Why uniQure Stock Is Trading Lower After FDA Meeting Update

uniQure N.V. (NASDAQ:QURE) stock is trading lower on Thursday.

• QURE shares are retreating from recent levels. Check the market position here.

The gene therapy company received final meeting minutes from the U.S. Food and Drug Administration regarding a pre-Biologics License Application (BLA) meeting held in October to discuss the application for AMT-130, an investigational gene therapy for Huntington’s disease (HD). 

In the final meeting minutes, and consistent with uniQure’s November press release, the FDA conveyed that data submitted from the Phase 1/2 studies of AMT-130 are currently unlikely to provide the primary evidence to support a BLA submission.

uniQure is evaluating the feedback and plans to urgently request a follow-up meeting with the FDA to take place in the first quarter of 2026.

William Blair on Thursday wrote, “We still view the agency's change in guidance as very surprising given the numerous interactions uniQure has had with the FDA since its November 2024 meeting.”

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Analyst Sami Corwin adds that “Furthermore, the trial design aligns with the FDA's recent plausible mechanism pathway publication and draft guidance on gene therapies for small populations, which specifically stipulates that external controls may be an appropriate option in certain contexts.”

Given that the path to and timing of BLA submission for AMT-130 are still unclear, William Blair writes that uniQure stock will remain under pressure until there is a line of sight to a reasonable and expeditious path to regulatory submission.

QURE Price Action: uniQure stock is down 11.24% at $22.60 at publication on Thursday.

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