First gene therapy that could slow Huntington's disease gets fast-track FDA review
The US Food and Drug Administration has agreed to review uniQure's AMT-130 gene therapy under its fast routes and will decide by 1 May 2027. There is currently no treatment that slows Huntington's. The caveat: the data compare treated patients with an external control group rather than a randomised trial, and the confirmatory study has not started yet.
One operation on the brain and, three years later, a disease that is progressing more slowly. That is what biotech company uniQure, based in Amsterdam and Lexington, Massachusetts, wants to prove with AMT-130, its gene therapy for Huntington’s disease, an inherited condition that destroys nerve cells and for which no treatment can slow the decline.
The US Food and Drug Administration (FDA) has accepted its application for approval, the company announced on 2 November. The agency will review it under the accelerated approval pathway with priority review, and has set a target date of 1 May 2027. It also plans to consult an advisory committee of outside experts.
A microRNA that switches the gene off
The therapy is given once, through MRI-guided neurosurgery, into the striatum, the part of the brain the disease hits hardest. It carries a microRNA designed to silence the huntingtin gene, which in these patients makes a toxic protein.
According to data uniQure presented in September, the 15 patients who received the high dose declined 80% more slowly than a comparison group at 36 months, on a scale that combines movement, thinking and daily living. At 48 months, with 12 patients, that difference fell to 44% and was no longer statistically significant; on the functional capacity scale it held at 61%.
What still has to be confirmed
The comparison group did not receive a placebo: they are patients from a large international database, Enroll-HD, matched to those treated. That is why the FDA is asking for a confirmatory trial of about 200 patients, two in three receiving the therapy, which is due to start screening in the US before the end of 2026.
In all, 51 people have received the therapy in two phase 1/2 trials in the US and Europe. It “has the potential to become the first disease modifying treatment for Huntington’s disease”, said uniQure’s chief executive, Matt Kapusta.
The next step is the FDA advisory committee meeting, which has not been scheduled yet.
