UniQure Shares Drop After FDA Shifts Stance On Huntington's Gene Therapy Data

Core Viewpoint - The FDA's feedback on uniQure's investigational gene therapy AMT-130 for Huntington's disease has led to a significant decline in the company's stock, indicating a shift in regulatory expectations and uncertainty regarding the BLA submission timeline [1][3][4]. Group 1: FDA Feedback and Company Response - The FDA expressed disagreement with uniQure's interpretation of data from Phase 1/2 studies of AMT-130, suggesting it may not be sufficient for a BLA submission [2][4]. - This feedback represents a notable change from previous communications with the FDA, which had indicated that the data could support a BLA submission under the Accelerated Approval pathway [3][4]. - The company plans to urgently engage with the FDA to explore options for the timely accelerated approval of AMT-130 [3][5]. Group 2: Market Reaction - Following the FDA's feedback, uniQure's stock price fell by 53.98%, trading at $31.15 at the time of publication [6]. Group 3: Future Plans - In addition to working with the FDA, uniQure intends to engage with other regulatory agencies, including those in the European Union and the United Kingdom, to advance discussions regarding AMT-130 [5].