Linear scleroderma, a form of localized scleroderma, is a chronic autoimmune skin disorder that manifests as excess production of extracellular matrix—specifically Type I collagen and Type III collagen—resulting in thickening of the skin and connective tissue. The localized areas of skin thickening may extend to underlying tissue and muscle in children which can impair growth and development. Lesions appearing across joints can be painful, impair motion and may be permanent.
Localized scleroderma is comprised of a number of forms or sub-types totaling slightly less than 200,000 people in the U.S.2 The U.S. population of patients who have linear scleroderma over a major joint and exhibit severe joint pain is estimated to be approximately 40,000.
Current treatments for linear scleroderma, including systemic or topical corticosteroids, UVA light therapy and physical therapy, only address the symptoms of the disorder.
FCX-013 is an autologous fibroblast cell genetically modified to express a protein to breakdown excess collagen I and III at the site of the localized disease. FCX-013 incorporates Intrexon’s proprietary RheoSwitch Therapeutic System®, a biologic switch activated by an orally administered compound which allows us to control future protein expression once the initial fibrosis has been resolved. FCX-013 is currently in pre-clinical development for the treatment of linear scleroderma, a form of localized scleroderma.
Fibrocell has received orphan drug designation from the U.S. Food and Drug Administration for FCX-013 for the treatment of localized scleroderma.
Dermal fibroblasts are collected from the patient, cultured and genetically modified to regulate collagen accumulation and then injected at the site of the localized disease.
Photo: © 2015 American College of Rheumatology. Used with permission.