uniQure Announces FDA Acceptance and Priority Review of BLA for Huntington's Disease Gene Therapy

The FDA accepted uniQure’s BLA for investigational Huntington’s disease gene therapy ifezuntirgene inilparvovec under the Accelerated Approval pathway and granted it Priority Review. The FDA set a May 1, 2027 PDUFA target date and tentatively plans to discuss the application at an Advisory Committee meeting. The filing follows Phase I/II studies in which 51 patients received the therapy.