FILSUVEZ (refined dry extract of birch bark) - Dystrophic epidermolysis bullosa

The clinical benefit of FILSUVEZ is low, only in the treatment of partial thickness wounds associated with recessive dystrophic epidermolysis bullosa (EB) in patients 6 months and older.

Considering:

• evidence in a study of good methodological quality of the superiority of FILSUVEZ (refined dry extract of birch bark) gel compared to a placebo gel, in terms of achievement of first complete closure at D45, in 223 patients (children and adults) with inherited EB receiving the standard treatment, including the application of non-adherent dressings and interfaces, but a modest difference in favour of FILSUVEZ,
• the predominance of patients with recessive dystrophic EB in the study, representing 78.5% of the sample size, and results in this subpopulation suggesting a greater clinical benefit in terms of complete closure of the wound at D45 (44.0% vs. 26.2%),
• uncertainties in terms of the efficacy in patients with dominant dystrophic EB and junctional EB, representing only 9% (n = 20) and 11.7% (n = 26) of patients included in the primary analysis population,
• the absence of:
  • evidence of the superiority of FILSUVEZ compared to placebo gel on the first of the ranked secondary endpoints (time to complete closure of the target wound until the end of the treatment period at D90) and, consequently, on none of the other ranked secondary endpoints,
  • evidence of efficacy on maintenance of healing, which is the real challenge of treatment, although this was initially scheduled in the protocol, limiting the clinical relevance of the primary endpoint,
  • robust data on the quality of life of patients or caregivers,
• the safety profile marked, in particular, by a risk of wound complications (increase in wound size, reopening of the wound and wound pain: 61.5% in the FILSUVEZ group versus 53.5 % in the placebo gel group),
• the medical need currently partially met by symptomatic non-medicinal management including skincare delivered by nurses, dressings and interfaces (medical devices) and monitoring for complications,

the Committee considers that FILSUVEZ provides no clinical added value (CAV V) in the current care pathway for partial thickness wounds associated with recessive dystrophic bullosa epidermolysis bullosa (EB) in patients 6 months and older.