Fibrocell Receives Rare Pediatric Disease Designation from FDA for FCX-013 for Treatment of Localized Scleroderma

Gene Therapy Candidate under Control of RheoSwitch Therapeutic System® Technology is Potential First-in-Class Treatment for Chronic Disease with High Unmet Need

EXTON, Pa., June 12, 2017 (GLOBE NEWSWIRE) -- Fibrocell Science, Inc., (NASDAQ:FCSC), a gene therapy company focused on transformational autologous cell-based therapies for skin and connective tissue diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to FCX-013, Fibrocell’s gene therapy candidate for the treatment of moderate to severe localized scleroderma—a chronic autoimmune disease characterized by thickening of the skin and connective tissue. The Rare Pediatric Disease Designation augments the Orphan Drug Designation previously granted by the FDA to FCX-013 for the treatment of localized scleroderma, which includes linear scleroderma.

“We are pleased the FDA has awarded Rare Pediatric Disease Designation to FCX-013, which in addition to its Orphan Drug Designation provides important incentives to Fibrocell for developing therapies for rare pediatric diseases,” said John Maslowski, Chief Executive Officer of Fibrocell. “Moderate to severe forms of localized scleroderma, including the linear subtype, can result in significant morbidity, including pain, restricted motion, disfigurement and developmental issues.  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.”

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 FDA in the fourth quarter of 2017. 

FCX-013 and FCX-007 are being developed in collaboration with Intrexon Corporation (NYSE:XON), a leader in synthetic biology.

About Rare Pediatric Disease Designation

The FDA defines a “rare pediatric disease” as a disease that affects fewer than 200,000 individuals in the U.S. that is a serious or life-threatening disease affecting individuals primarily aged from birth to 18 years.  Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, upon the approval of a qualifying new drug application (NDA) or biologics license application (BLA) for the treatment of a rare pediatric disease, the sponsor of such application would be eligible for a Rare Pediatric Disease Priority Review Voucher that can be used to obtain priority review for a subsequent NDA or BLA. The Priority Review Voucher may be sold or transferred an unlimited number of times.

About FCX-013

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.

About Fibrocell   

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 Intrexon Corporation (NYSE:XON), a leader in synthetic biology.  For more information, visit or follow Fibrocell on Twitter at @Fibrocell.


Fibrocell, the Fibrocell logo and Fibrocell Science are trademarks of Fibrocell Science, Inc. and/or its affiliates.  All other names may be trademarks of their respective owners.

Forward-Looking Statements
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 Intrexon Corporation; and the risks, uncertainties and other factors discussed under the caption “Item 1A. Risk Factors” in Fibrocell’s most recent Form 10-K  filing and Form 10-Q filings. As a result, you are cautioned not to place undue reliance on any forward-looking statements. While Fibrocell may update certain forward-looking statements from time to time, Fibrocell specifically disclaims any obligation to do so, whether as a result of new information, future developments or otherwise.


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