uniQure N.V. (QURE) shares declined 2.98% to $24.09 on Monday, losing $0.74 during the trading session. The drop extended recent losses despite a major regulatory milestone for the company's experimental Huntington's disease treatment, and it followed a break below the $24.83 support level as the company's gene therapy application advanced through the U.S. Food and Drug Administration (FDA) review process.

FDA Grants Priority Review, Sets May 2027 Target Decision Date

The FDA accepted uniQure's Biologics License Application (BLA) for ifezuntirgene inilparvovec, an investigational gene therapy targeting Huntington's disease, a progressive inherited disorder that affects movement, cognition, and behavior. Available treatments for the disorder address its symptoms rather than its underlying cause, a mutation in the huntingtin gene. The agency granted the application Priority Review under the Accelerated Approval pathway, setting May 1, 2027, as the target decision date. Priority Review directs the agency to act on an application on an expedited timetable, while the Accelerated Approval pathway permits approval based on evidence reasonably likely to predict clinical benefit, pending verification in confirmatory trials.

The FDA also indicated that it tentatively plans to discuss the application at an Advisory Committee meeting, although the agency has not finalized the meeting date or schedule. The regulatory review marks an important step toward potential approval of the experimental treatment.

The filing relies on 36-month results from uniQure's Phase I/II clinical program, in which treated patients were compared with a propensity score-matched external control group drawn from the Enroll-HD natural history database. Enroll-HD is a long-running observational study of people with Huntington's disease, and propensity score matching pairs treated patients with untreated individuals of similar baseline characteristics, providing a comparison group in place of a concurrent placebo arm. uniQure announced that analysis on September 24, 2025, and it forms the clinical evidence supporting the submission.

Confirmatory Trial to Enroll Roughly 200 Patients

uniQure has aligned with the FDA on key elements of its planned confirmatory clinical trial. The study will enroll approximately 200 patients and compare the gene therapy against standard care, with participants allocated in a 2:1 ratio between treatment and standard care.

Total Functional Capacity (TFC), a measure of patients' ability to perform everyday activities and maintain functional independence, will serve as the primary endpoint at 36 months. uniQure plans to begin patient screening in the United States before the end of 2026, and the company expects the trial to progress substantially by the time of any potential approval. The confirmatory study is intended to provide additional clinical evidence on the treatment's effectiveness and safety and remains an important part of the regulatory process. Because the BLA is seeking Accelerated Approval, results from this study would serve as the verification step for the clinical benefit suggested by the external-control analysis.

AMT-130 Built on Proprietary Gene-Silencing Platform

Ifezuntirgene inilparvovec, also known as AMT-130, is developed on uniQure's proprietary miQURE gene-silencing platform. The therapy incorporates a microRNA designed to reduce huntingtin gene activity and the potentially toxic exon 1 protein fragment, an approach intended to reduce production of the disease-associated protein at its source. It requires a single administration through MRI-guided neurosurgical delivery directly into the brain's striatum.

AMT-130 holds Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations from the FDA, and the agency has also granted the treatment Fast Track status. These designations reflect the regulatory support surrounding its development for Huntington's disease.

uniQure has administered AMT-130 to 51 patients across its Phase I/II clinical studies. The U.S. randomized study enrolled 26 patients, including 16 treatment recipients and 10 participants assigned to sham procedures, with four control participants later crossing over to treatment after approximately 12 months. The European open-label study included another 13 patients across low-dose and high-dose groups. In addition, 12 patients received both doses alongside immunosuppression in a separate cohort, and a further six U.S. patients are evaluating high-dose treatment with lower striatal volumes.

Next Milestones for uniQure

Despite the FDA's Priority Review decision, QURE shares remained under selling pressure during Monday's session. The stock's move below $24.83 leaves that former support level as an important price reference, while the May 1, 2027 target decision date stands as the next major regulatory milestone for uniQure. The tentative Advisory Committee discussion and the planned start of U.S. confirmatory-trial screening before the end of 2026 round out the calendar of regulatory markers ahead of the decision.

Source: uniQure (QURE) Stock: Falls 3% Despite FDA Priority Review for Huntington's Disease Gene Therapy — Blockonomi