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FDA Grants Orphan Drug Status to Gene-Therapy Candidate for Localized Scleroderma
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Fibrocell Science announced in a press release that it has received orphan drug designation from the FDA for its FCX-013 gene-therapy candidate to treat localized scleroderma.
FCX-013, an autologous fibroblast cell genetically modified to express a protein to breakdown excess collagen I and III at the site of localized disease, is in preclinical development for treating linear scleroderma, a form of localized scleroderma, according to the release. Fibrocell is an autologous cell and gene therapy company focused on developing therapies for diseases of the skin, connective tissues and joints.
“FCX-013 incorporates Intrexon Corporation’s RheoSwitch Therapeutic System, a biologic switch activated by an orally administered compound, which allows control of future protein expression once the initial fibrosis has been resolved,” according to the release.
“Achieving orphan drug designation for FCX-013 is an important regulatory milestone for us,” David Pernock, chairman and CEO of Fibrocell, stated in the 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 preclinical dose ranging and toxicology/biodistribution studies for product optimization.”
Fibrocell reports that it expects to submit an investigational new drug application to the FDA for FCX-013 in 2017.
Fibrocell previously received orphan drug designation for another gene therapy product, FCX-007, which was given allowance from the FDA to begin a phase I/II clinical trial in adults for treating recessive dystrophic epidermolysis bullosa.
The FDA offers orphan drug designation to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,00 patients in the U.S., providing the sponsor with a 7-year period of U.S. marketing exclusivity from launch, tax credits and the ability to apply for annual grant funding, according to the release.
Reference: www.fibrocell.com