uniQure (QURE) Stock: Falls 3% Despite FDA Priority Review for Huntington’s Disease Gene Therapy

TLDR

• QURE stock falls 2.98% to $24.09 despite FDA Priority Review for AMT-130.
• FDA sets May 1, 2027, as the target decision date for uniQure’s gene therapy.
• uniQure plans to begin screening 200 patients for its confirmatory trial by year-end.
• AMT-130 targets Huntington’s disease through uniQure’s proprietary gene-silencing platform.
• QURE shares break below $24.83 support despite a major regulatory milestone.

uniQure (QURE) shares declined 2.98% to $24.09 on Monday, losing $0.74 during the trading session. The stock extended its recent losses despite a major regulatory milestone for its experimental Huntington’s disease treatment. The decline followed a break below the $24.83 support level, as the company advanced its gene therapy application through the FDA review process.

uniQure N.V., QURE

FDA Grants Priority Review to uniQure’s Huntington’s Disease Treatment

The U.S. Food and Drug Administration (FDA) accepted uniQure’s Biologics License Application (BLA) for ifezuntirgene inilparvovec. The investigational gene therapy targets Huntington’s disease, a progressive inherited disorder that affects movement, cognition, and behavior. The agency granted the application Priority Review under the Accelerated Approval pathway, setting May 1, 2027, as its target decision date.

The FDA indicated that it tentatively plans to discuss the application at an Advisory Committee meeting. However, 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 application relies on 36-month results from uniQure’s Phase I/II clinical program. The company compared treated patients with a propensity score-matched external control from the Enroll-HD natural history database. uniQure previously announced the analysis on September 24, 2025, providing the clinical evidence supporting its submission.

AMT-130 Confirmatory Trial Targets 200 Huntington’s Disease Patients

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

The trial will use Total Functional Capacity (TFC) as its primary endpoint at 36 months. This measure evaluates patients’ ability to perform everyday activities and maintain functional independence. uniQure plans to begin patient screening in the United States before the end of 2026.

The confirmatory study remains an important part of the regulatory process. The company expects the trial to progress substantially by the time of potential approval. This approach will provide additional clinical evidence on the treatment’s effectiveness and safety.

uniQure Advances Gene Therapy Platform With Multiple FDA Designations

Ifezuntirgene inilparvovec, also known as AMT-130, uses uniQure’s proprietary miQURE gene-silencing platform. The treatment incorporates a microRNA designed to reduce huntingtin gene activity and the potentially toxic exon 1 protein fragment. It requires a single administration through MRI-guided neurosurgical delivery directly into the brain’s striatum.

Furthermore, AMT-130 holds Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations from the FDA. 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. Four control participants later crossed over to treatment after approximately 12 months.

The European open-label study included another 13 patients across low-dose and high-dose groups. Additionally, 12 patients received both doses alongside immunosuppression in a separate cohort. A further six U.S. patients are evaluating high-dose treatment with lower striatal volumes.

Despite the FDA’s Priority Review decision, QURE shares remained under selling pressure during Monday’s session. The stock’s decline below $24.83 leaves that former support level as an important price reference. Meanwhile, the May 1, 2027, FDA deadline establishes the next major regulatory milestone for uniQure.

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