On Thursday, the U.S. Food and Drug Administration (FDA) extended the Prescription Drug User Fee Act (PDUFA) goal date for Agios Pharmaceuticals, Inc.’s (NASDAQ:AGIO) supplemental New Drug Application (sNDA) of Pyrukynd (mitapivat) for 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.
