FDA extends review of Agios' mitapivat for sickle cell disease to Feb. 1, 2027
AGIO•The FDA extended its review deadline for Agios Pharmaceuticals' mitapivat application for sickle cell disease by three months, to Feb. 1, 2027, from Nov. 1, 2026. The agency classified updated safety information as a major amendment and is evaluating whether a risk evaluation and mitigation strategy is necessary.
1. Review extended
The FDA extended the review deadline for Agios Pharmaceuticals' mitapivat application for sickle cell disease by three months, to Feb. 1, 2027, from Nov. 1, 2026. The agency classified updated safety information as a major amendment while evaluating whether a risk evaluation and mitigation strategy is necessary.
2. Safety and labeling
Additional follow-up showed no hepatocellular injury cases matching the pattern and severity observed in mitapivat's adult thalassemia trials. The application has Priority Review under the accelerated approval pathway, and labeling discussions continue, including potential risk mitigation requirements.
