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Fibrocell Receives Orphan Drug Designation from the FDA for FCX-013 for the Treatment of Localized Scleroderma
FCX-013 Gene-Therapy Product Candidate Offers Potential to Address Painful Fibrotic Disease
EXTON, Pa., April 25, 2016 (GLOBE NEWSWIRE) -- Fibrocell Science, Inc., (NASDAQ:FCSC), an autologous cell and gene therapy company focused on developing transformational therapies for diseases affecting the skin, connective tissue and joints, today announced that it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for FCX-013 for the treatment of localized scleroderma, a chronic autoimmune disease characterized by thickening of the skin and connective tissue that can be debilitating and painful.
FCX-013 is Fibrocell's second gene-therapy product candidate to receive orphan drug designation. The Company's other orphan gene-therapy product candidate, FCX-007, recently received allowance from the FDA to initiate a Phase I/II clinical trial in adults for the treatment of recessive dystrophic epidermolysis bullosa.
About Orphan Drug DesignationOrphan drug designation is granted by the FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the United States. Subject to certain conditions, the designation provides the sponsor with a seven-year period of U.S. marketing exclusivity from launch, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance, and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
About FibrocellFibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs. Fibrocell's most advanced product candidate, azficel-T, uses its proprietary autologous fibroblast technology and is in a Phase II clinical trial for the treatment of vocal cord scarring resulting in chronic or severe dysphonia. In collaboration with Intrexon Corporation (NYSE:XON), a leader in synthetic biology, Fibrocell is also developing gene therapies for diseases affecting the skin, connective tissue and joints using genetically-modified autologous fibroblasts. Fibrocell recently received allowance from the FDA to initiate a Phase I/II clinical trial of FCX-007 in adult subjects, and expects to initiate the Phase I portion of the trial in the second quarter of 2016. FCX-007 is Fibrocell's lead gene-therapy product candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Fibrocell is in pre-clinical development of FCX-013, its gene-therapy product candidate for the treatment of linear scleroderma. In addition, Fibrocell and Intrexon are in collaboration to develop a gene therapy for the treatment of arthritis. For more information, visit www.fibrocell.com or follow us on Twitter at @Fibrocell.
Fibrocell TrademarksFibrocell™ and Fibrocell Science® are trademarks of Fibrocell Science, Inc. and/or its affiliates. All other names may be trademarks of their respective owners.
Fibrocell Forward-Looking StatementsThis press release contains, and our officers and representatives may from time to time make, statements that are "forward-looking statements" within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. All statements that are not historical facts are hereby identified as forward-looking statements for this purpose and include, among others, statements relating to: the initiation, design and timing of our Phase I/II clinical trial for FCX-007; the timing of the submission of an IND for FCX-013; the potential benefits of orphan drug designation; the potential advantages of our product candidates; and other statements regarding our future operations, financial performance and financial position, prospects, strategies, objectives and other future events.
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