U.S. FDA Grants Priority Review to Agios’ sNDA for Mitapivat in Sickle Cell Disease

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CAMBRIDGE, Mass., July 07, 2026 (GLOBE NEWSWIRE) -- 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 Priority Review designation for the mitapivat sNDA marks an important milestone for the sickle cell community – a large, underserved population that has long needed new treatment options to help manage the significant burden of disease," said Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. "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. We look forward to working collaboratively with the FDA throughout this review, with the goal of delivering the first oral PK activator in sickle cell disease." 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). About U.S. Accelerated Approval The U.S. Food and Drug Administration's (FDA) accelerated approval pathway expedites the availability of medicines that can fill a medical need for a serious condition. U.S. accelerated approval is subject to the same rigorous FDA review standards as medicines reviewed through the traditional approval pathway. A confirmatory clinical trial to further demonstrate the medicine's clinical benefit is required to convert the accelerated approval to a traditional approval, and this trial must be underway when the FDA makes its approval decision.

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