uniQure said the FDA accepted its Biologics License Application for ifezuntirgene inilparvovec (AMT-130) and set a May 1 PDUFA target action date under Priority Review. The agency’s acceptance reverses prior opposition that emerged last year, according to the company, though uniQure was told a future advisory committee discussion is tentatively planned. The FDA signaled it may involve the Cellular, Tissue, and Gene Therapies Advisory Committee (CTGTAC), but no date had been set. uniQure submitted its BLA in September and also filed a parallel Marketing Authorization Application with the UK’s MHRA. Despite the regulatory milestone, shares reportedly fell after the announcement, with investor caution tied to the later-than-expected action date and questions raised by longer-term dataset trends and ongoing trial risk considerations. If approved, the gene-silencing therapy—delivered via the company’s miQURE platform using microRNA designed to silence the huntingtin gene—would represent the first disease-modifying option for Huntington’s disease and meaningfully reduce remaining regulatory uncertainty ahead of an anticipated decision window.