uniQure files U.S. and U.K. applications for Huntington’s gene therapy

  • uniQure submitted U.S. and U.K. licensing applications for its investigational Huntington’s gene therapy.
  • The three-year Phase I/II analysis showed slowed disease progression versus an external control.
  • uniQure plans to present four-year Phase I/II data before the current third quarter ends.

uniQure N.V. submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA), seeking accelerated approval for ifezuntirgene inilparvovec, also known as AMT-130, an investigational gene therapy for Huntington’s disease. The company also filed a Marketing Authorisation Application (MAA) with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA). Both applications are supported by a three-year analysis from Phase I/II studies, compared with a propensity score-matched external control from the Enroll-HD natural history database, which showed slowed disease progression. uniQure requested priority review for the U.S. application; if granted, the FDA review cycle would be six months after its 60-day filing review. The company plans to present four-year Phase I/II data before the end of the current third quarter. The therapy has received Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT), and Fast Track designations from the FDA, but regulatory approval remains uncertain and depends on agency reviews and additional clinical evidence.

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