The FDA has recommended a randomized, double-blind, sham surgery–controlled trial for uniQure’s AMT‑130 gene therapy program in Huntington’s disease, the company said after a recent regulator meeting. UniQure disclosed that agency staff told them the existing single-arm data compared to external controls are insufficient to support a marketing application. Company executives plan to request a Type B meeting to define trial design and timelines. The recommendation could add two to three years to development and raises ethical and operational challenges around sham neurosurgical controls, according to company disclosures and analyst commentary. UniQure’s stock moved sharply on the news, reflecting investor concern about both schedule risk and the potential need for a large, complex pivotal study. Sham surgery controls involve a simulated surgical procedure used to preserve blinding in trials where operative risk and ethical trade-offs must be carefully managed.