Topical Treatment for Epidermolysis Bullosa Wins FDA Approval

The FDA approved birch triterpenes (Filsuvez) topical gel for the treatment of epidermolysis bullosa (EB), Chiesi Global Rare Diseases announced on Tuesday.

The approval stipulates use for partial thickness wounds in adults and pediatric patients at least 6 months old with dystrophic and junctional EB (JEB). The topical gel is the first approved treatment specifically indicated for JEB, a rare, moderate-to-severe form of EB that causes blisters beginning in infancy.

“The FDA’s decision to approve Filsuvez 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 of debra of America, in the company statement.

Support for the approval came from the international phase III randomized EASE trial comparing birch triterpenes gel with a control gel in 223 patients with EB, including 156 pediatric patients. The trial met the primary endpoint, showing that 41.3% of patients treated with the active gel achieved first complete target wound closure within 45 days, as compared with 28.9% of the control group (P=0.013).

Adverse events occurred in a similar proportion of patients in both treatment groups, the most common of which were application site reactions. Most were grade 1/2 in severity.

The birch triterpenes topical gel contains dry extract from two species of birch bark, according to the company statement. The bark contains naturally occurring triterpenes, including betulin, betulinic acid, erythrodiol, lupeol, and oleanolic acid.

According to the product labeling, birch triterpenes gel should be applied to a wound surface at a thickness of 1 mm and covered by a wound dress until healed. The gel may also be applied directly to the dressing. It should not be rubbed into the wound.

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