Epidermolysis Bullosa: Developing Drugs for Treatment of Cutaneous Manifestations; Guidance for Industry June 2019
- Docket Number:
- Issued by:
Guidance Issuing OfficeCenter for Drug Evaluation and ResearchCenter for Biologics Evaluation and Research
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.
You can submit online or written comments on any guidance at any time (see 21 CFR 10.115(g)(5))
If unable to submit comments online, please mail written comments to:
Food and Drug Administration
5630 Fishers Lane, Rm 1061
Rockville, MD 20852
All written comments should be identified with this document's docket number: FDA-2018-D-2016.