The FDA told uniQure regulators are not persuaded that data from the completed single‑arm AMT‑130 trial are sufficient for approval and strongly recommended a prospective, randomized, double‑blind, sham‑surgery‑controlled study. uniQure held a meeting with FDA in late January and said regulators requested the randomized design, blocking the company’s immediate path to a marketing application. The agency’s stance has prompted fresh investor concern and further delays for a program once viewed as close to approval. uniQure has said it intends to engage with regulators on next steps while evaluating trial design options. STAT reported on the outcome of the FDA meeting and the company issued formal statements reflecting the agency’s preference. For sponsors of surgical gene therapies, the FDA’s demand underscores heightened evidentiary expectations for single‑arm datasets—especially where sham controls are feasible. The recommendation carries implications for timeline, cost and trial feasibility for other in‑operating‑room gene therapy programs.