FDA designates investigational drug for treating epidermolysis bullosa as breakthrough therapy

The FDA has designated an investigational treatment for patients with inherited epidermolysis bullosa as breakthrough therapy, Scioderm announced this week.

SD-101, a topical cream developed by the Durham, N.C.,-based pharmaceutical company, is being evaluated for treating skin blistering and erosions associated with inherited epidermolysis bullosa (EB), according to a press release. The rare genetic connective tissue condition results in fragile skin that tears easily from the slightest friction or trauma.

The FDA designation was based on clinical data from a phase 2 study of SD-101 for treating 101 children with simplex, recessive dystrophic or junctional EB. Within 1 month, SD-101 treatment resulted in complete closure of 88% of target chronic lesions and a 57% reduction in body surface area coverage of lesions and erosions after 3 months of daily treatment. Scioderm reported that SD-101 was well tolerated by patients.

There is no cure or effective treatment for EB, according to the release. Daily wound care, pain management and preventive bandaging are currently the only options. More severe forms of EB can lead to scarring, disfigurement, disability and early death, often before age 30 years, Scioderm reported.

“We are … pleased with the FDA’s decision to place our product in a category that may enable expedited development and review for patients with epidermolysis bullosa,” Robert Ryan, PhD, president and chief executive officer of Scioderm, said in the release. “We are looking forward to the opportunity to collaborate more closely with the FDA and potentially expedite the availability of an important new treatment option.”

The breakthrough therapy designation, created in 2012, allows expedited development and review of drugs shown in preclinical studies to offer potentially substantial improvements over existing therapies for patients with serious or life-threatening diseases.