The FDA accepted uniQure’s BLA for ifezuntirgene inilparvovec (AMT-130) under Priority Review and set a May 1 target action date under PDUFA. The decision reverses earlier FDA opposition encountered over the past year, signaling a major regulatory inflection point for the company’s Huntington’s disease gene-silencing approach. uniQure also filed a UK MAA with the MHRA. The FDA cautioned the company that it was tentatively planning discussion at an advisory committee meeting, most likely the CTGTAC, though no meeting date had been set at deadline. The therapy uses uniQure’s miQURE platform and incorporates a microRNA intended to silence huntingtin and the potentially toxic exon 1 fragment. While the review milestone reduced some regulatory uncertainty, the stock reaction reflected investor caution around data maturity and the timing of agency steps. Analyst William Blair’s Lachlan Hanbury-Brown pointed to BLA acceptance as removing regulatory risk, but market participants appeared focused on expected timing and longer-term clinical slowing signals seen in earlier datasets.