regulatory
confidence high
sentiment positive
materiality 0.80
uniQure submits BLA to FDA for AMT-130 in Huntington's disease; also files MAA in UK
uniQure N.V.
- Submitted BLA to FDA for accelerated approval of ifezuntirgene inilparvovec (AMT-130) for Huntington's disease.
- Also submitted MAA to UK MHRA for same therapy; both supported by 3-year Phase I/II data.
- Requested FDA priority review; if granted, review cycle shortened to 6 months after 60-day filing review.
- Plans to present 4-year data analysis from Phase I/II before end of Q3 2026.
- AMT-130 holds FDA Breakthrough Therapy, RMAT, and Fast Track designations.