CAMBRIDGE, Mass., Dec. 08, 2025 (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 not yet issued a regulatory decision on the supplemental New Drug Application (sNDA) for mitapivat for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia and that the sNDA remains under active review. The Prescription Drug User Fee Act (PDUFA) goal date issued by the FDA was December 7, 2025.
Agios is collaborating closely with the FDA to finalize the labeling documents and Risk Evaluation and Mitigation Strategy (REMS) materials. The FDA has not requested, and the company has not submitted, any new or additional efficacy or safety data.
The FDA has not provided a timeline for its regulatory decision. Agios continues to work expeditiously with the FDA to conclude the review of the sNDA.
About Agios: Fueled by Connections to Transform Rare Diseases™
At Agios, our vision is to redefine the future of rare disease treatment. Fueled by connections, we build trusted partnerships with communities – collaborating to develop and deliver innovative medicines that have the potential to transform lives. With a foundation in hematology, we combine biological expertise with real-world insights to advance a growing pipeline of rare disease medicines that reflect the priorities of the people we serve. Agios is a commercial-stage biopharmaceutical company headquartered in Cambridge, Massachusetts. To learn more, visit www.agios.com and follow us on LinkedIn and X.