uniQure Advances AMT-130 Toward FDA Filing in Huntington’s Disease
uniQure has outlined plans to move forward with a Biologics License Application for AMT-130, its investigational gene therapy for Huntington’s disease, under the FDA’s accelerated approval pathway.
According to reports, the FDA has agreed that three-year Phase 1/2 data from AMT-130 can serve as the primary basis for the application. uniQure plans to submit the BLA in Q3 2026.
Key points
- Three-year P1/2 data as key basis
- BLA planned via accelerated approval
- Filing planned for Q3 2026
- Confirmatory study design under FDA discussion
Huntington’s disease is an inherited neurodegenerative disorder affecting movement, cognition, and psychiatric function. There are currently no approved therapies that slow disease progression, leaving a significant unmet need for disease-modifying approaches.
AMT-130 is designed to reduce expression of the huntingtin protein. Previously reported three-year data showed a reduction in disease progression among patients receiving the high dose.
This update is also notable from a regulatory perspective. For CNS gene therapy, the use of early clinical data, external controls, confirmatory studies, and accelerated approval remains a key development question.
Gene therapy for neurodegenerative disease requires specialised capability across delivery strategy, long-term follow-up, safety evaluation, manufacturing, and regulatory execution. AMT-130’s progress suggests that CNS gene therapy is moving into a new clinical and regulatory stage.
Congratulations to uniQure and everyone involved in the Huntington’s disease field.