Posted by Michael Wonder on 12 Aug 2021
VTX-801 receives U.S. FDA fast track designation for the treatment of Wilson disease
12 August 2021 - Vivet Therapeutics and Pfizer today announced the U.S. FDA has granted fast track designation to VTX-801, Vivet’s clinical stage gene therapy for the treatment of Wilson Disease – a rare, genetic disorder that reduces the ability of the liver and other tissues to regulate copper levels, causing severe hepatic damage, neurological symptoms, and potentially death.
VTX-801 is a novel investigational gene therapy to be evaluated in a Phase 1/2 clinical trial to determine the safety, tolerability, and pharmacological activity of a single intravenous infusion in adult patients with Wilson disease.
