The FDA accepted uniQure’s Biologics License Application (BLA) for ifezuntirgene inilparvovec (AMT-130) under Priority Review, setting a May 1 action date. The company disclosed that the agency is “tentatively planning” a future advisory committee discussion, likely through the Cellular, Tissue, and Gene Therapies Advisory Committee, after reviewers took the application for formal review. uniQure previously faced regulatory resistance, and the current acceptance represents a reversal of earlier opposition that intensified through the current year. The gene therapy uses uniQure’s gene-silencing miQURE platform with a microRNA designed to silence the huntingtin gene and reduce the potentially toxic exon 1 protein fragment. Despite the regulatory step forward, shares fell after the announcement, reflecting investor scrutiny of late-stage durability and safety signals, including questions about how long-term data compare with earlier, shorter follow-up snapshots. uniQure will now work through the BLA review process toward a decision that could define a first disease-modifying option for Huntington’s disease.