Rare Daily Staff

The U.S. Food and Drug Administration granted Fast Track designation to Vivet Therapeutics’ and Pfizer’s VTX-801 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.

Wilson Disease 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, causing severe hepatic damage, neurologic symptoms and potentially death.

VTX-801 is an experimental rAAV-based gene therapy vector designed to deliver a miniaturized ATP7B transgene encoding, a functional protein that has been shown to restore copper homeostasis, reverse liver pathology and reduce copper accumulation in the brain of a mouse model of Wilson Disease. VTX-801’s rAAV serotype was selected based on its demonstrated tropism for transducing human liver cells. It is 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. Pfizer is collaborating with Vivet on the clinical supply of VTX-801 for the phase 1/2 clinical trial.

“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,” said Jean-Philippe Combal, CEO and co-founder of Vivet.

The FDA’s Fast Track program is designed to facilitate the development and expedite the review of novel potential therapies that are designed to treat serious conditions and fill an unmet medical need, by providing a therapy where none exists or providing a therapy which may be potentially better and shows some advantage over available therapy. Fast Track designation includes opportunities for more frequent meetings with the FDA to discuss trial design, development plans, data needed to support drug approval, submission of a New Drug Application on a rolling basis, and eligibility for accelerated approval and priority review, if relevant criteria are met.

“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,” said Seng Cheng, senior vice president and chief scientific officer of Pfizer’s Rare Disease Research unit.

VTX-801 has been granted Orphan Drug designation by the FDA and the European Commission.

Photo: Jean-Philippe Combal, CEO and co-founder of Vivet

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