Core Viewpoint - UniQure is required by the FDA to conduct another study to demonstrate the efficacy of its gene therapy for Huntington's disease, amidst ongoing disputes regarding the ethical implications of such a trial [1][2][3] Group 1: FDA's Position - A senior FDA official stated that UniQure must perform a placebo-controlled trial for its gene therapy, which is administered directly into the brain [2] - The FDA official disputed UniQure's claim that conducting such a study would be unethical due to the need for general anesthesia [2] - The FDA is facing scrutiny for recent rejections of drug approval applications, with companies alleging that the agency has reversed previous guidance [3] Group 2: UniQure's Response - UniQure has indicated that the requested type of study is not ethical, suggesting that it would pose significant risks to participants [2] - The FDA official implied that UniQure may be aware of the shortcomings of its previous trials and is not conducting the necessary clinical studies [3] - The ongoing conflict highlights a broader issue within the industry regarding regulatory expectations and the approval process for new therapies [3]