The FDA told uniQure it requires a prospective, randomized, double‑blind, sham surgery–controlled trial to support marketing of AMT‑130, the company’s one‑time AAV‑based Huntington’s disease therapy. Agency feedback followed a Type A meeting in which regulators said Phase I/II data compared to external controls were insufficient for primary evidence of effectiveness. AMT‑130 delivered large effects in early studies—investigators reported slowed disease progression—but FDA’s stance raises logistical and ethical concerns about withholding a potentially life‑extending surgical therapy. Clinicians and patient advocates warned that multi‑year delays from a sham‑controlled study could render some participants ineligible for later treatment. The FDA’s demand sets a high bar for surgical gene therapies and will shape uniQure’s trial design and timelines.