The FDA has told UniQure it expects a randomized, double-blind, sham surgery–controlled trial before it will consider a marketing application for AMT‑130, the company’s gene therapy for Huntington’s disease. UniQure disclosed the agency’s recommendation after a meeting with regulators, which raises the prospect of a two‑ to three‑year setback and renewed ethical and operational hurdles for the program. Investors reacted quickly: the company’s shares plunged after the announcement, reflecting heightened investor concern about regulatory stringency for rare-disease gene therapies. UniQure plans to request a Type B meeting with the FDA to clarify trial design and next steps. The agency’s stance echoes recent FDA demands for gold‑standard controls in other gene‑therapy programs and signals tighter scrutiny of externally controlled data.