FDAnews Drug Daily Bulletin
Pharmaceuticals / Regulatory Affairs

FDA Issues Recommendations for Developing Epidermolysis Bullosa Drugs

June 21, 2018

New FDA draft guidance outlined the agency’s approach to drugs for treatment of cutaneous epidermolysis bullosa.

In designing clinical trials for such drugs, sponsors should document any genetic testing performed on the trial population, according to the guidance. However, testing should not be required for study entry if mutational analysis does not tie into the desired claim. Repeat testing is not necessary for patients with adequate prior documented diagnosis.

Trials should demonstrate a clinically meaningful improvement in at least one sign or symptom of EB. This could include significant reduction of itching or pain, wound healing or blister prevention.

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