uniQure, a leading gene therapy company advancing transformative therapies for patients with severe medical needs, has announced that, during a recent type B meeting with the US Food and Drug Administration (FDA), they communicated that the three-year analysis from the phase 1/2 study would be acceptable as the primary basis of a Biologics License Application (BLA) for the accelerated approval of AMT-130 in Huntington’s disease.
In addition, the FDA seeks to align on the confirmatory study design prior to the BLA submission, including consideration of concurrent control on standard-of-care therapy instead of a sham procedure. FDA communicated that they would work as expeditiously as possible with uniQure on this effort.
The company is committed to conducting the confirmatory study without delay and expects to further align with the FDA on the details of such a study prior to BLA submission. The company intends to submit the BLA in the third quarter of 2026.
‘Today’s announcement reflects the outcome we have worked toward throughout our continued regulatory engagement with FDA, and we are deeply grateful for FDA’s genuine commitment to addressing the unmet need of Americans living with Huntington’s disease’, said Matt Kapusta, chief executive officer at uniQure.
The company expects to receive final minutes within 30 days of the recent type B meeting.
AMT-130 has been granted Regenerative Medicine Advanced Therapy (RMAT) designation by the FDA – the first RMAT designation for Huntington’s disease – as well as breakthrough therapy designation and Fast Track designation.
About the phase 1/2 clinical programme of AMT-130
uniQure is conducting two multi-centre, dose-escalating, phase 1/2 clinical studies to explore the safety, tolerability, and exploratory efficacy signals of AMT-130 for the treatment of Huntington’s disease. Based on interactions with the FDA, it was agreed that data from cohorts one and two in the phase 1/2 studies could be compared to a propensity score-matched external control derived from the Enrol-HD natural history data set, under a prespecified statistical analysis plan, which may serve as the primary basis for a BLA submission.
In the US study, a total of 26 patients with early manifest Huntington’s disease were randomised to treatment (n=6 low dose; n=10 high dose) or an imitation (sham) procedure (n=10). Treated patients received a single administration of AMT-130 through MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum (caudate and putamen).
The study consists of a blinded 12-month core study period followed by unblinded long-term follow-up of treated patients for five years. An additional four control patients crossed over to treatment. The European open-label phase 1b/2 study of AMT-130 enrolled 13 patients with early manifest Huntington’s disease (n=6 low dose; n=7 high dose).
A third cohort enrolled an additional 12 patients across sites in the US and EU. This cohort was randomised to explore both doses of AMT-130 in combination with immunosuppression, using the current, established stereotactic administration procedure.
A fourth US-based cohort enrolled six patients and is evaluating high-dose AMT-130 in patients with lower striatal volumes compared to those of patients enrolled in previous cohorts.
