Scioderm announced its investigational product SD-101 has received Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with inherited Epidermolysis Bullosa (EB). SD-101 is Scioderm's investigational therapy that is being evaluated for the treatment of skin blistering and erosions associated with this disease, including facilitation of healing of skin lesions and reduction of the incidence and/or severity of new lesions.
"We are truly honored to have received Breakthrough Therapy designation for SD-101 and 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," said Robert Ryan., Ph.D., President and Chief Executive Officer of Scioderm. "Given the recent important milestones for Scioderm, including the Series A $16M closing along with this designation, 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 for patients with a disease that has no current effective treatment."
Breakthrough Therapy Designation was enacted as part of the 2012 Food and Drug Administration Safety and Innovation Act (FDASIA). The designation of an investigational drug as a Breakthrough Therapy is intended to expedite the development and review of a candidate that is planned for use to treat a serious or life-threatening disease or condition when preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. A breakthrough therapy designation conveys all of the fast track program features, as well as more intensive FDA guidance on an efficient drug development program. The FDA also has an organizational commitment to involve senior management in such guidance.
The FDA Breakthrough Therapy Designation for SD-101 in EB patents was based on clinical data from a previously conducted open-label Phase II study with topical administration of SD-101 in children with either Simplex, Recessive Dystrophic (RDEB), or Junctional EB. SD-101 has the potential to facilitate the healing of skin lesions and erosions in this serious and potentially life-threatening disease which has a poor prognosis.