Agios Application Accepted for PYRUKYND® (mitapivat) in Alpha- or Beta-Thalassemia

 Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) accepted the company’s supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat) for the treatment of adult patients with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The review classification for this application is Standard and the Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025.

https://www.globenewswire.com/news-release/2025/01/08/3006111/31990/en/FDA-Accepts-Agios-Supplemental-New-Drug-Application-for-PYRUKYND-mitapivat-in-Adult-Patients-with-Non-Transfusion-Dependent-and-Transfusion-Dependent-Alpha-or-Beta-Thalassemia.html