The FDA has strongly recommended that uniQure conduct a prospective, randomized, double‑blind, sham surgery‑controlled study before it will consider approval of AMT‑130 for Huntington’s disease. Company disclosures show the agency was unconvinced by data from single‑arm studies with external controls and recommended the gold‑standard trial design. Markets reacted immediately: uniQure shares plunged and analysts warned the requirement could delay the program by two to three years and raise ethical and logistical questions about sham surgery controls. UniQure said it plans to request a Type B meeting with regulators to clarify next steps. The episode highlights regulators’ renewed insistence on randomized controls in certain gene‑therapy filings and the operational, ethical and valuation consequences for sponsors and investors.