FDA Approves Vyjuvek for Treatment of Wounds in Patients with Dystrophic Epidermolysis Bullosa

The US Food and Drug Administration (FDA) recently announced approval of VYJUVEK (beremagene geperpavec [B-VEC]) for the treatment of wounds in patients ages 6 months and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain gene following the incorrect NDC number for the vial label listed in the FDA Supplement Approval Letter, dated May 30, 2023.

B-VEC is a herpes-simplex virus type 1 vector-based gene therapy. It is a biologic suspension mixed into excipient gel for topical application, which is supplied as a 1.0 mL extractable volume in a single-dose vial at a nominal concentration of 5×109 PFU/mL. Additionally, the excipient gel is supplied as a 1.5 mL fill volume in a separate single-use vial.

The approval was granted following data from the Phase 3 study (Study B-VEC-03) and review of the Phase 1/2 study (Study KB103-001). In the Phase 3 study (Study B-VEC-03), 31 participants with DEB, aged 6 months or older at time of consent were enrolled for a trial duration roughly about 6 months, with administration occurring once weekly. Results showed that complete wound healing at 3 and 6 months in patients with DEB was more likely with topical administration of B-VEC than with placebo. In the Phase 1/2 study (Study KB103-001), the primary objectives were the evaluation of safety, including incidences of adverse events associated with the administration of B-VEC compared with placebo.

Adverse effects include itching, chills, redness, rash, cough, and runny nose of which the incidence rate was greater than 5%. Currently, there is no established safety profile in patients who are pregnant, lactating, and/or breastfeeding.

References
1. FDA approves first topical gene therapy for treatment of wounds in patients with dystrophic epidermolysis bullosa. News release. Krystal Biotech. May 19, 2023. Accessed August 9, 2023. https://www.fda.gov/news-events/press-announcements/fda-approves-first-topical-gene-therapy-treatment-wounds-patients-dystrophic-epidermolysis-bullosa

2. Gurevich I, Agarwal P, Zhang P, et al. In vivo topical gene therapy for recessive dystrophic epidermolysis bullosa: a phase 1 and 2 trial. Nat Med. 2022;28(4):780-788. doi:10.1038/s41591-022-01737-y

3. Guide SV, Gonzalez ME, Bağcı IS, et al. Trial of beremagene geperpavec (B-VEC) for dystrophic epidermolysis bullosa. N Engl J Med. 2022;387(24):2211-2219. doi:10.1056/NEJMoa2206663