周二,基因疗法公司uniQure N.V.股价在盘中大涨93.7%,引发市场高度关注。这一突出表现主要源于公司与美国食品药品监督管理局(FDA)就其重大基因疗法产品的审批途径达成协议,无疑是利好消息。
根据公司发布的消息,在与FDA的协商会议中,FDA同意公司可以使用正在进行的I/II期临床试验数据与外部自然史对照组进行比较,作为加速批准途径下提交生物制品许可申请(BLA)的主要依据,避免需要额外的预提交研究。这将有助于加快该疗法审评审批步伐。
此外,FDA还同意该疗法在临床试验中采用cUHDRS作为中间临床终点评估疗效,并将脑脊液中神经纤维轻链(NfL)水平的降低作为支持疗效的辅助证据。这一系列协议的达成大大增加了该产品最终获批上市的预期,同时也反映出FDA对该疗法的乐观预期,为投资者带来了较大的利好刺激。
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