The FDA has told uniQure that its current data package for AMT‑130, a gene therapy for Huntington’s disease, is not sufficient and recommended a prospective, randomized, double‑blind, sham surgery‑controlled study. Company disclosures and analyst notes indicate regulators "strongly recommended" the sham‑controlled design, a requirement that could add two to three years to the program and raise complex ethical and operational questions. UniQure reported discussions with the agency and plans to request a Type B meeting to define an acceptable trial design and safety monitoring. The FDA’s stance reflects a tightening of evidentiary expectations for one‑time gene therapies in neurodegenerative indications, where single‑arm or external‑control data have frequently been proposed. Investors reacted quickly: uniQure shares fell sharply on the news, with analysts calling the recommendation a “worst‑case scenario” for near‑term approval. The company must now weigh trial design, sham surgery ethics, patient selection and timelines before deciding a regulatory path forward.