The FDA has publicly defended reviewers’ request for a sham‑controlled randomized study for uniQure’s AMT‑130 Huntington’s gene therapy, rejecting the company’s characterisation of the procedure as ethically untenable. A senior, anonymous FDA official clarified that the agency asked for scalp incisions and anesthesia rather than full burr holes, and affirmed reviewers’ view that a randomized design is needed. UniQure has pushed back, citing prior pivotal Phase 1/2 results and accusing the agency of changing earlier guidance. The exchange highlights tension over trial design standards for surgically delivered gene therapies and underscores regulators’ insistence on randomized evidence for high‑risk neurologic interventions.