FDA sets May 2027 decision date on AMT-130 gene therapy for HD

On November 2nd, 2026, uniQure announced that the US Food and Drug AdministrationFood and Drug Administration The government regulatory authority in the US responsible for approving new drugs (FDA) has accepted the next step of its application for approval of AMT-130, now officially named ifezuntirgene inilparvovec. The FDA has also granted the application Priority Review status, setting a target date of May 1st, 2027 for its decision. This is an encouraging step towards what could become the first approved treatment designed to slow the progression of HD. But there’s still a road ahead as AMT-130 has not been approved, and the FDA has not yet concluded that it works.

What exactly has happened?

AMT-130 is a gene therapy designed to lower production of the huntingtin proteinhuntingtin protein The protein produced by the HD gene.. It is delivered directly into the brain through a one-time brain surgery procedure, with the aim of producing long-lasting effects. 

uniQure has submitted a Biologics License Application (BLA), essentially a detailed package of evidence asking the FDA for permission to market and sell the treatment in the United States. The FDA has now accepted that application for review and granted it Priority Review status, which shortens the agency’s usual review timeline (typically 6 months, rather than 10). Importantly, this doesn’t mean the FDA endorses the company’s conclusions, just that the formal evaluation can now begin.

The application uses the accelerated approval pathway, which can allow earlier approval of promising treatments for serious diseases, provided that data from larger and longer studies continues to show that the drug works. 

What evidence is the FDA reviewing?

The application draws on previously reported results from uniQure’s Phase 1/2 studies, including a three-year analysis suggesting that people receiving AMT-130 experienced slower disease progression than expected.

However, these findings come with important limitations. The main comparison was not against a traditional placeboplacebo A placebo is a dummy medicine containing no active ingredients. The placebo effect is a psychological effect that causes people to feel better even if they’re taking a pill that doesn’t work. control group. Instead, researchers compared treated participants with people from the Enroll-HD observationalobservational A study in which measurements are made in human volunteers but no experimental drug or treatment is given study who had similar characteristics. This approach can provide valuable insights, particularly for rare diseases, but differences between groups can influence the results. Furthermore, four-year data was hopeful but also complex. The FDA must now decide whether the available evidence is convincing enough to support approval.

Today’s announcement contains no new data from the ongoing clinical studies uniQure is running. It is instead an update on how the existing evidence will be reviewed, rather than additional proof that AMT-130 slows HD.

What happens next?

The FDA has set May 1st, 2027 as its target date to complete the review and take action. But this is not a guaranteed approval date. The agency could approve the treatment, request additional information, or decide that more evidence is needed. The FDA has also indicated that it is tentatively planning an advisory committee meeting, where independent experts could publicly discuss the evidence. No date has been confirmed for this meeting either.

Meanwhile, uniQure is preparing a larger confirmatory study involving approximately 200 participants. Around two-thirds will receive AMT-130, while the remainder will receive standard care for HD. Researchers will focus on comparing changes in Total Functional CapacityTotal Functional Capacity A standardized rating scale for function in HD, used to assess capacity to work, handle finances, perform domestic chores and self-care tasks, a measure of people’s ability to manage everyday activities, over three years. Participant screening is expected to begin in the United States before the end of 2026. This trial will be crucial for strengthening the evidence about whether AMT-130 genuinely changes the course of HD.

Hope, with important questions still to answer

For families waiting for treatments that might slow HD, this is a meaningful milestone. The possibility of a first disease-modifying treatment moving through regulatory review is exciting. But approval is not guaranteed, and questions remain about the strength of the evidence, long-term safety, and who might ultimately benefit. The next six months will be important. HDBuzz will continue following updates on the FDA review and confirmatory trial, watch this space. 

Summary: 

• The FDA has accepted uniQure’s application for AMT-130 (now called ifezuntirgene inilparvovec), an experimental gene therapy for HD, and granted it Priority Review, with a target decision date of May 1st, 2027.
• This is an important regulatory milestone, not an approval or new evidence that AMT-130 works. The FDA must still determine whether the existing clinical data are convincing enough to show that AMT-130 slows HD.
• A larger confirmatory trial involving around 200 participants is expected to begin screening by the end of 2026, helping establish whether AMT-130 truly slows HD progression.