Fibrocell Science, Inc., recently announced that the U.S. Food and Drug Administration (FDA) has designated FCX-013 an orphan drug for the potential treatment of localized scleroderma, a chronic autoimmune disease in which the skin and the connective tissue thicken in debilitating ways.
Fibrocell Science, specialized in personalized biologics, is currently working on the pre-clinical development of FCX-013 for linear scleroderma, a form of localized scleroderma.
FCX-013 is an autologous fibroblast cell that has been genetically modified to express a protein capable of breaking down excess collagen I and III at localized scleroderma sites. The treatment uses Intrexon’s proprietary RheoSwitch Therapeutic System, described as a biologic switch, activated by the oral compound, that allows for control of future protein expression once the initial fibrosis has been resolved.
Fibrocell’s FCX-013 is the company’s second gene therapy product candidate to receive orphan drug designation. The other is FCX-007, which has recently been given FDA clearance to begin a Phase 1/2 clinical trial in adults for the treatment of recessive dystrophic epidermolysis bullosa (RDEB).
“Achieving orphan drug designation for FCX-013 is an important regulatory milestone for us,” David Pernock, Fibrocell’s chairman and chief executive officer, said in a press release. “We recently reported the successful completion of a proof-of-concept study in which FCX-013 reduced the dermal thickness of fibrotic tissue in a scleroderma rodent model. Based on these results, we advanced FCX-013 into pre-clinical dose ranging and toxicology/biodistribution studies for product optimization. We expect to submit an Investigational New Drug application for FCX-013 to the FDA in 2017.”
An orphan drug designation is a status given by the FDA’s Office of Orphan Products Development (OOPD) to novel drugs or biologics that are indicated for the treatment of a rare disease or condition, defined in the U.S. as those affecting less than 200,000 people. Once granted, the designation gives the drug’s sponsor a seven-year period of U.S. marketing exclusivity from its launch, should the drug be approved for use, in addition to tax credits for clinical research costs and clinical trial design assistance, among other potential incentives.
In many patients, scleroderma damages structures beyond the skin, targeting blood vessels, the lungs and other organs, and the digestive tract.