U.S. FDA grants Vivet/Pfizer VTX-801 fast track qualification

Pfizer and Vivet Therapeutics, a clinical-stage biotechnology company, recently announced that the U.

Fast Track Qualification (FTD) aims to accelerate drug development and rapid review for serious diseases to address serious unmet medical needs in key areas

VTX-801 is a new type of research gene therapy, which will be evaluated in the Phase 1/2 GATEWAY trial (NCT04537377) to determine the safety, tolerability and pharmacological activity of a single intravenous infusion in the treatment of adults with Wilson disease

Wilson disease (WD) is a rare genetic disease that is caused by mutations in the gene encoding the ATP7B protein, which reduces the ability of the liver and other tissues to regulate copper levels, leading to severe liver damage, neurological symptoms, and potential death

VTX-801 is a new type of research gene therapy vector based on recombinant adeno-associated virus (rAAV).

The GATEWAY trial (NCT04537377) is a multicenter, non-randomized, open-label Phase 1/2 clinical trial designed to evaluate the safety of a single intravenous infusion of VTX-801 in adults with Wilson disease before and after background WD treatment is withdrawn , Tolerability and pharmacological activity

The trial will be carried out in six leading clinical centers in the United States and Europe, and up to 16 adult patients with Wilson disease are expected to be enrolled

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Original source: VTX-801 Receives US FDA Fast Track Designation for the Treatment of Wilson Disease