Understanding the efficacy and tolerability of topical therapies for epidermolysis bullosa

Epidermolysis Bullosa (EB) is a group of rare inherited blistering disorders of the skin, characterized by fragile skin and mucous membranes. Topical therapies are central to symptom management and wound care, and recent advances provide potential for topical treatments to be disease-modifying approaches.

This review examines the clinical efficacy and tolerability of topical therapies for EB, with a particular focus on approved treatments such as beremagene geperpavec (B-VEC) and Oleogel-S10, as well as emerging agents like allantoin, diacerein, sirolimus, gentamicin, gentian violet and calcipotriol. Using evidence from clinical trials and pilot studies, their mechanisms and outcomes can be evaluated for different EB subtypes.

Topical therapies for EB are evolving, with more gene-based treatments and anti-inflammatory agents showing potential for improving wounds, reducing pain and overall increasing quality of life. Challenges remain in trial designs, accessibility and long-term safety studies, especially in the context of the heterogeneity of EB. The continued use of standardized outcome measures will affect the future progress of studies, as well as using real-world data. Whilst a definitive cure is unlikely for EB, evolving topical strategies have strong potential in providing control of the disease's symptoms and ultimately can improve long-term outcomes.