Update provided on PDUFA goal date for Pyrukynd (mitapivat) in thalassemia – Agios Pharmaceuticals

Agios Pharmaceuticals Inc. announced that the FDA has extended the Prescription Drug User Fee Act (PDUFA) goal date for the supplemental New Drug Application (sNDA) of Pyrukynd (mitapivat), an oral pyruvate kinase (PK) activator, for the treatment of adult patients with non-transfusion-dependent (NTD) and transfusion-dependent (TD) alpha- or beta-thalassemia by three months to December 7, 2025. Following a recent information request from the FDA, Agios submitted a proposed Risk Evaluation and Mitigation Strategy (REMS) to mitigate the risk of hepatocellular injury that was described in the original Pyrukynd sNDA. The submission of the REMS is a major amendment to the Pyrukynd sNDA, resulting in a three-month review extension. This extension is not the result of new or additional efficacy or safety data requested by the FDA or submitted by Agios. The Pyrukynd sNDA is supported by results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase III trials in adults with NTD and TD alpha- or beta-thalassemia, respectively.

“We remain confident in the favorable benefit-risk profile of PYRUKYND in thalassemia,” said Brian Goff, Chief Executive Officer, Agios. “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.”