AMT-130

September 02, 2026: uniQure N.V. (NASDAQ: QURE) announced submission of a BLA to the FDA seeking accelerated approval of ifezuntirgene inilparvovec (AMT-130) for Huntington’s disease, and submission of a Marketing Authorisation Application to the U.K. MHRA. The company requested FDA priority review; if granted, review would be shortened to six months after the FDA’s 60-day filing review period.

Both filings are supported by previously announced 3-year Phase I/II data showing slowing of disease progression versus a propensity score-matched external control from the Enroll-HD natural history database. uniQure said it plans to present a 4-year analysis from the ongoing Phase I/II studies before the end of Q3 2026.

The Phase I/II program includes a U.S. randomized study of 26 patients (low dose n=6, high dose n=10, sham control n=10, with four controls later crossing over), a European open-label study of 13 patients (low dose n=6, high dose n=7), a 12-patient cohort testing both doses with immunosuppression, and a six-patient U.S. cohort evaluating the high dose in patients with lower striatal volumes.

The therapy has received FDA Breakthrough Therapy, RMAT, and Fast Track designations.