FDA Extends Agios Mitapivat Sickle Cell Review to February 2027; AGIO Shares Fall 17.55%

Agios Pharmaceuticals (AGIO) said the U.S. Food and Drug Administration extended its review of the supplemental New Drug Application for mitapivat in sickle cell disease, setting a new PDUFA goal date of February 1, 2027. Shares were trading at $30.69, down 17.55%. The application, submitted under the accelerated approval pathway with Priority Review, had an original PDUFA goal date of November 1, 2026. On October 9, 2026, the FDA requested additional information to assess whether a Risk Evaluation and Mitigation Strategy is needed. Agios responded October 19 with roughly nine months of updated safety data through October 9 from the open-label extension periods of the RISE UP Phase 2 and Phase 3 trials, showing no cases of hepatocellular injury with a pattern and severity similar to that seen in thalassemia trials. The FDA classified the response as a major amendment on October 28, triggering a three-month extension. Agios said the benefit-risk profile remains unchanged and will continue labeling discussions, including potential REMS elements. Mitapivat is approved in the U.S. for pyruvate kinase deficiency and alpha- or beta-thalassemia.