US-based biopharmaceutical firm Scioderm has started a Phase III registration trial (SD-005) of its new topical therapy, Zorblisa (SD-101), to treat blisters and lesions in patients with Epidermolysis Bullosa (EB), a rare genetic connective tissue disorder.
Currently there are no approved treatments for epidermolysis bullosa, which typically manifests at birth or early childhood.
A total of 130 patients with various subtypes of EB will be included in the randomised, double-blind, placebo-controlled Phase III trial, which is being conducted in multiple sites in the US and Europe to evaluate the safety and efficacy of Zorblisa.
During the trial, patients will be randomised 1:1 to two treatment groups receiving either Zorblisa or placebo applied over their entire body once daily for three months.
The trial’s primary efficacy endpoint will be evaluation of closure of a selected target chronic wound as well as improvement in itching, pain, full-body wound and lesion coverage.
Scioderm president and chief executive officer Robert Ryan said: “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 programme.”
Patients who complete the SD-005 trial will be offered an open-label extension trial (SD-006), which will evaluate long-term safety.
Debra of America is a national not-for-profit group that offers all inclusive care to those living with the disease by funding research and providing free services to the EB community.
Debra of America executive director Brett Kopelan said: “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.”
Recently, the company has completed a Phase II trial (SD-003) of Zorblisa in EB patients, which showed acceleration in wound healing and closure of chronic wounds.
The company secured breakthrough therapy designation for Zorblisa from the US Food and Drug Administration (FDA), as well as orphan drug designation from the FDA and European Medicines Agency (EMA).