The Food and Drug Administration (FDA) has granted Fast Track designation to VTX-801 for the treatment of Wilson disease.

Wilson disease is a rare inherited disorder caused by mutations in the ATP7B gene, which leads to a lack of ATP7B production, a protein responsible for transporting copper. This results in the accumulation of copper in various body tissues, particularly the liver and central nervous system. 

VTX-801 is a novel replication-deficient recombinant adeno-associated viral vector (rAAV) gene therapy that contains a single-stranded DNA genome with a shortened version of the ATP7B gene. By delivering the ATP7B transgene, the Company believes VTX-801 will restore copper homeostasis, reverse liver pathology and reduce copper accumulation.

The Company is evaluating the safety, tolerability, and pharmacological activity of a single intravenous infusion of VTX-801 in adult patients with Wilson disease, prior to and following background therapy withdrawal, in the phase 1/2 GATEWAY trial ( Identifier: NCT04537377).

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Dr Michael Schilsky, Principal Investigator at Yale University School of Medicine (Connecticut, United States), said, “We are proud to participate in this important clinical trial. If VTX-801 is successfully developed, it has the potential to be a truly innovative medicine with the ability to restore copper metabolism after a single injection, addressing significant unmet medical needs for patients with Wilson disease.”

The FDA’s Fast Track designation allows for expedited review of therapies that are meant to treat serious or life-threatening conditions. Generally, the designation is granted to therapies that are expected to have an impact on factors such as survival and daily functioning.


VTX-801 receives U.S. FDA Fast Track designation for the treatment of Wilson disease. News release. Vivet Therapeutics. Accessed August 12, 2021. 

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