FDA Extends Agios’ PYRUKYND sNDA Review For Thalassemia To December 2025

Agios Pharmaceuticals, Inc., a commercial-stage biopharmaceutical company advancing treatments for rare diseases, announced that the U.S. FDA has extended the Prescription Drug User Fee Act (PDUFA) goal date for the supplemental New Drug Application (sNDA) of PYRUKYND® (mitapivat). The oral pyruvate kinase (PK) activator is under review for the treatment of adult patients with non-transfusion-dependent (NTD) and transfusion-dependent (TD) alpha- or beta-thalassemia. The new decision date has been set for December 7, 2025, representing a three-month delay.

“We remain confident in the favorable benefit-risk profile of PYRUKYND in thalassemia. We look forward to continuing our collaborative engagement with the FDA, with the goal of bringing this disease-modifying oral medicine to adult patients with thalassemia in the U.S,” said Brian Goff, Chief Executive Officer, Agios. 

The extension follows an FDA request for Agios to submit a Risk Evaluation and Mitigation Strategy (REMS) to address potential risks of hepatocellular injury highlighted in the original sNDA. Since the REMS submission qualifies as a major amendment, the review timeline was extended. Importantly, the FDA has not requested any additional efficacy or safety data, and Agios has not submitted new data outside of the REMS proposal.

The PYRUKYND sNDA is backed by data from the ENERGIZE and ENERGIZE-T Phase 3 trials, global randomized, double-blind, placebo-controlled studies conducted in adults with NTD and TD alpha- or beta-thalassemia. These pivotal studies support the potential of PYRUKYND to expand treatment options for patients living with these rare blood disorders.