uniQure said a recent pre‑BLA meeting with the U.S. Food and Drug Administration signaled the agency no longer views Phase I/II data using an external control as sufficient to support a biologics license application for AMT‑130, its Huntington’s disease gene therapy. The company had previously reported three‑year data showing marked slowing of disease measures in high‑dose patients. uniQure called the change a key shift from prior guidance; investors responded with a steep selloff. Analysts likened the episode to other recent FDA skepticism about single‑arm or external‑control submissions, and the company said it will urgently engage regulators to define a path forward.