Krystal's Vyjuvek becomes first topical gene therapy with FDA nod to treat rare skin disease

The first FDA-approved treatment for a rare, devastating skin disease has arrived in Krystal Biotech’s Vyjuvek.

The disease, called dystrophic epidermolysis bullosa (DEB), is hallmarked by extremely fragile skin that rips and blisters with even the slightest friction, leading to open wounds that are prone to skin infections and fibrosis. DEB sufferers are also at an increased risk of aggressive skin cancer.

Aside from being the first drug to treat this disease, Vyjuvek is also the first topical gene therapy to pass muster with regulators.

There are two types of DEB: recessive and dominant. The dominant form of the disease is usually more mild, while the recessive type is more debilitating and can lead to disfigurement, vision loss and potential fatal complications, according to the FDA.

The disease and its wounds are caused by one or more mutations in the COL7A1 gene, which is responsible for the protein that glues the inner layer of skin to the outer layer. If the gene is deficient, the skin layers can easily separate.

That’s where Vyjuvek comes in. The topical gel is a genetically modified herpes simplex type 1 virus that delivers copies of the COL7A1 gene when directly applied to DEB wounds. In a 31-patient clinical trial, 65% of Vyjuvek-treated wounds closed completely, compared with 26% of placebo-treated wounds, the FDA said in its release.

The approval covers patients 6 months of age and older. The regulatory nod “ushers in a whole new paradigm to treat genetic diseases” and is a major milestone for DEB patients and their families, Krystal Biotech CEO Krish Krishnan said in the company’s statement.

Vyjuvek is Krystal’s first approved product, and the company expects it to be available by this year’s third quarter.  

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