- uniQure submitted a BLA to the FDA seeking accelerated approval for AMT-130, its investigational gene therapy for Huntington’s disease, and requested priority review.
- The company also submitted a U.K. MAA; if the FDA grants priority review, the agency’s review cycle would be six months after the 60-day filing review period.
- The applications are supported by three-year Phase I/II data showing slowed disease progression versus a propensity score-matched external control, with four-year data due before the end of Q3 2026.
Quotes
“The submission of licensing applications for ifezuntirgene inilparvovec represents an important milestone for the Huntington’s disease community”