June 17, 2026 —
uniQure plans to submit an approval application to the U.S. Food and Drug Administration for AMT-130, its investigational gene therapy for Huntington’s disease, following renewed alignment with the agency on the evidentiary package needed to support regulatory review.
According to the company, FDA staff agreed during a recent meeting that three-year data from a key clinical study may support the submission of a marketing application. uniQure expects to file the application between July and the end of September. The FDA has also requested that the company conduct an additional confirmatory trial to further evaluate the treatment’s effects, and the agency wants agreement on the study design before the marketing application is submitted.
AMT-130 is designed as a one-time gene therapy for Huntington’s disease, a progressive, inherited neurodegenerative disorder caused by mutations in the huntingtin gene. The therapy is intended to reduce production of mutant huntingtin protein, which is believed to drive neuronal dysfunction and disease progression.
The regulatory update follows a period of uncertainty for AMT-130. uniQure previously disclosed plans to seek approval based on data from a study evaluating two dose levels of the therapy in 29 participants, with outcomes compared against an external control group from a large observational study. Among 12 patients who received the higher dose, three-year data showed a 75% slowing of disease progression compared with the external control group.
Those results generated strong interest across the Huntington’s disease community, where no disease-modifying treatment is currently available. However, subsequent FDA feedback raised concerns about whether the evidence package was sufficient to support approval, including questions around the use of an external control group. The agency had strongly recommended that uniQure conduct another trial with a sham-surgery control group.
The latest update suggests a more defined regulatory path. While the FDA is still requesting confirmatory evidence, agreement that the three-year clinical data may support an application represents an important step for the program. If accepted for review, AMT-130 could become one of the most advanced gene therapy candidates for Huntington’s disease.
The case also highlights broader regulatory questions facing genetic medicines for rare and serious neurological diseases. Developers often face challenges in designing traditional randomized controlled trials when patient populations are small, disease progression is slow, procedures are invasive, and unmet need is high. The use of external controls, surrogate or functional endpoints, and confirmatory study requirements remains an important area of regulatory discussion.
For uniQure, the next key milestones will be finalizing the confirmatory study design with the FDA and submitting the approval application in the third quarter of 2026. If successful, AMT-130 could represent a major step toward a disease-modifying genetic medicine for Huntington’s disease.
