• Company has already submitted proposed confirmatory trial for FDA review, as required under accelerated approval pathway 
• Company plans to submit mitapivat sNDA in sickle cell disease in the coming months, and is actively working with FDA to achieve alignment on confirmatory trial required for submission

 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a commercial-stage biopharmaceutical company focused on delivering innovative medicines for patients with rare diseases,  announced that it will pursue U.S. accelerated approval for mitapivat, an oral pyruvate kinase (PK) activator, in sickle cell disease, following completion of its pre-supplemental New Drug Application (sNDA) meeting with the U.S. Food and Drug Administration (FDA).

The purpose of the pre-sNDA meeting was to present the data from the mitapivat RISE UP clinical program in sickle cell disease, including both the Phase 2 and Phase 3 trials. Based on the discussion, the FDA recommended submission of a proposal for a confirmatory clinical trial to support U.S. accelerated approval of mitapivat. The FDA’s accelerated approval pathway expedites the availability of medicines that can fill a medical need for a serious condition, with the requirement of a confirmatory clinical trial to convert to a traditional approval.

“Our engagements with the FDA continue to underscore both the unmet need in sickle cell disease and the importance of expeditiously advancing new treatment options for patients living with this complex, debilitating, and deadly disease,” said Sarah Gheuens, M.D., Ph.D., Chief Medical Officer and Head of R&D, Agios. “The clinically meaningful benefits observed in the RISE UP clinical program, combined with our ongoing, constructive, and collaborative dialogue with the FDA, reinforce our confidence in mitapivat’s potential in sickle cell disease. We are focused on advancing mitapivat as rapidly as possible with the rigor required for potential U.S. accelerated approval.”

Agios has already submitted its proposal for the required confirmatory clinical trial to the FDA for review. This proposal incorporates a primary endpoint that is different from those in the RISE UP clinical program and is informed by both analyses of RISE UP data and discussions with the FDA. Based on current planning assumptions, the proposed confirmatory clinical trial is not expected to change the company’s previously issued operating expense guidance, which remains approximately flat compared to 2025.

The company plans to submit a mitapivat sNDA for sickle cell disease in the coming months, and is actively working with the FDA to achieve alignment on the confirmatory clinical trial required for submission.