UniQure submitted applications to the FDA and the UK’s MHRA seeking approval for AMT-130, its gene therapy for Huntington’s disease, after previously disclosed three-year data showed slowed disease progression. The company asked the FDA for priority review, targeting a decision in about eight months, and is pursuing accelerated approval, with confirmatory evidence planned later. The filing follows a contentious period in which the FDA requested a new double-blind trial design including a sham surgery control, after UniQure’s earlier analysis relied on comparisons to the external Enroll-HD natural history database. UniQure says the new regulatory submissions reflect the agency’s demands and its updated path to demonstrate clinical benefit. For the field, the AMT-130 review underscores how regulator expectations around internal controls, trial design integrity, and evidence packages remain decisive for first-in-class neurogenetics programs—particularly for therapies requiring intensive procedures like hours-long surgeries.