The FDA has established a May 1 target action date under the Prescription Drug User Fee Act (PDUFA) for uniQure’s Huntington’s disease gene therapy candidate ifezuntirgene inilparvovec, following the acceptance of the company’s Biologics License Application (BLA) under Priority Review. This decision marks a significant turnaround from previous agency opposition. The FDA has indicated plans to discuss the BLA at an upcoming advisory committee meeting, although no date has been set.
Despite the positive news, uniQure’s stock dipped 3% as investors expressed caution, citing a later-than-expected PDUFA date and concerns regarding the efficacy data from ongoing trials. The gene therapy utilizes uniQure’s miQURE® platform to silence the huntingtin gene and is administered through a targeted neurosurgical delivery method. The company plans to begin patient screening in the U.S. by the end of 2026, with the confirmatory study designed to enroll approximately 200 patients.
Source: genengnews.com · Image: Stem Cell Research (0498) (14001968553) by The U.S. Food and Drug Administration, Public domain, via Wikimedia Commons
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