SD-101 Cream for Epidermolysis Bullosa

SD-101 was a topical cream being developed by Amicus Therapeutics together with Scioderm to treat skin blistering and lesions in patients with epidermolysis bullosa (EB), a rare genetic connective tissue disorder currently with no available treatments. 

The cream failed to show improvement in wound healing in a Phase 3 clinical trial, and Amicus has announced that it will no longer develop the treatment.

How does SD-101 cream work?

SD-101 contains 6% allantoin, which is a natural product present in human diets. Although researchers did not fully understand its mechanism of action, they thought that the cream could help in pain management and wound healing, while also reducing blistering and itching.

SD-101 in clinical trials

A Phase 2 clinical trial (NCT02014376) assessed the safety and effectiveness of SD-101 in 48 patients with various types of EB. Researchers randomized patients to use one of two doses of SD-101 (3% or 6% allantoin), or a placebo. They applied the cream topically once a day to the entire body for 90 days. The results indicated that the higher dose stimulated faster, greater wound closure, and was most effective and better-tolerated, compared to the placebo and the lower dose of SD-101. These results led to the advancement of SD-101 into a Phase 3 clinical trial.

The double-blind, placebo-controlled Phase 3 ESSENCE trial (NCT02384460) evaluated the safety and effectiveness of SD-101 in patients with various types of EB. Researchers randomized patients 1:1 to two treatment groups and gave them either SD-101 at 6% or a placebo that was applied over their entire body once daily for three months. The primary goal of the study was to measure the number of patients with complete closure of a target wound, improvement in itching, pain, and full-body wound, and lesion coverage.

The results, published in the Orphanet Journal of Rare Diseases in June 2020, indicated that the trial did not meet its primary objectives. No significant differences were found between patients who used SD-101 and those who used a placebo. Researchers had permitted patients to continue in an open-label extension study (NCT02090283), which has since been terminated. No further studies are planned.

Additional information

The U.S. Food and Drug Administration had granted SD-101 its breakthrough therapy designation, but clinical development of the treatment has been discontinued. 

Last updated: July 7, 2020

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