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uniQure：FDA 不再认为 I/II 期数据足以支持 AMT-130 的 BLA 申请
FDA 推翻了之前的指导意见，现在表示 I/II 期数据与外部对照相比可能不足以作为 BLA 申报的主要证据。AMT-130 计划中的 BLA 申报时间现已不明。uniQure 将寻求最终会议纪要，并与 FDA 紧急跟进讨论，以确定后续路径。
关键事实
来源摘录
EX-99.1 2 qure-20251103xex99d1.htm EX-99.1 Exhibit 99.1 uniQure Provides Regulatory Update on AMT-130 for Huntington ’ s Disease Lexington, MA and Amsterdam, the Netherlands, November 3, 2025 — uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that it received feedback from the U.S. Food and Drug Administration (FDA) during a recent pre-Biologics License Application (BLA) meeting regarding AMT-130, an investigational gene therapy for Huntington ’ s disease (HD). Though final meeting minutes have not yet been received, based on the discussions at the meeting, uniQure believes that the FDA currently no longer agrees that data from the Phase I/II studies of AMT-130 in comparison to an ex