The Food and Drug Administration has extended its review of Agios Pharmaceuticals’ supplemental application for mitapivat in sickle cell disease by three months, moving the PDUFA goal date from November 1, 2026 to February 1, 2027, the company announced on October 30. The application, for the oral pyruvate kinase activator already approved in pyruvate kinase deficiency and thalassemia, was submitted under the accelerated approval pathway and holds Priority Review.
The sequence runs through a safety programme question. On October 9 the FDA asked for additional information to evaluate whether a Risk Evaluation and Mitigation Strategy is necessary for mitapivat in sickle cell disease; on October 19 Agios replied with about nine months of updated safety data from the open-label extensions of its RISE UP trials; on October 28 the FDA classified that response as a major amendment, which carries the three-month extension. Agios said the added follow-up still shows no hepatocellular injury cases in sickle cell patients with a pattern or severity similar to those seen in thalassemia trials, that its benefit-risk assessment is unchanged, and that labeling discussions, including elements of a potential REMS, continue. Dow Jones reported Agios shares fell 19 percent to $30.26 on Friday.