uniQure announced on January 9, 2026, that it has scheduled a Type A meeting with the U.S. Food and Drug Administration to discuss the Biologics License Application data package supporting accelerated approval of AMT-130, its investigational gene therapy for Huntington’s disease. The company plans to present detailed clinical and manufacturing data, including safety profiles from its Phase 1/2 trial, and is targeting a resolution on the accelerated pathway by mid-year. CEO Matt Kapusta emphasized the unmet medical need, citing input from over 120 patient advocacy groups and clinicians who have registered interest in early access to AMT-130.

In its 36-month analysis, uniQure reported a 75% slowing of disease progression as measured by the composite Unified Huntington’s Disease Rating Scale (cUHDRS) in treated patients compared with historical controls. However, the FDA has reversed its prior receptivity to using external control arms for approval, raising questions about the adequacy of the current dataset. The agency’s shift will likely require uniQure to plan a randomized, controlled confirmatory study—potentially extending the development timeline by 18 to 24 months.

As of September 30, 2025, uniQure held $694 million in cash, cash equivalents and marketable securities, sufficient to fund operations through at least the fourth quarter of 2027 without additional capital raises. Management projects that, even with the anticipated need for a Phase 3 trial, the existing balance sheet provides runway to complete both the confirmatory study and ongoing manufacturing scale-up for AMT-130. Investors will watch closely for updated guidance following receipt of the FDA meeting minutes and any formal feedback on trial design.