FDA Sets May 1 PDUFA Action Date for uniQure’s Huntington’s Disease Gene Therapy
uniQure has aligned with the FDA on what the company calls key elements of the confirmatory study design: The study is being designed as a randomized, standard-of-care trial enrolling approximately 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care, with Total Functional Capacity (TFC) at 36 months serving as the primary endpoint. The post FDA Sets May 1 PDUFA Action…
The FDA has set May 1 as the target action date under the Prescription Drug User Fee Act (PDUFA) for uniQure's Huntington's disease gene therapy candidate ifezuntirgene inilparvovec. If approved, this treatment would be the first to modify the fatal, inherited neurodegenerative disorder. This marks a reversal of the FDA's opposition that previously existed.
UniQure submitted the BLA to the FDA in September, along with an MAA to the UK's Medicines and Healthcare products Regulatory Agency (MHRA). CEO Matt Kapusta called the FDA's acceptance an important milestone for the Huntington's disease community. However, investors reacted cautiously, as uniQure shares dropped 3% following the news.
The PDUFA target action date was later than expected, and the four-year data showed lower slowing of disease progression than earlier three-year data. There are also concerns about risk in the ongoing trials. UniQure's BLA for ifezuntirgene inilparvovec has received Priority Review, Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), and Fast Track designations from the FDA.
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