UniQure's AMT-130 Huntington's Gene Therapy Gains FDA Support for Accelerated Approval Pathway

UniQure has successfully aligned with the FDA regarding the accelerated approval pathway for its gene therapy AMT-130, designed to treat Huntington's disease. During a recent Type B meeting, the FDA agreed to allow the use of data from the ongoing Phase I/II studies, supported by natural history external controls, as the primary basis for the Biologics License Application, thereby avoiding the necessity for an additional trials[1][2]. The FDA also authorized the utilization of the Unified Huntington’s Disease Rating Scale as an intermediate clinical endpoint and endorsed the use of neurofilament light chain levels as supportive evidence to demonstrate AMT-130’s efficacy[1]. This progression is seen as a promising advancement for UniQure, marking a significant step towards potentially obtaining accelerated approval for their therapeutic approach to Huntington's disease[2].