Agios Pharmaceuticals, Inc. (NASDAQ: AGIO) has received acceptance from the U.S. Food and Drug Administration (FDA) for its supplemental New Drug Application (sNDA) for mitapivat, an oral pyruvate kinase activator, aimed at treating sickle cell disease.
The application has been granted Priority Review status, which accelerates the review process to six months, with a Prescription Drug User Fee Act (PDUFA) target date set for November 1, 2026.
The sNDA submission is supported by data from the RISE UP Phase 2 and Phase 3 trials, which are global, randomized, double-blind, and placebo-controlled studies involving patients aged 16 years or older with sickle cell disease.
The FDA’s Priority Review designation is reserved for drugs that could significantly improve the safety or efficacy of treatments for serious conditions.
Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D at Agios, stated, “Mitapivat is well positioned to address this treatment gap, supported by results from the RISE UP clinical program and a foundational dataset spanning more than a decade of research that includes over 1,300 patient-years of clinical experience across multiple hemolytic anemias.”
