Investing.com -- Agios Pharmaceuticals (NASDAQ:AGIO) stock fell 7% after the company announced that the U.S. Food and Drug Administration (FDA) has not yet issued a regulatory decision on its supplemental New Drug Application (sNDA) for mitapivat.
The application, which seeks approval for mitapivat in the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, remains under active review despite passing its December 7, 2025, Prescription Drug User Fee Act (PDUFA) goal date.
The commercial-stage biopharmaceutical company, which focuses on delivering medicines for patients with rare diseases, stated it is working closely with the FDA to finalize labeling documents and Risk Evaluation and Mitigation Strategy (REMS) materials for the drug.
In its announcement, Agios emphasized that the FDA has not requested any new or additional efficacy or safety data, and the company has not submitted such information. However, the regulatory agency has not provided a timeline for when it will make its final decision.
Agios indicated it continues to work "expeditiously" with the FDA to conclude the review process for the supplemental application. The delay creates uncertainty for investors who had anticipated a decision by the scheduled PDUFA date.
Mitapivat is being evaluated as a potential treatment option for thalassemia, a group of inherited blood disorders characterized by reduced or abnormal production of hemoglobin, which can lead to anemia and other complications.
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