11/02/2026
Today, uniQure announced that the FDA has started its formal review of AMT-130, a one-time gene therapy for HD delivered through brain surgery. This review is the next step before any decision about approval.
The FDA granted Priority Review and set a target date of May 1, 2027 to finish its review. This date is a goal, not a guarantee of a decision or approval, and it may change.
The FDA also plans to hold an Advisory Committee meeting, where outside experts review the evidence and give the FDA advice. The FDA makes the final decision. People with HD and families may have a chance to share their experiences with the committee, and we’ll let you know how to take part once details are announced.
uniQure also shared details of its next study. About 200 people will take part, and 2 out of every 3 will receive AMT-130. The others will continue their usual care, with no placebo surgery.
The main measure will be daily function (TFC) after three years. Screening is expected to begin in the US by the end of 2026, with international sites to be announced in the first half of 2027.
To learn more, you can read the full press releases by visiting: https://hdsa.org/news/uniqure-announces-fda-acceptance-and-priority-review-of-the-biologics-license-application-for-ifezuntirgene-inilparvovec-for-the/ and:https://hdsa.org/news/uniqure-regulatory-bla-update-community-letter/
