The FDA has pushed back on uniQure’s Huntington’s disease program, recommending a randomized, double‑blind, sham‑surgery–controlled trial before it will consider approval. Company disclosures and regulator comments indicate agency reviewers found the existing single‑arm, externally controlled data insufficient to demonstrate therapeutic benefit. The recommendation could add two to three years and major costs to AMT‑130’s path to market. Investors reacted swiftly, driving uniQure shares down as the company flagged the need to discuss trial design, ethics and feasibility with the agency. The agency’s insistence on a sham control reflects a broader FDA trend toward gold‑standard evidence for invasive gene therapies, particularly where external controls or surrogate endpoints were previously accepted. Clinical, ethical and enrollment logistics for sham surgery controls remain central questions for sponsors and IRBs.