uniQure N.V. has received an important regulatory milestone in its effort to develop a gene therapy for Huntington’s disease after the US Food and Drug Administration (FDA) accepted its Biologics License Application (BLA) for ifezuntirgene inilparvovec.
The FDA has granted the application Priority Review under the Accelerated Approval pathway, with a target decision date of May 1, 2027. If approved, the treatment could become the first disease-modifying gene therapy for Huntington’s disease.
The FDA has also indicated that it is tentatively planning to discuss the application at an Advisory Committee meeting. The final date and details of the meeting have not yet been confirmed.
Ifezuntirgene inilparvovec is an investigational gene therapy designed to address the underlying cause of Huntington’s disease. The treatment uses a modified adeno-associated virus (AAV) delivery system to introduce genetic material into brain cells, with the aim of reducing the production of huntingtin protein, which plays a central role in the development and progression of the disease.
Huntington’s disease is a rare, inherited neurological disorder caused by a mutation in the huntingtin (HTT) gene. The condition gradually damages nerve cells in the brain and affects movement, thinking and behaviour. Patients may experience involuntary movements, difficulty with coordination, memory problems and changes in personality.
There is currently no approved treatment that can stop or reverse the progression of Huntington’s disease, creating a significant need for therapies that can target the disease at its source.
The BLA submission is supported by 36-month data from uniQure’s Phase I/II clinical study. The company compared patient outcomes with an external control group developed using data from the Enroll-HD natural history database.
According to the company, the findings provide clinical evidence supporting the potential of ifezuntirgene inilparvovec to slow disease progression. The application was supported by data previously announced on September 24, 2025.
Matt Kapusta, Chief Executive Officer of uniQure, said the FDA’s acceptance of the application represents an important milestone for the Huntington’s disease community. He highlighted the potential of the therapy to become the first disease-modifying treatment for the condition.
Alongside the regulatory review, uniQure is preparing a confirmatory clinical trial in agreement with the FDA. The planned study will enrol approximately 200 patients and compare ifezuntirgene inilparvovec with standard care.
Participants will be assigned in a 2:1 ratio, with more patients receiving the gene therapy than standard care. The primary endpoint will measure changes in Total Functional Capacity (TFC) at 36 months. This assessment evaluates a patient’s ability to perform everyday activities, including work, household tasks and personal care.
The company expects to begin screening patients in the United States by the end of 2026. The confirmatory study is expected to be well underway by the time a potential regulatory approval is granted.
Ifezuntirgene inilparvovec has also received several important designations from the FDA, including Breakthrough Therapy, Regenerative Medicine Advanced Therapy (RMAT) and Fast Track status. These designations are intended to support the development and review of treatments that may address serious medical conditions.
The Priority Review decision brings uniQure closer to a potential regulatory milestone in Huntington’s disease treatment. However, the therapy remains investigational, and its approval will depend on the FDA’s assessment of the submitted clinical evidence and other regulatory requirements.
If approved, the treatment could represent a major development in gene therapy research and offer new hope to patients and families affected by this progressive neurological condition.