Agios Pharmaceuticals: FDA Extends PDUFA Goal Date for PYRUKYND in Thalassemia to December 7, 2025

Agios Pharmaceuticals has announced that the FDA has extended the PDUFA goal date for PYRUKYND, an oral pyruvate kinase activator for treating thalassemia, by three months to December 7, 2025. This extension is due to a major amendment to the PYRUKYND sNDA, including a proposed Risk Evaluation and Mitigation Strategy to mitigate the risk of hepatocellular injury. The company remains confident in the drug's favorable benefit-risk profile and looks forward to bringing it to adult patients with thalassemia in the US.