FDA official says UniQure fell short on Huntington's trial, defends new study request

Core Viewpoint - UniQure's trial design for its gene therapy for Huntington's disease was deemed insufficient by a top FDA official, leading to a request for a new placebo-controlled study to support approval [1][3]. Group 1: FDA's Position - The FDA has historically rejected non-randomized and non-placebo-controlled studies for Huntington's disease treatments [2]. - The FDA official emphasized that the agency never indicated that UniQure's previous study would suffice for approval, stating that any submitted data would be critically evaluated [5]. Group 2: UniQure's Response and Market Impact - Following the FDA's announcement, UniQure's shares fell sharply from approximately $15 to $9.04 [3]. - UniQure's studies last year reported a statistically significant improvement in disease progression over three years, but the FDA found the comparison to external patient datasets unacceptable [4]. Group 3: Study Design and Requirements - The FDA suggested that a randomized placebo-controlled study could involve a sham procedure with a small scalp incision, as the gene therapy requires neurosurgery [6]. - UniQure's medical chief described the FDA's request in a controversial manner, which was later disputed by the FDA official [5].