FDA backs uniQure's three-year data package for AMT-130 accelerated approval filing

The Netherlands-based [uniQure N.V. (Nasdaq: QURE) has announced plans](https://app.allsci.com/news/ASC-NR-0000003833756-1.0-1781695267) to submit a Biologics License Application (BLA) to the US FDA seeking accelerated approval of [AMT-130 ](https://app.allsci.com/drugs/ASC-DR-0000000034330-1.0-1772718717)for the treatment of Huntington's disease, with the filing targeted for Q3 2026. The decision follows a Type B meeting in which the FDA indicated that three-year data from the company's Phase I/II studies could serve as the primary basis for the application, marking a significant shift from the agency's position earlier this year. The filing seeks accelerated approval for AMT-130 in patients with early manifest Huntington's disease, the company said. No dosing regimen details specific to the BLA were disclosed beyond the single-administration approach evaluated in the trials. AMT-130 holds Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA — the first such designation granted for Huntington's disease — as well as Breakthrough Therapy and Fast Track designations. The FDA indicated it would work expeditiously with uniQure to align on the design of a required confirmatory study prior to submission, with the agency considering a concurrent standard-of-care control arm rather than a sham surgical procedure as the comparator. uniQure said it expects to receive final meeting minutes within 30 days of the Type B meeting. The evidentiary basis for the BLA rests on data from two multi-center, dose-escalating [Phase I/II clinical studies](https://clinicaltrials.gov/study/NCT04120493) of AMT-130. In the US study, 26 patients with early manifest Huntington's disease were randomized to treatment (six low dose, ten high dose) or a sham procedure (ten patients), with an additional four control patients crossing over to treatment. A European open-label Phase I/II study enrolled 13 additional patients. Per an agreement with the FDA, data from cohorts 1 and 2 were compared to a propensity score-matched external control derived from the Enroll-HD natural history dataset under a prespecified statistical analysis plan. [Three-year results](https://app.allsci.com/news/ASC-NR-0000000577411-1.0-1774444659) showed a statistically significant 75% slowing of disease progression at the high dose as measured by the composite Unified Huntington's Disease Rating Scale compared to the matched external control (p\=0.003), with a key secondary endpoint of Total Functional Capacity demonstrating a statistically significant 60% slowing (p\=0.033). A mean reduction of 8.2% from baseline in cerebrospinal neurofilament light protein was also observed at 36 months in the high-dose group. AMT-130 was reported to be generally well-tolerated, with no new drug-related serious adverse events observed since December 2022. The regulatory path to this point has been notably turbulent. Following a pre-BLA meeting in October 2025, the FDA communicated in November 2025 that it no longer agreed the Phase I/II data compared to an external control were adequate to provide primary evidence of effectiveness. A [Type A meeting held in January 2026](https://app.allsci.com/news/ASC-NR-0000000562700-1.0-1774444644) confirmed that position, with the FDA strongly recommending a prospective, randomized, double-blind, sham surgery-controlled study. That stance triggered a sharp share price decline. The [March 2026 regulatory update](https://app.allsci.com/news/ASC-NR-0000000563162-1.0-1774444644) indicated uniQure would seek a Type B meeting to explore alternative study design approaches. This week's announcement represents a materially different outcome from that engagement, with the FDA now indicating the three-year dataset can support an accelerated approval pathway — contingent on alignment over the confirmatory study design. The confirmatory study design remains a point of active negotiation. The FDA's shift from requiring a sham surgery-controlled design to considering a concurrent standard-of-care control arm is clinically and operationally significant. A sham procedure arm had been a central point of contention, with uniQure raising ethical objections and the FDA disputing the company's characterization of what the procedure would entail. The resolution of this dispute, and the specific design of the confirmatory trial, will be material to the long-term commercial and clinical trajectory of AMT-130. AMT-130 uses an adeno-associated virus serotype 5 (AAV5) vector to deliver a microRNA targeting the huntingtin gene transcript, administered via MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum. The approach is designed to reduce production of mutant huntingtin protein, the toxic species driving neurodegeneration in Huntington's disease — a rare, autosomal dominant condition caused by a CAG repeat expansion in the huntingtin gene affecting approximately 75,000 people across the US, EU, and UK. There are currently no approved therapies that modify the course of the disease. uniQure is also [pursuing a Marketing Authorization Application with the UK MHRA](https://app.allsci.com/news/ASC-NR-0000002346778-1.0-1777547168), also targeted for Q3 2026 and based on the same three-year dataset. AMT-130 enters a field where no disease-modifying therapy has yet been approved for Huntington's disease. Several huntingtin-lowering approaches remain in development, including Roche's tominersen and programs from Wave Life Sciences and [Harness Therapeutics](https://app.allsci.com/news/ASC-NR-0000000534468-1.0-1774444614). AMT-130 is differentiated by its one-time surgical administration and gene-silencing mechanism, although its invasive delivery procedure and the design of the required confirmatory study remain key regulatory and commercial considerations. *** This article was generated with AI assistance and reviewed and edited by the AllSci editorial team Explore more at AllSci News: [https://allsci.com/news/](https://allsci.com/news/) --- Spot something wrong? [Report an issue with this article](https://newsgen-prod.reframedata.com/feedback/amt-130-fda-approval-backs-uniqures-three-year)