智通财经APP获悉，基因治疗公司uniQure(QURE.US)原本寄望其亨廷顿病候选药物AMT-130能获得美国FDA的加速批准，但这一期望近日落空。FDA明确告知这家生物制药公司，需开展一项随机、双盲、假手术对照的III期临床试验。
受此消息影响，公司股价周一收盘大跌约33%。
根据公司发布的新闻稿，uniQure此前计划基于一项与外部对照比较的I/II期研究数据申请批准。然而FDA认为，现有数据尚不足以“作为支持AMT-130上市申请所需的主要有效性证据”。
uniQure表示，将在第二季度请求与FDA召开B类会议，商讨后续推进路径。
在随后召开的电话会议中，首席执行官Matthew Kapusta透露，I/II期数据显示该药物能将疾病进展速度减缓75%，总功能能力下降速度减缓60%。但他也承认，在去年10月召开的pre-BLA会议中，“FDA就已表明，当时提交的数据……不太可能作为支持BLA申报的主要依据。”
Disclaimer: Investing carries risk. This is not financial advice. The above content should not be regarded as an offer, recommendation, or solicitation on acquiring or disposing of any financial products, any associated discussions, comments, or posts by author or other users should not be considered as such either. It is solely for general information purpose only, which does not consider your own investment objectives, financial situations or needs. TTM assumes no responsibility or warranty for the accuracy and completeness of the information, investors should do their own research and may seek professional advice before investing.