FDA grants Priority Review to uniQure’s Huntington’s disease gene therapy

uniQure has received U.S. Food and Drug Administration acceptance and Priority Review for its Biologics License Application for ifezuntirgene inilparvovec, an investigational gene therapy for Huntington’s disease.

The application has been submitted under the Accelerated Approval pathway, with the FDA setting a Prescription Drug User Fee Act target action date of May 1, 2027.

If approved, ifezuntirgene inilparvovec could become the first disease-modifying treatment for Huntington’s disease.

The FDA has also indicated that it is tentatively planning to discuss the application at an Advisory Committee meeting, with further details expected once the timing is finalised.

The BLA is supported by 36-month data from uniQure’s Phase I/II clinical programme.

Those results compared patients treated with ifezuntirgene inilparvovec with a propensity score-matched external control derived from the Enroll-HD natural history database.

Matt Kapusta, Chief Executive Officer of uniQure, said acceptance of the application represents an important milestone for the Huntington’s disease community.

He said the company believes the Phase I/II findings support the potential for the therapy to alter the course of the disease and that uniQure will continue working with the FDA during the review.

The company is also preparing a confirmatory clinical study.

uniQure and the FDA have aligned on key elements of the trial design, which is expected to enrol approximately 200 patients.

Participants will be randomised in a 2:1 ratio to receive ifezuntirgene inilparvovec or standard of care.

The study’s primary endpoint will be Total Functional Capacity at 36 months.

Patient screening in the U.S. is expected to begin by the end of 2026.

Ifezuntirgene inilparvovec, previously known as AMT-130, is a gene therapy designed to reduce production of the huntingtin protein.

The therapy uses uniQure’s gene-silencing miQURE platform and incorporates a microRNA designed to silence the huntingtin gene as well as the potentially toxic exon 1 protein fragment.

Treatment is administered once through targeted MRI-guided, convection-enhanced stereotactic neurosurgical delivery directly into the striatum.

uniQure’s Phase I/II programme has treated 51 patients to date.

The U.S. randomised study enrolled 26 patients, including low-dose and high-dose treatment groups as well as a sham control group.

A European open-label study enrolled a further 13 patients, while additional cohorts have evaluated immunosuppression and treatment in patients with lower striatal volumes.

Ifezuntirgene inilparvovec has previously received Breakthrough Therapy, Regenerative Medicine Advanced Therapy and Fast Track designations from the FDA.

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Related Topics and Keywords

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