uniQure submits BLA for AMT-130 in Huntington's disease, seeks accelerated approval
- Sponsor
- uniQure N.V. $QURE
- Drug
- ifezuntirgene inilparvovec
- Indication
- Huntington's disease
uniQure submitted a Biologics License Application to the FDA seeking accelerated approval of AMT-130 (ifezuntirgene inilparvovec), an investigational one-time gene therapy for Huntington's disease. A parallel Marketing Authorisation Application was submitted to the UK MHRA the same day. Filings are supported by three-year Phase I/II data showing slowed disease progression vs external control.
What this means
Huntington's disease is an inherited brain disorder that causes progressive movement, thinking and mood problems and has no approved treatment that slows it down. AMT-130 is a one-time gene therapy delivered directly into the brain by MRI-guided surgery; it lowers production of the toxic mutant huntingtin protein that drives the disease. uniQure has submitted its Biologics License Application to the FDA and a parallel application to the UK MHRA, based on three-year data from its Phase I/II study showing patients receiving AMT-130 had slower disease progression than an external comparator group. FDA is being asked to grant accelerated approval, and the program already holds Breakthrough Therapy, RMAT and Fast Track designations.