FDA Reviews Agios’ PYRUKYND® For Thalassemia Treatment

Agios Pharmaceuticals, Inc. is a state-of-the-art company focused on cellular metabolism and pyruvate kinase (PK) activation, pioneering therapies for rare disease therapies. The firm has announced that the U.S. Food and Drug Administration (FDA) has accepted its supplemental New Drug Application (sNDA) for PYRUKYND® (mitapivat). 

The application seeks approval for PYRUKYND® to treat adult patients with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia. The FDA review classification for this application is Standard, and the Prescription Drug User Fee Act (PDUFA) goal date is September 7, 2025.

Sarah Gheuens, M.D., PhD, chief medical officer and head of R&D at Agios, stated, “Thalassemia is a rare, lifelong inherited blood disorder that causes chronic anaemia and can lead to severe complications, including organ damage, stroke, and other serious health issues, with patients today having limited or no effective treatment options. We look forward to collaborating with the FDA in the coming months as they continue to review our application to bring PYRUKYND, a disease-modifying oral medication, to thalassemia patients regardless of their genotype or transfusion needs.”

The submission is supported by data from two pivotal Phase 3 studies: the ENERGIZE trial, which evaluated mitapivat in adults with non-transfusion-dependent thalassemia, and the ENERGIZE-T trial, which focused on transfusion-dependent thalassemia patients. Results from these trials were shared at major scientific conferences, including the European Hematology Association Hybrid Congress in June 2024 and the American Society of Hematology Annual Meeting in December 2024.