The Food and Drug Administration (FDA) has approved Vyjuvek™ (beremagene geperpavec-svdt) for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.
Dystrophic epidermolysis bullosa (DEB) is a chronic and progressive skin disease caused by mutation(s) in the COL7A1 gene that leads to extremely fragile skin, blistering, and skin loss at the slightest friction. Vyjuvek is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy designed to deliver 2 copies of the COL7A1 gene when applied directly to DEB wounds.
The approval was based on data from the double-blind, placebo-controlled phase 3 GEM-3 trial (ClinicalTrials.gov Identifier: NCT04491604), which included 31 patients aged 1 year and older with DEB. Patients provided at least 1 pair of primary target wounds for evaluation, with 1 wound from each pair treated with Vyjuvek and the other with placebo for 26 weeks.
Results showed that the study met its primary endpoint; 65% of wounds treated with Vyjuvek completely closed at 24 weeks (defined as durable wound closure evaluated at 2 consecutive visits 2 weeks apart) compared with 26% of those treated with placebo (treatment difference, 39%; 95% CI, 14-63; P =.012). Moreover, a greater proportion of wounds treated with Vyjuvek completely closed at weeks 8 and 10 or weeks 10 and 12 (secondary endpoint) compared with those treated with placebo (68% vs 23%; treatment difference, 45%; 95% CI, 22-69; P =.003). The most common adverse reactions reported were itching, chills, redness, rash, cough, and runny nose.
“Vyjuvek both heals patient wounds and prevents skin from re-blistering because it actually corrects the underlying skin defect of dystrophic EB,” said M. Peter Marinkovich, MD, primary investigator of the GEM-3 trial, Director of the Blistering Disease Clinic at Stanford Health Care and Associate Professor of Dermatology at the Stanford University School of Medicine. “Because it’s safe and easy to apply directly to wounds, it doesn’t require a lot of supporting technology or specialized expertise, making Vyjuvek highly accessible even to patients who live far away from specialized centers.”
Vyjuvek gel is applied topically to wound(s) once a week. The biological suspension is mixed into an excipient gel vial prior to administration. The product is expected to be available in the third quarter of 2023. Treatment is administered only by a health care professional.
- Krystal Biotech receives FDA approval for the first-ever redosable gene therapy, Vyjuvek™ (beremagene geperpavec-svdt) for the treatment of dystrophic epidermolysis bullosa. News release. Krystal Biotech. Accessed May 19, 2023. https://ir.krystalbio.com/news-releases/news-release-details/krystal-biotech-receives-fda-approval-first-ever-redosable-gene.
- Vyjuvek. Package insert. Krystal Biotech; 2023. Accessed May 19, 2023. https://www.krystallabel.com/pdf/vyjuvek-us-pi.pdf.