UNIQureの株は,ハンチントン病の遺伝子療法の試験を実施したFDAが、実効の十分な証拠を欠いたため,42%減少した. UniQure's stock dropped 42% after the FDA said its gene therapy trial for Huntington’s disease lacked sufficient evidence of effectiveness.

2026年3月2日,FDAがハンティントン病の遺伝子治療薬AMT-130の第I/II相試験のデータが,販売申請を支持するのに不十分であると述べた後,UniQureの株式は,市場取引前取引で42%下落した. UniQure's shares fell 42% in premarket trading on March 2, 2026, after the FDA said data from its Phase I/II trials of the gene therapy AMT-130 for Huntington’s disease were insufficient to support a marketing application. 同機関は,外部の規制と比較して,結果には効力の強い証拠がないことを認め,新たなランダム化,二重盲検,誤認手術の試験を推薦した. The agency found the results, compared to an external control, lacked strong evidence of effectiveness and recommended a new randomized, double-blind, sham surgery-controlled trial. UNIQureはFDAと連携し,2026年2月2日閲覧. ^ "第3回研究デザインの潜在的検討について"タイプB会議"を要請". UniQure plans to work with the FDA and request a Type B meeting in the second quarter of 2026 to discuss potential Phase III study designs.