Awaits Pyrukynd Decision in Thalassemia as FDA Misses Due Date

Core Insights - Agios Pharmaceuticals (AGIO) is awaiting a decision from the FDA regarding its supplemental new drug application (sNDA) for Pyrukynd (mitapivat) to treat thalassemia, which has missed the expected deadline of December 7, 2025 [1][2][7] - The sNDA is under active review, and no additional efficacy or safety data have been requested by the FDA [2][8] - Pyrukynd is already approved in the U.S. and Europe for treating hemolytic anemia in adults with pyruvate kinase deficiency [3][7] Company Performance - Year-to-date, Agios shares have declined by 17%, contrasting with a 19.3% increase in the industry [4] - The FDA extended the review timeline for the sNDA by three months in September 2025 due to a request for a Risk Evaluation and Mitigation Strategy (REMS) related to potential liver injury risks [5] Regulatory Developments - The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for Pyrukynd's label expansion in thalassemia, with a final decision from the European Commission expected in early 2026 [8] - Besides thalassemia, Pyrukynd is also being studied for sickle cell disease (SCD), where recent phase III trial results were mixed, meeting one primary endpoint but failing another [9]