FDA accepts uniQure's Huntington's gene therapy for priority review

What's the news? On 2 November 2026, uniQure announced that the US Food and Drug Administration (FDA) has accepted its application (a Biologics License Application, the US marketing filing) for ifezuntirgene inilparvovec, also known as AMT-130, a gene therapy for Huntington's disease. The FDA granted the filing Priority Review, which shortens the review timeline, and set a decision date (PDUFA target action date) of 1 May 2027. The application was submitted under the FDA's Accelerated Approval pathway, designed to speed up access to therapies for serious conditions based on promising early evidence.

Why it matters: If approved, AMT-130 would be the world's first disease-modifying treatment for Huntington's disease, a fatal inherited condition in which a faulty gene produces a toxic protein that destroys brain cells. Today only symptom-relief drugs exist. The filing is supported by 36-month data from the company's Phase I/II clinical study, in which patients received a single dose of the therapy, compared against a matched group from a large natural-history database. The FDA has said it tentatively plans to discuss the application at an advisory committee meeting; uniQure expects the confirmatory trial needed for full approval to start screening before the end of 2026. In total, 51 patients have been dosed across the Phase I/II studies to date.

Read more: uniQure press release, 2 November 2026