The FDA has told uniQure that its existing evidence for the AMT‑130 Huntington’s gene therapy is insufficient and has recommended a randomized, sham surgery–controlled study before considering approval. uniQure disclosed the agency’s stance in its latest filings and the company’s shares plunged on the news, reflecting investor concern about a multi‑year setback. The regulator cited weaknesses in external controls and surrogate endpoints used in the submitted program, arguing they do not adequately demonstrate therapeutic benefit. A sham‑surgery control involves a placebo surgical procedure to maintain blinding in trials where a surgical intervention might otherwise reveal treatment allocation. UniQure plans to meet with the FDA to discuss next steps; the agency’s demand echoes recent, more conservative signals from regulators on single‑arm data for rare disease gene therapies. Stakeholders are watching for how regulators will balance randomized evidence requirements with ethical and feasibility concerns in small patient populations.