Dystrophic epidermolysis bullosa

Active Ingredient: Beremagene geperpavec

Indication for Beremagene geperpavec

Population group: only infants (40 days - 1 year old) , children (1 year - 12 years old) , adolescents (12 years - 18 years old) , adults (18 years old or older)
Therapeutic intent: Curative procedure

Beremagene geperpavec is indicated for the treatment of wounds in patients 6 months of age and older with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.

For this indication, competent medicine agencies globally authorize below treatments:

For patients 6 months to <3 years old 1.6 × 10⁹ PFU maximum weekly dose and for patients ≥3 years old 3.2 × 10⁹ PFU maximum weekly dose

For:

Dosage regimens

Regimen A: In case that patient age in months is ≥ 6 and patient age in years is ≤ 3, cutaneous, 1,600,000,000 plaque-forming units beremagene geperpavec, divided weekly.

Regimen B: In case that patient age in years is ≥ 3, cutaneous, 3,200,000,000 plaque-forming units beremagene geperpavec, divided weekly.

Detailed description

The recommended dose of beremagene geperpavec is based on age. Beremagene geperpavec is applied topically to wound(s) once a week.

Maximum weekly dose by age:

Age rangeMaximum weekly dose
(plaque forming units;
PFU)
6 months to <3 years old 1.6 × 109
≥3 years old 3.2 × 109

Apply beremagene geperpavec to wounds until they are closed before selecting new wound(s) to treat. Prioritize weekly treatment to previously treated wounds if they re-open.

If a dose is missed, apply beremagene geperpavec as soon as possible and resume weekly dosing thereafter.

Active ingredient

Beremagene geperpavec

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.

Read more about Beremagene geperpavec

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