FDA Accepts Review of First Huntington's Disease Gene Therapy With Priority Review, Decision Due May 1, 2027
The U.S. Food and Drug Administration (FDA) has accepted for review uniQure's Biologics License Application (BLA) for ifezuntirgene inilparvovec and granted it Priority Review. uniQure announced the news on November 2 from Lexington, Massachusetts, and Amsterdam: the application was submitted under the Accelerated Approval pathway, with a Prescription Drug User Fee Act (PDUFA) target action date of May 1, 2027. If approved, the therapy would become the world's first disease-modifying treatment for Huntington's disease.
In its acceptance letter, the FDA said it is tentatively planning to discuss the application at an Advisory Committee meeting, and will notify the company once the date and timelines are finalized. "The FDA's acceptance of our BLA for review marks an important milestone for the Huntington's disease community, which has waited generations for a treatment that changes the course of this devastating disease," said Matt Kapusta, chief executive officer of uniQure. He said the company will work with the agency, clinicians and the Huntington's community to support the review process.
The BLA is supported by the 36-month data analysis from the therapy's Phase I/II clinical studies, announced on September 24, 2025, compared against a propensity score-matched external control drawn from the Enroll-HD natural history database. In total, the two multi-center Phase I/II studies have dosed 51 patients, including 26 in the U.S. randomized study and 13 in the European open-label study, with further cohorts exploring dosing and immunosuppression.
The company and the FDA have aligned on the design of the confirmatory study: a randomized, standard-of-care trial enrolling approximately 200 patients in a 2:1 ratio of treatment to standard of care, with Total Functional Capacity (TFC) as the primary endpoint at 36 months. uniQure plans to initiate patient screening in the U.S. by the end of 2026, and expects the study to be well underway by the time of a potential approval.
Huntington's disease is a rare, inherited neurodegenerative disorder caused by a CAG repeat expansion in the first exon of the huntingtin gene. It leads to motor symptoms including chorea, behavioral abnormalities and cognitive decline, with progressive physical and mental deterioration. According to research cited by uniQure, approximately 75,000 people have Huntington's disease in the U.S., the EU and the UK, with hundreds of thousands more at risk of inheriting it, and no approved therapy can yet delay onset or slow progression. The therapy is delivered once, directly into the striatum (caudate and putamen), through MRI-guided, convection-enhanced stereotactic neurosurgery. It is the first investigational therapy for Huntington's disease to have received Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT) and Fast Track designations from the FDA.
Source: GSM · USA (original Chinese edition)
Photo: 图：美国食品药品监管局（FDA）位于马里兰州 Silver Spring 白橡树园区的总部入口（资料图）。美国食品药品监管局摄，公共领域，经 Wikimedia Commons。 / The entrance to FDA headquarters at the White Oak campus in Silver Spring, Maryland (file photo). Photo by the U.S. Food and Drug Administration, public domain, via Wikimedia Commons.
