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Pfizer (PFE) Gets Fast Track Tag for Wilson Disease Candidate

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Pfizer Inc. (PFE - Free Report) , along with privately held gene-therapy company Vivet Therapeutics, announced that the FDA has granted a Fast Track designation to the latter’s investigational gene therapy candidate, VTX-801, which is being developed for the treatment of Wilson disease, a rare, genetic and potentially life-threatening liver disorder.

The FDA provides a Fast Track designation to help the development and faster review of drugs that treat serious and unmet medical conditions. The designation will also make the candidate eligible for priority review and accelerated approval upon successful development.

Pfizer collaborated with Vivet in September 2020 for clinical supply of VTX-801 for the phase I/II study. The study is expected to begin shortly and will evaluate the safety, tolerability, and pharmacological activity of a single intravenous infusion of VTX-801 in adult patients with Wilson disease. Pfizer believes that if successfully developed, VTX-801 has the potential to be a transformational therapy and serve an area of significant medical need for patients living with Wilson disease.

Shares of Pfizer have rallied 28.4% so far this year compared with the industry’s growth of 14.9%.

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We remind investors that, in November 2020, the FDA cleared Vivet’s investigational new drug (“IND”) application for the phase I/II GATEWAY study evaluating VTX-801 for the potential treatment of Wilson disease.

VTX-801 has already received an Orphan Drug designation from both the FDA and the European Commission for the treatment of Wilson disease.

Another company developing a treatment for Wilson disease is Ultragenyx Pharmaceutical (RARE - Free Report) .

In January 2021, the FDA cleared Ultragenyx’s IND application of investigational AAV type 9 gene therapy, UX701, being evaluated for the treatment of Wilson Disease. The company plans to begin a phase I/II/III study on UX701 shortly with the first patient expected to be dosed in the second half of 2021.

The FDA has also granted an Orphan Drug designation to UX701 for the given indication.

Zacks Rank & Stocks to Consider

Pfizer currently carries a Zacks Rank #3 (Hold). Top-ranked stocks in the biotech sector include Catalent, Inc. (CTLT - Free Report) and Vertex Pharmaceuticals Incorporated (VRTX - Free Report) , both carrying a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

Catalent’s earnings estimates have been revised 0.3% upward for 2021 and 6.4% for 2022 over the past 60 days. The stock has surged 15.6% year to date.

Vertex’s earnings estimates have been revised 9.4% upward for 2021 and 5.5% for 2022 over the past 60 days.

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