Agios Provides Update on U.S. sNDA for Mitapivat in Thalassemia

Core Viewpoint - Agios Pharmaceuticals has announced that the FDA has not yet made a regulatory decision on the supplemental New Drug Application (sNDA) for mitapivat, which is under active review for treating adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia [1][2] Group 1: Regulatory Status - The FDA's Prescription Drug User Fee Act (PDUFA) goal date for the sNDA was December 7, 2025, but no decision has been issued yet [1] - Agios is collaborating with the FDA to finalize labeling documents and Risk Evaluation and Mitigation Strategy (REMS) materials, without any new efficacy or safety data being requested or submitted [2] Group 2: Company Overview - Agios Pharmaceuticals is a commercial-stage biopharmaceutical company based in Cambridge, Massachusetts, focused on innovative medicines for rare diseases [3] - The company aims to redefine the future of rare disease treatment by building partnerships and advancing a growing pipeline of medicines [3]