Agios said the FDA delayed its decision on a supplemental sNDA for mitapivat in sickle cell disease, extending the PDUFA target action date by three months to February 1, 2027. Agios attributed the delay to a “major amendment” request tied to whether a Risk Evaluation and Mitigation Strategy (REMS) would be needed in the SCD population. In response, the company submitted additional safety data following FDA guidance, reflecting updated information from the ongoing RISE UP trial. The change prompted a sharp market reaction, underlining investor focus on safety controls and labeling requirements for disease-modifying therapies in rare hematologic indications.