Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and pyruvate kinase (PK) activation pioneering therapies for rare diseases, 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.

“Thalassemia is a rare, lifelong inherited blood disorder that causes chronic anemia 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,” said Sarah Gheuens, M.D., Ph.D., chief medical officer and head of R&D at Agios. “We look forward to collaborating with the FDA in the coming months as they continue to review our application, with the goal of bringing PYRUKYND, a disease-modifying oral medication, to thalassemia patients regardless of their genotype or transfusion needs.”

The sNDA is based on the results from the ENERGIZE and ENERGIZE-T Phase 3 trials evaluating mitapivat versus placebo in adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, respectively. The ENERGIZE randomized clinical trial results were presented at the European Hematology Association 2024 Hybrid Congress in June 2024, and the ENERGIZE-T randomized clinical trial results were presented at the 66ᵗʰ American Society of Hematology Annual Meeting and Exposition in December 2024.