Beremagene Geperpavec: First Approval

Drugs. 2023 Aug;83(12):1131-1135. doi: 10.1007/s40265-023-01921-5.

Abstract

Beremagene geperpavec-svdt (VYJUVEK™) is a topically applied, redosable, live, replication defective herpes simplex virus-1 (HSV-1) vector -based gene therapy that is being developed by Krystal Biotech to deliver functional human collagen type VII alpha 1 chain (COL7A1) genes in patients with both, dominant and recessive dystrophic epidermolysis bullosa. Beremagene geperpavec can transduce both keratinocytes and fibroblasts and restore functional COL7 protein. In May 2023, beremagene geperpavec received its first approval in the US for the treatment of wounds in patients ≥ 6 months of age with dystrophic epidermolysis bullosa with mutation(s) in the COL7A1 gene. A Marketing Authorization Application for beremagene geperpavec in Europe is planned for the second half of 2023. This article summarizes the milestones in the development of beremagene geperpavec leading to this first approval for dystrophic epidermolysis bullosa.

Publication types

  • Review

MeSH terms

  • Collagen / genetics
  • Collagen / metabolism
  • Collagen Type VII / genetics
  • Collagen Type VII / metabolism
  • Epidermolysis Bullosa Dystrophica* / genetics
  • Epidermolysis Bullosa Dystrophica* / metabolism
  • Genetic Therapy
  • Humans
  • Keratinocytes / metabolism
  • Mutation

Substances

  • Collagen
  • COL7A1 protein, human
  • Collagen Type VII