Agios Pharmaceuticals (NASDAQ:AGIO) dropped 7% after revealing that the U.S. Food and Drug Administration has yet to issue a ruling on its supplemental New Drug Application (sNDA) for mitapivat.
The filing seeks expanded approval for mitapivat to treat adults with both non–transfusion-dependent and transfusion-dependent forms of alpha- and beta-thalassemia. Although the application cleared its Prescription Drug User Fee Act (PDUFA) target date of December 7, 2025, the FDA has kept it under active review.
Agios, a commercial-stage biotech focused on rare disease therapies, said it is continuing to work with the agency to finalize product labeling and Risk Evaluation and Mitigation Strategy (REMS) documentation. The company stressed that the FDA has not asked for any new or additional efficacy or safety data, nor has Agios submitted such data independently. The regulator has also not indicated when a final decision should be expected.
The company noted it is proceeding “expeditiously” with the FDA toward completing the review, though the delay adds uncertainty for investors who had anticipated an approval decision by the scheduled PDUFA deadline.
Mitapivat is being developed as a treatment option for various forms of thalassemia—genetic blood disorders that impair hemoglobin production and can result in chronic anemia and other health complications.