FILSUVEZ® (birch triterpene gel), the world's first approved topical therapy for Epidermolysis Bullosa (EB), has been administered to a patient in China for the first time, marking a significant milestone in the country's efforts to improve care for patients living with the rare genetic skin disorder.

The treatment was carried out at the Boao Lecheng International Medical Tourism Pilot Zone, a special medical access zone that enables patients in China to receive innovative therapies approved overseas before they become widely available domestically.

The first patient to receive the therapy, a teenage boy from Jiangxi Province in eastern China, has lived with EB since childhood. Like many people affected by the condition, he has endured chronic wounds, persistent pain, and the physical and emotional burden of intensive daily wound care.

A multidisciplinary team led by Professor Li Ming of the Children's Hospital of Fudan University and Professor Huang Xiaoyan of the Hainan Women and Children's Medical Center developed an individualized treatment plan and implemented comprehensive safety measures for the inaugural use of the therapy in China. According to the medical team, the patient has tolerated the treatment well, with no treatment-related adverse events reported to date.

EB is a group of rare inherited disorders that leave the skin exceptionally fragile. Even minor friction can cause painful blisters and open wounds, earning many affected children the nickname "butterfly children" because of the delicate nature of their skin.

The disease is included in China's first national list of rare diseases, and an estimated 15,000 people in the country are believed to be living with the condition.

Until now, treatment options have largely been limited to supportive wound care and symptom management.

Physicians monitor the patient's condition during treatment.

FILSUVEZ® is the first approved medication specifically developed to promote wound healing in patients with certain forms of EB. The therapy has received orphan drug designation from both the European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA), and was approved in Europe in 2022 and in the United States in 2023.

Thanks to Boao Lecheng's special access policy, FILSUVEZ® is currently the only available treatment for EB in China’s mainland.

In addition to administering the therapy for the first time, researchers also launched China's first real-world study evaluating FILSUVEZ® in patients with inherited Epidermolysis Bullosa. Co-led by Professors Li and Huang, the study aims to generate real-world evidence on the therapy's effectiveness and safety in Chinese patients, providing data to support future clinical practice and broader access.

"This first treatment marks an important milestone," said Professor Li, one of the study's principal researchers. "We hope to accelerate the study, generate robust evidence on the safety and effectiveness of FILSUVEZ® in Chinese patients, and ultimately benefit more people living with EB."

Professor Huang said the successful treatment reflects the close collaboration between the two clinical teams and represents "the first step in bringing innovative EB therapies to patients in China." She added that the real-world study is expected to help advance the country's standards of care for EB and improve outcomes for more patients.

Under Hainan's special medical policies, patients receiving treatment at Boao Lecheng may continue therapy after discharge by taking home a physician-prescribed supply of FILSUVEZ®, supporting long-term disease management.

The therapy has also been included in Lecheng's specialty drug insurance program and several regional supplementary health insurance schemes, helping reduce the financial burden on affected families through shared reimbursement mechanisms.