On December 19, Chiesi Global Rare Diseases announced that the U.S. Food and Drug Administration (FDA) approved FILSUVEZ® (birch triterpenes), a topical gel for the treatment of partial thickness wounds in patients aged six months and older with Junctional Epidermolysis Bullosa (JEB) and Dystrophic Epidermolysis Bullosa (DEB).
Epidermolysis Bullosa (EB) is a family of rare genetic disorders characterized by blistering and fragility of the skin and mucous membranes that can affect the mouth, esophagus, lungs, nails, and other areas of the body. It is a debilitating condition for which there is no cure, making the news of promising treatment options all the more hopeful for both patients and their providers.
This latest approval makes FILSUVEZ the first approved treatment for wounds associated with JEB, a rare, moderate-to-severe form of EB that causes blisters beginning in infancy.
The European Commission first approved FILSUVEZ in July of 2022 for the treatment of skin wounds in adults and children aged six months and older with both JEB and DEB.
The approval of FILSUVEZ comes after positive results from the phase 3 EASE trial which included a total of 223 patients (including 156 pediatric patients) across 58 sites in 28 countries. The study comprised a double-blind, randomized controlled phase of three months followed by a 24-month open-label, single-arm phase. The study met its primary endpoint with statistical significance, demonstrating a total of 41.3% of patients achieving complete target wound closure within 45 days.
Executive Director, debra of America, Brett Kopelan, stated, “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.”