uniQure secured an FDA acceptance of its Huntington’s disease gene therapy BLA for ifezuntirgene inilparvovec (AMT-130), triggering a May 1 PDUFA action date under Priority Review. The company also indicated the FDA is “tentatively planning” an advisory committee meeting, most likely CTGTAC, which would provide additional scrutiny before a decision. uniQure said the milestone marks a reversal from prior FDA opposition earlier in the development history. Despite the regulatory progress, uniQure shares fell shortly after the update, reflecting investor caution around the timing of review, longer-term efficacy signals versus earlier datasets, and lingering trial risk questions.