FDA has moved key Huntington’s disease gene therapy applications forward, signaling renewed momentum in a high-profile class. uniQure said the agency accepted its Biologics License Application (BLA) for ifezuntirgene inilparvovec under Priority Review and set a May 1 PDUFA action date. The company also disclosed the FDA is tentatively planning an advisory committee discussion, likely through the Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC), after the BLA acceptance. The decision reverses prior regulatory resistance, but uniQure also faces investor caution tied to the gene therapy’s longer-term efficacy signals and ongoing trial risk. Separately, the FDA set a delayed PDUFA decision timeline for Agios’ mitapivat supplemental NDA in sickle cell disease after requesting additional information on whether a REMS (Risk Evaluation and Mitigation Strategy) is needed. Agios pointed to updated safety data, including 56 hemoglobin responders out of 138 patients in the mitapivat arm versus 2 of 69 on placebo reported in The Lancet, as it works to address the agency’s safety framework questions.