FDA presses for sham‑surgery control in uniQure’s Huntington gene trial — regulator unmoved by external controls

The FDA has told uniQure that external‑control data from Phase I/II studies are insufficient and recommended a prospective, randomized, double‑blind, sham‑surgery‑controlled trial of AMT‑130, an AAV‑based Huntington’s disease gene therapy. uniQure and patient advocates expressed ethical concerns about long sham arms that could withhold potentially life‑prolonging treatment for years. The agency argued it needs randomized blinded data to support a marketing application. Regulatory clarity: the FDA’s stance raises practical and ethical questions for one‑time neurosurgical gene therapies, where sham operations expose participants to surgical risk and delayed access. The decision will shape uniQure’s trial design, enrollment prospects and engagement with patient communities.