UniQure的库存下降了42%, 因为FDA说, 它对亨廷顿病的基因治疗试验 缺乏足够的有效证据。 UniQure's stock dropped 42% after the FDA said its gene therapy trial for Huntington’s disease lacked sufficient evidence of effectiveness.

UniQure公司在2026年3月2日的市场前交易中的份额下降了42%,因为林业发展局说,对亨廷顿病的基因治疗AMT-130的第一阶段/第二阶段试验数据不足以支持销售申请。 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. 该机构发现,与外部控制相比,结果缺乏有力的有效性证据,建议进行新的随机、双盲、假冒的外科手术控制试验。 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计划与FDA合作,并在2026年第二季度要求举行B型会议,讨论潜在的III期研究设计. 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.