UniQure’s FDA Nightmare: How A Single Therapy Sparked A $300M Meltdown!

UniQure’s stock nosedived by a staggering 52% on November 3, 2025, after the company revealed a major regulatory hurdle in the path of its flagship gene therapy program, AMT-130. Once considered a beacon of hope for Huntington’s disease patients, AMT-130 is now facing an uncertain future after the FDA unexpectedly indicated that Phase I/II data may not be sufficient to support a Biologics License Application (BLA). This announcement came as a shock to the market, especially given that the therapy had previously received Breakthrough Therapy designation and was seen as a frontrunner in the space. Just a few months prior, UniQure had raised $345 million in a public offering to prepare for what it described as the “potential commercial launch” of AMT-130. The biotech had surged over 283% year-to-date before this setback, fueled by enthusiasm over AMT-130’s potential and strong interim data suggesting disease-modifying benefits. However, the sharp reversal in sentiment now highlights the risks of regulatory dependency, investor overoptimism, and high-stakes biotech speculation.