The FDA insisted this week that uniQure conduct a sham‑controlled randomized trial for AMT‑130, the company’s gene therapy for Huntington’s disease, defending reviewers’ request despite company objections. Agency officials clarified the design would involve anesthesia and minor scalp incisions rather than the burr‑hole procedures uniQure warned would be ethically and logistically fraught. UniQure had cited pivotal Phase 1/2 results showing a 75% slowing of disease progression versus external controls; FDA reviewers countered that the earlier data are not adequate to support a biologics license application without a randomized, sham‑controlled study. The exchange has escalated into public criticism by agency and industry sources, raising questions about review consistency for intracranial gene therapies. The dispute underscores a broader regulatory tension over acceptable evidence standards for neurosurgical gene therapies and could set precedent for future CNS gene‑therapy submissions.