Agios Moves Toward FDA Accelerated Approval For Mitapivat In Sickle Cell Disease

Agios Pharmaceuticals, Inc. announced plans to pursue U.S. accelerated approval for mitapivat, an oral pyruvate kinase activator, for the treatment of sickle cell disease. This decision follows a pre-supplemental New Drug Application (sNDA) meeting with the U.S. Food and Drug Administration, where data from the RISE UP clinical program were reviewed.

The RISE UP program includes both Phase 2 and Phase 3 studies evaluating mitapivat in sickle cell disease. Based on discussions, the FDA advised Agios to submit a proposal for a confirmatory clinical trial to support accelerated approval. This pathway allows earlier access to therapies addressing serious conditions, provided that additional trials confirm long-term benefit.

“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 confirmatory trial proposal, incorporating a new primary endpoint informed by clinical data and regulatory feedback. The company noted that the proposed study is not expected to impact its current financial outlook, with operating expenses projected to remain in line with 2025 levels.

The company plans to submit the sNDA for mitapivat in the coming months and continues to work closely with the FDA to finalize the requirements for the confirmatory trial needed to support approval.