Krystal Biotech’s Redosable Gene Therapy Gel Approved for Rare Skin Condition

The FDA has approved Krystal Biotech’s Vyjuvek, a redosable gel formulation gene therapy for the treatment of dystrophic epidermolysis bullosa, a rare genetic condition caused by a missing gene resulting in extremely fragile skin that blisters and tears with minor friction or trauma.

The drug delivers functional copies of the missing COL7A1 gene to provide wound healing and sustained protein expression which anchors the fibrils needed to bind the inner layer of the skin to the outer layer as in normal skin.

The FDA approval of Vyjuvek is based on two clinical studies that showed that repeat topical applications of the gel were associated with durable wound closure, full-length cutaneous COL7 expression, and anchoring fibril assembly with minimal reported adverse events. Vyjuvek was well tolerated with no drug-related serious adverse events or discontinuations due to treatment-related events, the company said.

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