Rare Disease Report

Phase 3 Registration Trial Begins for Scioderm's Epidermolysis Bullosa Treatment

MARCH 19, 2015
James Radke

Scioderm announced it has started its Phase 3 registration trial to test the efficacy and safety of Zorblisa (SD-101), for the treatment of blisters and lesions in patients with epidermolysis bullosa (EB).

EB is a rare genetic connective tissue disorder that typically manifests at birth or early childhood and for which there are no approved treatments. There are many genetic and symptomatic variations of EB, but all forms share the common symptom of fragile skin that blisters and tears from the slightest friction or trauma. Current standard of care consists of bandaging and bathing the open wounds to prevent infection and trying to manage patients’ pain. There are approximately 20,000-30,000 people living with EB in the United States, and 300,000-400,000 worldwide.

Robert Ryan, PhD, president and chief executive officer said in a press release:

“Scioderm is dedicated to improving the lives of people with EB, a devastating disorder for which there are no effective treatment options available today.

“Initiating this study is an important milestone for the EB community and for our Zorblisa clinical development program.“


The Phase 3 trial is a randomized, double-blind, placebo-controlled trial being conducted in multiple sites in the U.S. and Europe involving 130 patients with various subtypes of EB (Simplex, Recessive Dystrophic and Junctional [non-Herlitz]). Participants will receive either Zorblisa or placebo applied over their entire body once daily for three months. The primary efficacy endpoint will be evaluation of closure of a selected target chronic wound. An open-label extension trial will be offered to patients at the end of the trial.

Brett Kopelan, executive director of debra of America added:

“EB is ‘the worst disease you’ve never heard of’ because it attacks those that live daily with it physically, emotionally and financially.

“Unfortunately, there are no approved treatments, so those with EB rely solely on pain management, daily wound care and preventative bandaging. We are excited to learn more about the potential of Zorblisa in helping to alleviate some of the most persistent and problematic symptoms of EB.”


For more information about this devastating disease, visit our coverage from the most recent EB conference.

Reference

Scioderm Initiates Phase 3 Registration Trial of ZorblisaTM (SD-101), a Novel Topical Therapy for Patients with Epidermolysis Bullosa [press release]. Durham NC; Scioderm Inc; March 19, 2015. http://www.businesswire.com/news/home/20150319005320/en/Scioderm-Initiates-Phase-3-Registration-Trial-ZorblisaTM#.VQq3GmboYiE  Accessed March 19, 2015.

Image of boy with EB By Master Sgt. Keith A. Milks [Public domain], via Wikimedia Commons

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