RegulatoryCompany release

FDA accepts uniQure's Huntington's gene therapy filing with priority review, sets May 2027 action date

🇺🇸 uniQure N.V. (QURE) · Mon 02 Nov 2026

The agency has accepted the accelerated approval application for AMT-130 and tentatively plans an advisory committee discussion, with a decision due by 1 May 2027.

uniQure announced on 2 November 2026 that the US Food and Drug Administration has accepted its Biologics License Application for ifezuntirgene inilparvovec (AMT-130), a gene therapy for Huntington's disease. The company submitted the application under the Accelerated Approval pathway.

The FDA granted Priority Review and set a PDUFA target action date of 1 May 2027. The agency also tentatively plans an advisory committee discussion, although the summary provided does not say when it might take place.

The filing rests on 36-month data from a Phase I/II programme, compared with an external control. Applications built on external comparators rather than concurrent randomised controls often draw close regulatory scrutiny of how the comparison group was selected and matched.

A confirmatory randomised trial of about 200 patients is expected to begin screening by the end of 2026. Under accelerated approval, which allows authorisation on the basis of a surrogate or intermediate endpoint reasonably likely to predict clinical benefit, sponsors are generally required to verify that benefit in further studies.

What it means for your team

  • Regulatory teams should note the 1 May 2027 PDUFA date and the planned advisory committee, which could shape the review timeline and public debate.
  • The external-control design is likely to be a focal point of review, relevant to sponsors considering similar approaches in rare neurological disease.
  • Market-access teams can start horizon scanning for a potential first gene therapy in Huntington's disease, though the outcome depends on the FDA decision.
  • The confirmatory trial's progress will matter for any accelerated approval, so post-approval evidence planning should start early.

Company's press release (SEC filing): uniQure N.V. ↗

Article written by VigiEye from the official source (uniQure N.V.). Claims are the company's own. AI-assisted; check the source before acting.

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