The FDA defended its decision to require a sham-controlled trial for uniQure’s AMT-130 gene therapy for Huntington’s disease, telling reporters that the agency asked the company to randomize patients to treatment versus an anesthetized control with superficial scalp incisions. UniQure has publicly criticized the requirement as ethically problematic and medically risky; the company contends prior Phase 1/2 data and external control comparisons warrant a biologics license application. Agency officials say the sham-control is necessary to generate robust randomized data because surgical delivery and placebo effects could confound the therapy’s clinical signal. UniQure maintains its pivotal data showed a 75% slowing of progression versus external controls, and it has framed the FDA’s request as a “key shift” from earlier guidance. The dispute has escalated into a public relations clash that underscores tensions between regulators seeking randomized evidence and developers of invasive gene therapies that present trial-design challenges. The outcome will set precedent for how surgical-delivered neurotherapies are evaluated going forward.