uniQure N.V. (QURE) Stock Plunges 57% Following FDA Setback on Huntington’s Gene Therapy
uniQure's shares collapsed by 57% in early trading after the FDA unexpectedly challenged the adequacy of clinical data for its Huntington's disease treatment AMT-130. The biotech firm had planned to submit its Biologics License Application using Phase I/II trial results compared against external controls—an approach previously discussed with regulators.
The regulatory reversal marks a significant setback for one of gene therapy's most promising pipelines. Huntington's disease, a rare neurodegenerative disorder, currently has no cure. AMT-130 represented a potential breakthrough treatment modality.
Market reaction was immediate and severe, with QURE shedding nearly two-thirds of its market capitalization within hours. The development underscores the binary risk profile of clinical-stage biotech investments, where regulatory decisions can erase billions in valuation overnight.
