VYJUVEK (beremagene geperpavec) - Dystrophic epidermolysis bullosa (DEB)

The clinical benefit of VYJUVEK (beremagene geperpavec) suspension and gel for gel is substantial in the MA indication.

Considering:

• an insufficiently covered medical need in dystrophic epidermolysis bullosa (DEB) from birth, consisting solely of skincare delivered by nurses, dressings and interfaces (medical devices);
• the demonstration of the superiority, with a substantial size effect, in a phase 3 randomised (intra-subject), double-blind study, versus a placebo (excipient gel) in 31 patients with DEB with genetically-confirmed mutation(s) in the COL7A1 gene on the proportion of patients with complete wound closure for 2 consecutive weeks at 6 months (primary endpoint, ∆ = 45.8%) and 3 months (∆ = 51.0%);
• the safety profile, characterised primarily by chills and pruritus;

despite:

• the absence of robust data for the assessment of pain associated with wound dressing changes on the primary wounds, assessed in an exploratory manner using a visual analogue scale (VAS) or by the FLACC-R score (children < 6 years of age);
• the lack of efficacy data for beremagene geperpavec in dominant DEB (only one patient included in the GEM-3 study) and in the most severe generalised forms of DEB since these patients practically all have a history of squamous cell carcinoma (exclusion criterion);
• the demonstration of efficacy in the population from birth based on data from patients ≥ 1 year of age included in the clinical study compared with real-world data from patients < 1 year of age who received beremagene geperpavec in the context of a compassionate access programme in Europe or post-marketing in the USA;

the Committee deems that VYJUVEK (beremagene geperpavec) suspension and gel for gel provides a moderate clinical added value (CAV III) in the treatment of wounds in patients with dystrophic epidermolysis bullosa (DEB) with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene, from birth.