FDA Accepts Agios' sNDA for Pyrukynd in Thalassemia

Agios Pharmaceuticals (AGIO) announced that the FDA has accepted its supplemental new drug application (sNDA) seeking label expansion for its sole marketed drug, Pyrukynd, in thalassemia indication.A final decision is expected by Sep. 7, 2025. If approved, Pyrukynd will become the first oral therapy for use in all thalassemia subtypes. A commercial launch in this indication is expected before this year’s end.The sNDA is supported by data from two phase III studies, ENERGIZE and ENERGIZE-T, which evaluated P ...