UniQure的库存量下跌了59%,因为FDA以证据不足为由拒绝提供亨廷顿病基因治疗数据。 UniQure's stock plunged 59% after the FDA rejected its gene therapy data for Huntington’s disease, citing insufficient evidence.

UniQure公司的股票在2025年11月3日市场前交易中下降了59%,因为林业发展局拒绝了该公司治疗亨廷顿病的基因疗法AMT-130临床数据,指出证据已经不足以申请生物许可。 UniQure's stock dropped 59% in premarket trading on November 3, 2025, after the FDA rejected the company’s clinical data for its gene therapy AMT-130 treating Huntington’s disease, stating the evidence is no longer sufficient for a biologics license application. 这一疗法在早期试验中有望将疾病增速减慢75%,但以前曾获得突破疗法称号。 The therapy, which showed promise in slowing disease progression by 75% in earlier trials, had previously received breakthrough-therapy designation. 林业发展局的决定使批准时限不确定,促使UniQure公司寻求与监管者紧急讨论,以确定下一步措施。 The FDA’s decision has left the approval timeline uncertain, prompting UniQure to seek urgent discussions with regulators to determine next steps. 结果突出显示了在推进复杂神经退化性障碍基因疗法方面持续存在的挑战。 The outcome highlights ongoing challenges in advancing gene therapies for complex neurodegenerative disorders.