Dystrophic epidermolysis bullosa (DEB) is caused by mutation(s) in the COL7A1 gene, which results in reduced or absent levels of biologically active COL7. Upon topical application to the wounds, beremagene geperpavec can transduce both keratinocytes and fibroblasts. Following entry of beremagene geperpavec into the cells, the vector genome is deposited in the nucleus. Once in the nucleus, transcription of the encoded human COL7A1 is initiated. The resulting transcripts allow for production and secretion of COL7 by the cell in its mature form. These COL7 molecules arrange themselves into long, thin bundles that form anchoring fibrils. The anchoring fibrils hold the epidermis and dermis together and are essential for maintaining the integrity of the skin.
This medicinal substance has been classified in the anatomical therapeutic chemical (ATC) classification according to its main therapeutic use as follows:
| ATC code | Group title | Classification |
|---|---|---|
| D03AX16 | D Dermatologicals → D03 Preparations for treatment of wounds and ulcers → D03A Cicatrizants → D03AX Other cicatrizants | |
Competent medicine agencies globally have authorized commercialization of this active ingredient according to these medication package inserts (MPIs):
| Title | Information Source | Document Type | |
|---|---|---|---|
| VYJUVEK Kit, biological suspension and excipient gel for topical application | FDA, National Drug Code (US) | MPI, US: SPL/PLR |
Beremagene geperpavec is an active ingredient of these brands:
United States (US)Note the following: The list of brand names is continuously updated, and thus does not include the total of products circulating worldwide. |
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