uniQure files for approval of a Huntington's gene therapy

uniQure said on 2 September 2026 that it had submitted a Biologics License Application to the US Food and Drug Administration and a Marketing Authorisation Application to the UK’s Medicines and Healthcare products Regulatory Agency for ifezuntirgene inilparvovec, known through its development as AMT-130. It is an AAV-delivered gene therapy for Huntington’s disease, given once by neurosurgery.

The BLA seeks accelerated approval, and the company has requested priority review, which if granted would shorten the FDA’s review to six months following a 60-day filing check. The dossier rests on the Phase I/II programme — a randomised US study of 26 patients, an open-label European study of 13, and further cohorts totalling 18 — measured against a propensity-score-matched external control drawn from the Enroll-HD natural history database. On that comparison, uniQure’s release says the therapy demonstrated a slowing of disease progression. AMT-130 holds FDA Breakthrough Therapy, Regenerative Medicine Advanced Therapy and Fast Track designations.

The external control is the thing to hold onto. Huntington’s progresses slowly and predictably enough that a matched historical cohort is a defensible comparator, and the FDA accepted that the Phase I/II data could support a filing. It is still not a randomised comparison against placebo, which is the circumstance accelerated approval exists to address. A filing is not an approval, and the review clock has not started.