FDA Approves First Drug for Rare Skin Disorder, and It’s a Redosable Gene Therapy

MedCity News May 21, 2023
Frank Vinluan

Krystal Biotech’s Vyjuvek is approved to treat recessive and dominant forms of dystrophic epidermolysis bullosa (DEB), a rare genetic skin disorder. The redosable gene therapy, applied to the skin, delivers a gene that gets cells to produce collagen that DEB patients lack.

A rare skin disorder that leads to fragile skin susceptible to frequent wounds and blisters now has its first FDA-approved therapy, a topically applied gel that facilitates healing. The Krystal Biotech drug is a gene therapy, and in a first for this treatment modality, dosing can be repeated as needed.

The regulatory decision announced Friday covers the treatment of dystrophic epidermolysis bullosa (DEB) in patients age 6 months or older. The approval spans both the recessive and dominant...

Today's Sponsors

Today's Sponsor