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uniQure Announces Submission of Biologics License Application for Ifezuntirgene Inilparvovec (AMT-130) in Huntington’s Disease

QURE
  • uniQure submitted a Biologics License Application (BLA) to the U.S. FDA for accelerated approval of ifezuntirgene inilparvovec (AMT-130) for Huntington's disease.
  • The company also submitted a Marketing Authorisation Application (MAA) to the U.K. MHRA for the same investigational gene therapy.
  • Both regulatory submissions are supported by three-year data from a Phase I/II study showing a slowing of disease progression compared to external controls.
  • uniQure has requested priority review for the BLA, which could shorten the FDA review cycle to six months if granted.
  • The company plans to present a four-year data analysis from the ongoing Phase I/II studies before the end of the third quarter of 2026.
  • Ifezuntirgene inilparvovec holds Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), & Fast Track designations from the FDA.
  • The therapy utilizes a gene-silencing platform to target the huntingtin gene & is administered via a single MRI-guided neurosurgical procedure into the striatum.
  • Clinical trials included a U.S. randomized study of 26 patients & a European open-label study of 13 patients, with additional cohorts evaluating doses & immunosuppression.
  • There are currently no approved therapies to delay onset or slow progression of Huntington's disease, which affects approximately 75,000 people in the U.S., EU, & U.K.