FDA Grants Priority Review to uniQure's Huntington's Gene Therapy AMT-130, Decision Due May 2027
On November 2, 2026, uniQure announced that the FDA has accepted its biologics license application (BLA) for ifezuntirgene inilparvovec (AMT-130), a one-time gene therapy for Huntington's disease, and granted it Priority Review. The FDA set a decision date of May 1, 2027. If approved, it would be the first gene therapy for Huntington's disease.
The application was filed under the accelerated approval pathway. The FDA also said it tentatively plans to discuss the application at an advisory committee meeting, according to uniQure's press release. No meeting date has been set.
How AMT-130 works
Huntington's disease is caused by an expanded CAG repeat in the HTT gene, which makes a toxic form of the huntingtin protein that slowly destroys brain cells. It affects about 75,000 people in the US, Europe and the UK, and there is no approved treatment that slows it down.
AMT-130 uses an AAV5 vector to deliver a microRNA that silences the huntingtin gene, including a potentially highly toxic exon 1 protein fragment. It's given once, through MRI-guided neurosurgery that infuses the therapy directly into the striatum (the caudate and putamen), the brain region hit earliest and hardest by the disease.
What the data show
The BLA rests on 36-month results from uniQure's Phase I/II program, in which 51 patients were dosed across US and European studies. The comparison is against a propensity score-matched external control drawn from the Enroll-HD natural history database, not a randomized placebo group. In the high-dose group at 36 months, as NeurologyLive reported:
Composite Unified Huntington's Disease Rating Scale (cUHDRS): 75% slowing of disease progression (P = .003).
Total Functional Capacity (TFC): a 60% difference (P = .033).
Cerebrospinal fluid neurofilament light (NfL), a marker of nerve damage: 8.2% below baseline.
uniQure says 3-year follow-up data presented in 2026 showed continued slowing on cUHDRS and no new long-term safety concerns.
A bumpy road to filing
The path here was not smooth. In December 2024, uniQure said the FDA agreed the Phase I/II data compared with an external control could support an accelerated approval filing. In November 2025, after the 36-month results were out, the FDA reversed course and said the data would not be enough at that time. In June 2026, uniQure said the FDA confirmed the 36-month analysis would be acceptable as the primary basis for a BLA, and the company submitted in September 2026.
What happens next
An FDA advisory committee meeting is tentatively planned before the May 1, 2027 decision.
A confirmatory trial of about 200 patients, randomized 2:1 to AMT-130 or standard of care, with TFC at 36 months as the main endpoint. US screening is expected to begin by the end of 2026.
AMT-130 already holds Breakthrough Therapy, RMAT and Fast Track designations. It is the first Huntington's therapy to receive both Breakthrough and RMAT.
Why it matters
Huntington's disease has had no disease-modifying treatment. A one-time therapy that slows decline would be a major change for families who often watch the disease pass through generations. The open question for the FDA is whether an external-control comparison is convincing enough, and the advisory committee will be the place to watch.
For readers new to AAV vectors and how they reach the brain, The Life-Changing Power of Gene Therapy (affiliate link) walks through the basics.
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