FDA accepts uniQure's BLA for Huntington's gene therapy, sets priority review

uniQure N.V. said on November 2, 2026 that the FDA has accepted for review the Biologics License Application for ifezuntirgene inilparvovec, an investigational gene therapy for Huntington's disease submitted under the Accelerated Approval pathway.¹ The application has been granted Priority Review with a PDUFA target action date of May 1, 2027.¹

In its acceptance letter, the FDA stated that it is tentatively planning to discuss this application at an Advisory Committee meeting.¹ uniQure said it will be notified once the date and timelines have been finalized.¹

The BLA is supported by the previously announced 36-month data analysis from the Phase I/II clinical study of ifezuntirgene inilparvovec, compared to a propensity score-matched external control derived from the Enroll-HD natural history database, as previously announced on September 24, 2025.¹

On the confirmatory trial, uniQure and the FDA have aligned on key elements of the study design, which is a randomized, standard-of-care trial enrolling approximately 200 patients in a 2:1 ratio of ifezuntirgene inilparvovec to standard of care, with Total Functional Capacity as the primary endpoint at 36 months.¹ The company plans to initiate patient screening in the U.S. by the end of 2026.¹

Ifezuntirgene inilparvovec is the first investigational therapy for Huntington's disease to have received Breakthrough Therapy and Regenerative Medicine Advanced Therapy designations from the FDA, and it also holds Fast Track designation.¹

On the science, the therapy uses a proprietary gene-silencing miQURE platform incorporating a miRNA designed to silence the huntingtin gene and the toxic exon 1 protein fragment, delivered as a single administration via targeted, MRI-guided, convection-enhanced stereotactic neurosurgical delivery into the striatum.¹

Across the Phase I/II program, 51 patients have been dosed in total: 26 in the U.S. randomized study (6 low dose, 10 high dose, and 10 sham, with four crossovers), 13 in a European open-label study (6 low dose, 7 high dose), 12 in a cohort combining both doses with immunosuppression, and 6 in a cohort evaluating high dose in patients with lower striatal volumes.¹

Approximately 75,000 people have Huntington's disease across the U.S., EU, and UK, and there are currently no approved therapies to delay onset or slow progression.¹