Vivet’s VTX-801 gene therapy gets FDA fast track status in Wilson Disease

Vivet Therapeutics, a biotech company backed by Pfizer, has been given fast track designation by the US Food and Drug Administration for its gene therapy candidate VTX-801 for the treatment of Wilson Disease.

Wilson Disease is a rare, genetic disorder that lessens the ability of the liver and other tissues in controlling copper levels, thereby leading to severe hepatic damage, neurological symptoms, and possibly death.

VTX-801 is an rAAV-based gene therapy vector candidate designed for infusing a miniaturized ATP7B transgene encoding, which is a functional protein known for restoring copper homeostasis, reverse liver pathology, and decrease copper accumulation in the brain of a mouse model of Wilson Disease.

The Fast Track program of the FDA is designed to encourage the development, and speed up the review of novel potential therapies that target treat serious conditions and address unmet medical need.

VTX-801 is planned to be assessed in a phase 1/2 clinical trial for determining its safety, tolerability, and pharmacological activity of a single intravenous infusion in adults with Wilson Disease.

Vivet’s VTX-801 gene therapy gets FDA fast track status in Wilson Disease. Image courtesy of Gerd Altmann from Pixabay .

Jean-Philippe Combal — CEO and co-founder of Vivet Therapeutics said: “With the FDA’s authorization of the IND application for VTX-801 – combined with Pfizer’s state-of-the-art gene therapy manufacturing capabilities – we are well-positioned to rapidly advance development of this potential therapy.”

Pfizer is collaborating with Vivet Therapeutics on the clinical supply of the VTX-801 gene therapy candidate for the early stage clinical trial.

Seng Cheng — Senior Vice President and Chief Scientific Officer of Pfizer Rare Disease Research Unit said: “The FDA’s decision to grant VTX-801 Fast Track designation underscores the urgent need for new therapeutic options to address this devastating disease, which, if left untreated, can be fatal.

“We are pleased to collaborate with Vivet on this important development program, which we believe, if successful, could make a meaningful difference in the lives of patients living with Wilson Disease.”