uniQure submitted its application for U.S. approval of a one-time gene therapy for Huntington's disease on Wednesday and asked the Food and Drug Administration to review it on an accelerated schedule. The company submitted a Biologics License Application seeking accelerated approval of ifezuntirgene inilparvovec, known through its development as AMT-130, and simultaneously submitted a marketing application to the United Kingdom's Medicines and Healthcare products Regulatory Agency.
The company also requested priority review from the agency, which if granted would shorten the FDA review to six months following the standard 60-day filing review period. That timing is the practical news for families. It converts an open-ended question about whether a treatment might exist into a defined regulatory calendar.
