The US Food and Drug Administration (FDA) has accepted a biologics licence application (BLA) under its accelerated approval pathway from uniQure for ifezuntirgene inilparvovec, an investigational gene therapy to treat Huntington’s disease.
The submission has been granted Priority Review, setting a target action date of 1 May 2027 under the Prescription Drug User Fee Act (PDUFA).
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The regulator also indicated provisional plans to convene an Advisory Committee meeting regarding the application, with timing to be finalised later.
uniQure CEO Matt Kapusta said: “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.
“Backed by compelling data from our Phase I/II studies, we believe ifezuntirgene inilparvovec has the potential to become the first disease-modifying treatment for Huntington’s disease. With the FDA’s priority review underway, we will work in conjunction with the agency, clinicians, and the Huntington’s community to support the review process.
“The confirmatory trial remains a key priority, with screening anticipated to begin by year-end and the study is expected to be well underway by the time of potential approval.”
The filing relies on 36-month findings from Phase I/II study compared with an external cohort from the Enroll-HD natural history registry, reported on 24 September 2025.
In addition, uniQure has aligned with the FDA on key parameters for a confirmatory clinical study.
This randomised standard-of-care trial will enrol around 200 patients in a 2:1 ratio favouring ifezuntirgene inilparvovec, assessing Total Functional Capacity at 36 months as its primary endpoint.
Patient screening is scheduled to begin in the US before the end of this year.
Also known as AMT-130, ifezuntirgene inilparvovec received breakthrough therapy and regenerative medicine advanced therapy designations from the FDA, alongside fast track status.
Delivered via a single, MRI-guided neurosurgical administration directly into the striatum, the therapy uses microRNA to silence the huntingtin gene and its exon 1 protein fragment.
Huntington’s disease is an inherited disorder that causes motor symptoms, including chorea, behavioural changes, and progressive cognitive decline.
