Agios Pharmaceuticals, Inc. (NASDAQ:AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for mitapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease with a Priority Review. The Prescription Drug User Fee Act (PDUFA) goal date for this sNDA, submitted under the FDA’s accelerated approval pathway, is November 1, 2026.

The FDA’s Priority Review designation is granted to applications for medicines that may offer significant improvements in safety or efficacy for serious conditions, shortening the target review timeline from the standard 10 months to six months.

The mitapivat sNDA is based on data from the global, randomized, double-blind, placebo-controlled RISE UP Phase 2 and Phase 3 trials in patients aged 16 years or older with sickle cell disease. Mitapivat is currently approved in the U.S. for adult patients in two hemolytic anemias: PK deficiency (2022) and thalassemia (2025).