The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to FX-013, a Fibrocell gene therapy candidate to treat children with moderate to severe localized scleroderma.
“Fibrocell CEO John Maslowski said in in a press release that the designation, in addition to FX-013’s Orphan Drug Designation, “provides important incentives to Fibrocell for developing therapies for rare pediatric diseases.”
Both FDA designations will help evaluate and develop FX-013, now in the pre-clinical stage. Fibrocell expects to ask the FDA for Investigational New Drug status for FX-013 by the end of 2018.
Localized scleroderma is a chronic autoimmune skin disorder that causes thickening of the skin in localized areas and may extend to underlying tissue and muscle. In children, this can impair growth and development; painful lesions may also appear, hindering movement.
“Moderate to severe forms of localized scleroderma, including the linear subtype, can result in significant morbidity, including pain, restricted motion, disfigurement and developmental issues,” Maslowski said, estimating that 40,000 patients have the linear subtype. “With no FDA-approved therapies available, we believe controlled gene therapy through FCX-013 offers promise to address this high unmet medical need of patients suffering from this chronic and often debilitating disease.”
FX-013 may help localized scleroderma patients by eliminating the underlying cause of the disorder: excessive production of collagen, a protein of great importance to provide structure and support to cells.
Fibrocell, based in Exton, Pennsylvania, created this genetic therapy to induce the formation of one specific protein that can break down the excess collagen formed by skin cells of scleroderma patients.
Fibrocell has designed a four-step therapy plan for treating localized scleroderma with FX-013. First, skin cells are removed from the patient through a small biopsy procedure and kept under laboratory conditions. Cells are then genetically modified using FX-013 gene therapy to start producing the protein that will break down collagen. The genetically modified skin cells are then injected back under the skin of the same patient from which they came, at the site of the lesion.
Taking an oral compound activates the genetically modified cells to form this new protein. Once the lesion has been treated, the patient will stop taking this compound and the collagen degrading protein will no longer be produced.