FDA Expands Krystal Biotech Gene Therapy Label for DEB from Birth

The FDA has approved a label update for Krystal Biotech’s gene therapy beremagene geperpavec-svdt (VYJUVEK), expanding its use to treat dystrophic epidermolysis bullosa (DEB) patients from birth and allowing patients and caregivers to apply the treatment at home. The update also permits wound dressings to be removed during the next dressing change rather than waiting 24 hours, enhancing patient convenience without compromising safety. VYJUVEK is a topical, redosable gene therapy that delivers copies of the COL7A1 gene to DEB wounds, addressing the disease at a molecular level by enabling skin cells to produce normal COL7 protein. Supported by real-world data and an open-label extension study, the therapy has shown sustained wound closure rates and high patient satisfaction with no new safety concerns. Krystal Biotech’s stock has surged following the label expansion, reflecting optimism about the therapy’s revenue potential and market position. Experts, including Brett Kopelan of DEBRA of America, praised the update as a significant advancement improving quality of life for those living with DEB.