The FDA has granted priority review to a Huntington’s disease gene therapy developed by uniQure, with a regulatory decision expected by 1 May 2027.
The US Food and Drug Administration (FDA) has accepted the company’s application seeking accelerated approval of AMT-130, now known as ifezuntirgene inilparvovec.
Priority review shortens the standard regulatory assessment period from 10 months to six. The FDA’s target decision date is 1 May 2027, but this does not mean the treatment has been approved or that approval is guaranteed.
UniQure submitted the application two months ago, seeking authorisation through the FDA’s accelerated approval pathway.
This allows a treatment to be approved based on preliminary evidence of effectiveness, with further clinical trial data required to confirm its benefits.
Matt Kapusta, chief executive of uniQure, said the FDA’s acceptance of the application was an important milestone for the Huntington’s disease community.
He added: “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.”
The FDA is also tentatively planning to discuss the application at an advisory committee meeting, where independent experts and patient representatives review the evidence and make recommendations.
UniQure has reached an agreement with the FDA on the design of a potential confirmatory trial, which could provide the additional evidence needed for full approval.
The study is expected to enrol approximately 200 people with Huntington’s disease, with two-thirds receiving the one-time gene therapy alongside standard care.
Patient screening in the US is expected to begin by the end of 2026.
Researchers will assess changes in daily functioning over three years using the Total Functional Capacity (TFC) scale.
The measure covers employment, finances, household tasks, daily activities and the level of care patients require.
Kapusta said: “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.”
A similar application has been submitted to regulators in the UK.
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