MedPath

AG-181

Generic Name
AG-181

Agios' Pyrukynd Faces FDA Decision for Thalassemia Treatment by September 2025

• The FDA has accepted Agios' sNDA for Pyrukynd (mitapivat) to treat adults with transfusion-dependent and non-transfusion-dependent alpha- or beta-thalassemia, setting a PDUFA goal date of September 7, 2025. • Pyrukynd, a pyruvate kinase activator, could become the first oral therapy approved for all thalassemia subtypes, addressing a significant unmet need for patients with limited treatment options. • Agios completed enrollment for the Phase 3 RISE UP study of mitapivat in sickle cell disease, with topline results expected in late 2025 and a potential U.S. commercial launch in 2026.
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