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

Fierce Pharma May 19, 2023
By Zoey Becker

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, both 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...

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