The FDA told uniQure that a sham‑controlled randomized trial is required for its surgically delivered gene therapy AMT‑130 for Huntington’s disease, with an unnamed senior official clarifying that the control arm would include anesthesia and minor scalp nicks rather than a partial burr hole. The agency reiterated its reviewers’ request following uniQure’s public objections that a sham would pose undue risk to patients. UniQure had argued pivotal Phase 1/2 data—showing a 75% slowing of progression versus external controls—should suffice for a BLA submission; the FDA has said the Phase 1/2 study design is not adequate as primary evidence. The exchange highlights the agency’s insistence on randomized, internally controlled evidence for high‑risk neurosurgical biologics and signals continued friction over acceptable trial designs in rare neurodegenerative diseases.