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Fibrocell Receives Rare Pediatric Disease Designation from FDA for FCX-013 for Treatment of Localized Scleroderma
“We are pleased the
FCX-013 is Fibrocell’s second gene therapy candidate awarded Rare Pediatric Disease Designation. The Company also received this designation for FCX-007, its clinical-stage gene therapy candidate for the treatment of recessive dystrophic epidermolysis bullosa (RDEB). Upon market approval, both candidates are eligible to receive a Priority Review Voucher.
Fibrocell continues to make progress in the pre-clinical development of FCX-013, and expects to submit an investigational new drug application (IND) to the
FCX-013 and FCX-007 are being developed in collaboration with
About Rare Pediatric Disease Designation
Fibrocell is in pre-clinical development of FCX-013, its gene therapy candidate for the treatment of linear scleroderma, a form of localized scleroderma. FCX-013 incorporates Intrexon’s proprietary RheoSwitch Therapeutic System® (RTS®), a biologic switch activated by an orally administered compound to control future protein expression once the initial fibrosis has been resolved. FCX-013 is designed to be injected under the skin at the location of the fibrosis where the genetically-modified fibroblast cells will produce a protein to break down excess collagen accumulation. The patient takes an oral compound to facilitate protein expression. Once the fibrosis is resolved, the patient will stop taking the oral compound which will stop further production of the subject protein by FCX-013.
About Localized Scleroderma
Localized scleroderma is a chronic autoimmune skin disorder that manifests as excess production of extracellular matrix, 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. Current treatments for localized scleroderma, which include systemic or topical corticosteroids, UVA light therapy and physical therapy, only address the symptoms of the disorder. We estimate that there are approximately 40,000 patients in the U.S. who have the linear scleroderma subtype of localized scleroderma over a major joint and exhibit severe joint pain.
Fibrocell is an autologous cell and gene therapy company translating personalized biologics into medical breakthroughs for diseases affecting the skin and connective tissue. Fibrocell’s most advanced product candidate, FCX-007, is the subject of a Phase 1/2 trial for the treatment of recessive RDEB. Fibrocell is in pre-clinical development of FCX-013, its product candidate for the treatment of linear scleroderma. Fibrocell’s gene therapy portfolio is being developed in collaboration with
Fibrocell, the Fibrocell logo and
This 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: Fibrocell’s expected initiation of a toxicology/biodistribution study and submission of an IND for FCX-013 in 2017; the potential benefits of Orphan Drug Designation and Rare Pediatric Disease Designation; the potential advantages of FCX-013 and Fibrocell’s other product candidates; the sufficiency of the Company’s cash and cash equivalents to fund operations into the second quarter of 2018 and other statements regarding Fibrocell’s future operations, financial performance and financial position, prospects, strategies, objectives and other future events.
Forward-looking statements are based upon management’s current expectations and assumptions and are subject to a number of risks, uncertainties and other factors that could cause actual results and events to differ materially and adversely from those indicated herein including, among others: uncertainties and delays relating to the initiation, enrollment and completion of pre-clinical studies and clinical trials; whether pre-clinical study and clinical trial results will validate and support the safety and efficacy of Fibrocell’s product candidates; unanticipated or excess costs relating to the development of Fibrocell’s gene therapy product candidates; Fibrocell’s ability to obtain additional capital to continue to fund operations; Fibrocell’s ability to maintain its collaboration with
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