Agios Continues Collaboration With FDA To Finalize Mitapivat sNDA Documentation For Thalassemia Treatment

Agios Pharmaceuticals announced that the U.S. FDA has not yet issued a final regulatory decision on its supplemental New Drug Application (sNDA) for mitapivat. The application seeks approval for the treatment of adult patients with both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The company had initially expected a decision by the December 7, 2025 PDUFA date, but the review remains ongoing.

According to Agios, the FDA is continuing to review the application, with efforts concentrated on finalizing product labeling and completing the required Risk Evaluation and Mitigation Strategy (REMS) documentation. Importantly, the agency has not requested new clinical efficacy or safety data, suggesting that the remaining steps are primarily administrative and related to documentation.

No updated timeline has been provided by the FDA. Agios stated that it is working diligently with the agency to bring the review process to completion as quickly as possible.

Agios reaffirmed its broader mission of transforming care for patients with rare diseases. With deep expertise in hematology and a growing pipeline supported by scientific and community partnerships, the Cambridge-based biopharmaceutical company continues to advance treatments designed around patient needs.