Agios Pharmaceuticals shares surge 14% after FDA accepts NDA for mitapivat with a November 1 decision target.
Agios Pharmaceuticals (NASDAQ: AGIO) stock rose over 14% following the FDA’s acceptance of its supplemental New Drug Application (NDA) for mitapivat to treat sickle cell disease (SCD). The agency granted Priority Review, setting a decision deadline of November 1.
Mitapivat, already approved for pyruvate kinase deficiency and thalassemia, met its primary endpoint of hemoglobin response in a Phase 3 SCD trial but failed to show a statistically significant reduction in sickle cell pain crises. The FDA’s decision suggests confidence in the drug’s potential despite the missed endpoint.
The Priority Review designation accelerates the regulatory process, signaling potential market expansion for Agios if approved.