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uniQure Announces FDA Acceptance and Priority Review of the Biologics License Application for ifezuntirgene inilparvovec for the Treatment of Huntington’s Disease

QURE
  • uniQure received FDA acceptance of its Biologics License Application (BLA) for ifezuntirgene inilparvovec, an investigational gene therapy for Huntington's disease, submitted under the Accelerated Approval pathway.
  • The application was granted Priority Review status with a PDUFA target action date of May 1, 2027.
  • The FDA plans to discuss the application at an Advisory Committee meeting, though specific dates & timelines remain to be finalized.
  • Approval of ifezuntirgene inilparvovec would make it the world's first disease-modifying treatment for Huntington's disease.
  • The BLA is supported by 36-month data from Phase I/II studies compared to an external control derived from the Enroll-HD natural history database.
  • uniQure & the FDA have aligned on a confirmatory study designed as a randomized, standard-of-care trial enrolling approximately 200 patients with a 2:1 dosing ratio.
  • The confirmatory study will use Total Functional Capacity (TFC) as the primary endpoint at 36 months, with patient screening in the U.S. anticipated to begin by the end of 2026.
  • Ifezuntirgene inilparvovec holds Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), & Fast Track designations from the FDA.
  • The therapy utilizes a miQURE platform to silence the huntingtin gene & is delivered via a single administration through MRI-guided stereotactic neurosurgery directly into the striatum.
  • Phase I/II clinical studies have dosed 51 patients across multiple cohorts, including U.S. randomized & European open-label groups.