FDA Grants Fast-Track Designation to Pfizer and Vivet Therapeutics for Treatment of Wilson Disease

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FDA has awarded Fast Track designation to Vivet Therapeutics’ novel gene therapy, VTX-801.

FDA has granted fast-track designation to VTX-801, a gene therapy developed by clinical-stage biotechnology company Vivet Therapeutics in conjunction with Pfizer, for the treatment of Wilson Disease.

VTX-801 is a novel investigational gene therapy that will now be evaluated in a Phase I/II clinical trial to determine the safety, viability, and pharmacological activity of a single intravenous infusion in adults with the disease. The Fast Track designation, given to therapies designed to treat serious conditions with unmet medical need, will facilitate its development and expedite the review process.

Wilson Disease is a rare genetic disorder that reduces the ability of the liver to regulate copper levels. This results in severe hepatic damage, neurological symptoms, and potential death.

“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, in a company press release from August 12, 2021.


“With the FDA’s authorization of the [Investigational New Drug] 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, in the press release.

Source: Pfizer