Simultaneous Regulatory Filings in the U.S. and U.K.

uniQure N.V. (QURE) submitted a Biologics License Application (BLA) to the FDA and a Marketing Authorization Application (MAA) to the U.K. MHRA for its gene therapy AMT-130 for Huntington’s disease on September 2, 2026. The brand name has not yet been assigned, and the International Nonproprietary Name (INN) is ifezuntirgene inilparvovec, with the developmental code name AMT-130. The FDA application requested accelerated approval and priority review. If granted priority review, the 60-day filing review would be followed by a 6-month review period, with a total decision expected in approximately 8 months. This is not final approval, as regulatory acceptance and review remain pending.

Single-Dose Therapy That Directly Lowers HTT

AMT-130 is a non-selective huntingtin-lowering therapy that uses an AAV5 vector and miQURE microRNA to suppress the expression of the huntingtin gene (HTT) and toxic exon 1 protein fragments. It is administered once via MRI-guided stereotactic surgery to the putamen and globus pallidus, fundamentally differing from oral symptomatic treatments. The U.S. randomized and European open-label trials are Phase 1/2, including 26 U.S. and 13 European patients, plus 12 patients receiving concomitant immunosuppression and 6 patients with small striatal volumes for additional evaluation. The invasive brain surgery and small sample size are key variables in commercial infrastructure and data interpretation.

3-Year Data and Regulatory Reversal

The approval package is based on a 3-year analysis comparing the treatment group to an external control group matched by Enroll-HD natural history data using propensity scores. The high-dose group delayed disease progression by 75% compared to the control group, with an average change of -0.38 vs. -1.52 and a p-value of 0.003 on the Unified Huntington’s Disease Rating Scale (cUHDRS). In March 2026, the FDA expressed difficulty in meeting the key evidence threshold for efficacy with external control data alone and requested an additional double-blind trial. However, in a Type B meeting in June and a final meeting in July, the agency agreed to use the existing Phase 1/2 data as the primary basis for the accelerated approval application. The confirmatory trial will use a standard-of-care concurrent control group instead of a sham surgery, improving ethical considerations and patient recruitment feasibility.

Market Potential of the First Disease-Modifying Therapy

There are approximately 75,000 patients with Huntington’s disease in the U.S., the European Union, and the U.K., and no therapies have yet been approved to slow disease progression. Current standard-of-care treatments include Xenazine’s tetrabenazine, Teva Pharmaceutical Industries (TEVA)’s Austedo and Austedo XR (deutetrabenazine), and Neurocrine Biosciences (NBIX)’s Ingrezza (valbenazine), which only alleviate chorea symptoms. Ingrezza received FDA approval on August 18, 2023, Austedo in 2017, and Xenazine in 2008. The global Huntington’s disease treatment market was estimated at $1.39 billion in 2025, with competitive development programs including Roche (RHHBY)’s Phase 2 tominersen and Novartis (NVS)’s Phase 3 botulinum toxin, which will test AMT-130’s single-dose value against surgical burden.