Filsuvez® (birch triterpenes) topical gel from Chiesi Global Rare Diseases has received FDA approval for the treatment of partial thickness wounds in patients 6 months and older with Junctional Epidermolysis Bullosa (JEB) and Dystrophic Epidermolysis Bullosa (DEB). This brings the number of FDA NDA approvals to 54 and marks the fourth NDA for a dermatologic indication this year.
Filsuvez is the first approved treatment for wounds associated with JEB. Chiesi acquired the at-home use agent as part of its acquisition of Amryt Pharma in January of this year.
The EASE trial, the largest ever global Phase 3 trial conducted in patients with EB, was performed across 58 sites in 28 countries to assess the safety and efficacy of Filsuvez. The study involved a 3-month double-blind randomized controlled phase followed by a 24-month open-label, single-arm phase. Patients with EB target wounds of between 10 and 50cm2 in size that were present for > 21 days and < 9 months were randomized in the double-blind phase to study treatment in a 1:1 ratio and wound dressings applied according to the standard of care. Of 223 patients enrolled in the trial (including 156 pediatric patients), 100% of those who completed the double-blind phase entered the open-label safety follow-up phase. The primary endpoint of the trial was the proportion of patients with complete closure of the target wound within 45 days of treatment. The primary endpoint was achieved with statistical significance (p = 0.013). While the key secondary endpoints did not achieve statistical significance, Chiesi says a number of favorable differences were observed.
Filsuvez was previously approved in June 2022 by the European Commission for the treatment of skin wounds in adults and children, ages 6 months and older with both JEB and DEB.
