Epidermolysis Bullosa: Developing Drugs for Treatment of Cutaneous Manifestations; Guidance for Industry; Availability

摘要

The Food and Drug Administration (FDA or Agency) is announcing the availability of a final guidance for industry entitled "Epidermolysis Bullosa: Developing Drugs for Treatment of Cutaneous Manifestations." 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). This guidance focuses on drug development and trial design issues specific to the treatment of EB, including FDA's current thinking on clinical trial endpoint considerations. There is not yet sufficient clinical trial experience to establish definitive endpoints. This guidance incorporates the comments received for and finalizes the draft guidance of the same name issued on June 18, 2018.