FDA Approves Birch Triterpenes Topical Gel for Treatment for Adult, Pediatric Patients With JEB, DEB

The FDA has approved the topical gel birch triterpenes (Filsuvez; Amryt Pharmaceuticals) for the treatment of partial thickness wounds in patients aged 6 months and older with junctional epidermolysis bullosa (JEB) and dystrophic epidermolysis bullosa (DEB). This approval makes birch triterpenes the first treatment for wounds associated with JEB to be approved by the FDA.

Birch triterpenes is administered at home and applied topically to wounds, meaning it can be integrated into existing treatment regimens. It is indicated for the treatment of wounds that are associated with dystrophic and junctional epidermolysis bullosa in both adult and pediatric patients who are aged 6 months and older.

The approval comes after results from the EASE trial (NCT03068780) which is the largest global phase 3 trial conducted in patients with EB. It had a 3-month double-blind, randomized, controlled phase followed by a 24-month open-label, single-arm phase. A total of 223 enrolled patients (pediatric patients: n = 156) had EB target wounds between 10 and 50 cm² in size that were present for at least 21 days and less than 9 months. Patients were randomly assigned to receive birch triterpenes and wound dressings, which were applied according to the standard of care.

The primary endpoint of the trial was to compare the efficacy of birch triterpenes gel vs the control gel according to the number of patients with complete closure of the target wound within 45 days of treatment. The primary endpoint was achieved with statistical significance, whereas the key secondary endpoints did not achieve statistical significance; however, several favorable differences were still observed.

“We are grateful for the support of those living with EB and their dedicated caregivers, which allowed us to reach this landmark FDA approval and proudly provide [birch triterpenes] as a solution for wound care management,” said Giacomo Chiesi, head of Chiesi Global Rare Diseases, in a press release. 

The most frequently reported adverse effects (AEs) in clinical trials were pruritis and pain at the wound application site (7.3%). In addition, local hypersensitivity and skin reactions (eg, urticaria, dermatitis) were reported in patients who received treatment with birch triterpenes.

“The FDA’s decision to approve [birch triterpenes] provides those living with EB a safe and effective treatment option for the most prominent and difficult symptom of EB, open wounds that may not heal,” said Brett Kopelan, executive director, debra of America, in the press release. “Today marks an important milestone for those living with junctional EB, as [birch triterpenes] is the first FDA approved treatment for this variant of the disease.”

Reference

GlobalNewswire. Chiesi Global Rare Diseases Receives FDA Approval for FILSUVEZ® (birch triterpenes) topical gel for the Treatment of Epidermolysis Bullosa. News release. December 19, 2023. Accessed December 19, 2023. https://www.globenewswire.com/news-release/2023/12/19/2798751/0/en/Chiesi-Global-Rare-Diseases-Receives-FDA-Approval-for-FILSUVEZ-birch-triterpenes-topical-gel-for-the-Treatment-of-Epidermolysis-Bullosa