FDA Approves First-Ever Topical Gene Therapy for Rare Skin Condition

The Food and Drug Administration on Friday approved the first-ever topical gene therapy to treat wounds in patients with dystrophic epidermolysis bullosa (DEB), a rare, inherited skin condition. The therapy, beremagene geperpavec-svdt (Vyjuvek, Krystal Biotech), is the first-ever redosable gene therapy and the only FDA-approved treatment for DEB.

Patients with DEB have a mutation on the collagen type VII alpha 1 chain (COL7A1) gene that affects the production of functional COL7 protein, which forms the anchoring fibrils necessary to bind the different layers of the skin. This mutation leads to extremely fragile skin that blisters and tears with minor friction or trauma. While symptoms vary between patients, those with severe disease can develop blistering that leads to vision loss, disfigurement and other serious medical complications.

Beremagene geperpavec-svdt is a genetically modified herpes-simplex virus that works by delivering functional copies of the COL7A1 gene and promoting wound healing. The therapy has also been modified to eliminate its ability to replicate in normal cells. To administer the treatment, health care professionals mix beremagene geperpavec-svdt into an excipient topical gel and apply it to patients’ wounds each week.

Additional information about clinical trials evaluating the efficacy of beremagene geperpavec-svdt is available in the FDA approval notice.