SD-101 is a topical cream developed by Amicus Therapeutics, together with Scioderm, to treat skin blistering and lesions associated with epidermolysis bullosa (EB). There are no approved treatments for EB, a rare genetic connective tissue disorder that is characterized by blistering or erosion of the skin.

How SD-101 cream works

Although the mechanism of not fully understood, it is known that the cream helps in pain-management and works by healing wounds and reducing blistering and itching. Pain, itching, and infections are some of the debilitating side effects of EB.

SD-101 in clinical trials

A Phase 2 clinical trial (NCT02014376) assessed the safety and effectiveness of SD-101 in 48 patients with various subtypes of EB. Patients were randomized to take one of two doses of SD-101 (3 percent or 6 percent), or a placebo. The cream was applied topically once a day to the entire body for 90 days. Results of the study indicated that the higher dose stimulated faster, greater wound closure, was most effective and better-tolerated, compared to placebo and the lower dose of SD-101.

These results led to the advancement of SD-101 to a Phase 3 clinical trial. A double-blind, placebo-controlled Phase 3 trial (NCT02384460) known as the ESSENCE trial evaluated the safety and effectiveness in patients with various subtypes of EB. In this trial, patients were randomized 1:1 to two treatment groups and received either SD-101 at 6 percent or a placebo applied over their entire body once daily for three months. The primary goal of the study was a complete closure of a target wound, improvement in itching, pain and full-body wound, and lesion coverage.

An open-label extension study (NCT02090283) is underway to assess the continued safety of 6 percent SD-101 cream in people with EB, but the clinical trial is no longer recruiting participants.

Additional information

SD-101 has been granted breakthrough therapy designation by the U.S. Food and Drug Administration (FDA) and can be used in conjunction with a patient’s current care plan.

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