The purpose of this guidance is to assist sponsors with the development of drugs for treatment or prevention of the serious cutaneous manifestations of the heterogeneous group of disorders collectively known as epidermolysis bullosa (EB). The paucity of effective treatment options for EB represents an important unmet medical need.

This guidance focuses on drug development and trial design issues specific to the treatment of EB, including FDA’s current thinking on trial endpoints. There is not yet sufficient clinical trial experience to establish definitive endpoints.