荷兰基因疗法开发商uniQure周一表示，美国FDA已受理其亨廷顿舞蹈症治疗药物的申请，并将于2027年5月1日前对该药物做出决定。该公司在美上市的股票在盘前交易中上涨2.5%。以下是一些细节：这一进展发生在uniQure宣布AMT-130在一项为期四年的试验中未能延缓疾病进展、导致其股价大幅下跌数月之后。FDA周一决定审查AMT-130，结束了与uniQure长期的僵持，期间该机构一位高级官员曾将该疗法描述为“失败的产品”。
Dutch gene therapy developer uniQure said on Monday the US FDA had accepted its application for its Huntington's disease treatment and would decide on the drug by May 1, 2027.
监管机构随后改变立场，同意审查该申请。uniQure表示，FDA给予该疗法更快的审查。该机构还在考虑召开外部专家会议讨论该申请。
US-listed shares of the company were up 2.5% in premarket trading.
uniQure的申请基于早期至中期研究结果，将接受治疗的患者与一个外部亨廷顿舞蹈症数据库中的患者群体进行比较。该公司表示，迄今已在其研究中治疗了51名患者。
Here are some details:
FDA正根据其加速批准途径审查该申请，该途径可允许针对严重疾病的治疗基于早期证据更快获批，同时要求公司在未来研究中确证获益。
The development comes months after uniQure said that AMT-130 failed to slow disease progression in a four-year trial, sending its shares sharply lower The FDA's Monday decision to review AMT-130 ends a lengthy standoff with uniQure, during which a senior agency official described the treatment as a "failed product"
uniQure计划于年底前开始美国患者筛选，开展一项200名患者的后续试验，其中三分之二接受基因疗法，其余接受标准护理。
The regulator later reversed course and agreed to review the application The FDA granted the therapy a faster review, uniQure said. The agency is also considering holding a meeting of outside experts to discuss the application UniQure's application is based on early- to mid-stage study results comparing treated patients with a group of patients from an external Huntington's disease database. The company said it has treated 51 patients across its studies so far The FDA is reviewing the application under its accelerated approval pathway, which can allow speedier approval for treatments targeting serious diseases based on early evidence, while requiring the company to confirm the benefit in future studies UniQure plans to begin US patient screening by year-end for a 200-patient follow-up trial, with two-thirds receiving the gene therapy and the rest standard care