The FDA has concluded that UniQure's experimental Huntington's disease treatment AMT-130 does not benefit patients based on Phase I/II clinical data to date.[1][2] The agency is not convinced of the drug's therapeutic benefit and has rejected the request for a marketing application.[1][3] The FDA recommended a prospective, randomized, double-blind, sham-controlled study as a phase III study.[2][3][4] This decision was announced after the Type A meeting on January 30, 2026.[2][3][4] UniQure plans to request a Type B meeting in the second quarter of 2026 to discuss the design of Phase III.[2][3][4] The firm emphasizes the durability of its data and continues to dialogue with the regulator over unmet needs in Huntington's disease.[2][4] UniQure shares fell more than 40% following the announcement.[3]