Agios’ Mitapivat sNDA Receives FDA Priority Review with November 2026 PDUFA
AGIO•FDA accepted Agios’s sNDA for mitapivat in sickle cell disease with Priority Review and a PDUFA goal date of November 1, 2026. If approved, mitapivat would become the first oral pyruvate kinase activator for sickle cell disease, backed by RISE UP Phase 2/3 data.
1. FDA Grants Priority Review
FDA accepted Agios Pharmaceuticals’ supplemental New Drug Application for mitapivat in sickle cell disease under the accelerated approval pathway and granted Priority Review, reducing the target review period from ten to six months with a PDUFA goal date of November 1, 2026.
2. Mitapivat Development and Trials
Mitapivat is an oral pyruvate kinase activator currently approved for pyruvate kinase deficiency and thalassemia, supported by global RISE UP Phase 2 and Phase 3 trials involving over 1,300 patient-years that demonstrated improvements in hemoglobin and reduced hemolysis markers.
3. Next Steps and Timeline
A confirmatory REIGNITE Phase 3 trial is underway to assess transfusion-free status and hemolytic biomarkers over 52 weeks, and a successful Priority Review could enable Agios to launch the first oral PK activator for sickle cell disease late next year.
