FDA Grants Priority Review to Agios’ Mitapivat sNDA for Sickle Cell Disease

The US Food and Drug Administration (FDA) has accepted Agios Pharmaceuticals, Inc.’s supplemental New Drug Application (sNDA) for mitapivat in sickle cell disease (SCD) and granted the application Priority Review, along with assigning a Prescription Drug User Fee Act (PDUFA) goal date of November 1, 2026.

If approved, mitapivat would become the first oral pyruvate kinase (PK) activator for patients with sickle cell disease, providing a potential new treatment approach for a population with significant unmet medical needs.

Agios submitted the sNDA on May 12, 2026, under the FDA’s accelerated approval pathway, and was supported by results from the global RISE UP Phase 2 and Phase 3 clinical program.