FDA反转！公司股价暴跌…

Core Viewpoint - The FDA has unexpectedly withdrawn its recognition of the clinical trial data for the Huntington's disease gene therapy AMT-130, leading to uncertainty regarding the planned submission of a Biologics License Application (BLA) by uniQure in Q1 2026 [1][2]. Group 1: Clinical Trial Results - The initial results from the Phase 1/2 clinical trial showed a 75% reduction in disease progression among 12 high-dose patients after three years compared to an external control group based on natural history studies [1]. - This data was previously hailed as a "game-changing" breakthrough by experts in the field, significantly boosting investor confidence and leading to a 300% increase in uniQure's stock price [1]. Group 2: FDA's Regulatory Stance - The FDA's recent feedback indicates a significant deviation from prior guidance, stating that the Phase 1/2 data alone is insufficient to support the BLA application, contradicting earlier agreements [2]. - The tightening regulatory stance of the FDA on cell and gene therapies is evident, as seen in recent rejections of other therapies due to insufficient evidence of efficacy [4]. Group 3: Company Response and Future Actions - uniQure is awaiting the final minutes from the FDA's upcoming pre-meeting and plans to urgently communicate with the agency to explore possible pathways for expedited approval of AMT-130 [4]. - The company remains committed to collaborating with the FDA to bring this potential therapy to patients, despite the setback [5].