The FDA has concluded that data from the Phase I/II study of UniQure's AMT-130 gene therapy are insufficient to support an application for approval for the treatment of Huntington's disease.[2][3][4] The study used an external control group from the natural history of the disease instead of a randomized control.[2][5][6] Three-year data showed a 75% slowing of disease progression in 12 of 17 high-dose patients compared to external controls, as measured by the composite Unified Huntington's Disease Rating Scale.[2][5] The FDA recommended a prospective, randomized, double-blind, sham surgery-controlled trial.[3][4][5] UniQure plans another meeting with the FDA in Q2 2026 to discuss the Phase III study design.[4][5] The dispute marks another hurdle for AMT-130, although experts have previously called the data significant.[2][3] A total of 45 patients received AMT-130.[1]