Fibrocell Science Receives Orphan Drug Designation for Genetically-Modified Autologous Human Fibroblasts to Advance Treatment Development for Recessive Dystrophic Epidermolysis Bullosa (RDEB)

In Press Release by Cameron

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RDEB recognized as most severe form of all epidermolysis bullosa conditions

June 13, 2014

Fibrocell Science, Inc., an autologous cell therapy company primarily focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to its genetically-modified autologous human fibroblasts to treat dystrophic epidermolysis bullosa (DEB), a broad category of rare and severe genetic skin diseases that includes RDEB. Fibrocell is working with leading medical centers—whose research focuses on all forms of epidermolysis bullosa, including DEB and RDEB—to advance development of its lead therapeutic candidate for the most severe form of epidermolysis bullosa, RDEB.

Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products to drugs intended to treat a rare disease or condition affecting fewer than 200,000 people in the U.S. There are between 2,800 and 5,600 RDEB patients in the U.S. and typically, they do not live beyond 30 years of age. No cure or treatment is available for this disease beyond basic wound care.

The genetically-modified fibroblasts combine Fibrocell’s autologous fibroblast technology with Intrexon Corporation’s (NYSE:XON) UltraVector® synthetic biology platform to target the fundamental source of this devastatingly debilitating, genetic disease that causes severe blistering and areas of missing skin.

“The fibroblast cells of RDEB patients lack the ability to produce functional collagen VII, the essential protein that forms anchoring fibrils responsible for attaching the layers of the skin together,” said David Pernock, chairman and chief executive officer of Fibrocell. “In collaboration with Intrexon, the gene that encodes functional collagen VII can be inserted into the patient’s own fibroblast cells and administered as a therapeutic solution. Our goal is to treat the underlying genetic cause of the disease and relieve the suffering of these patients. We are working with leading EB centers to complete proof-of-concept animal work, and look forward to filing our IND in the first half of 2015 and initiating a Phase 1 trial.”

Suma Krishnan, senior vice president of product development at Intrexon, added, “We are excited to see continued progress within the Fibrocell Exclusive Channel Collaboration program. The collaborative effort between the Intrexon and Fibrocell teams has helped realize this important stage in developing a cell-based therapy for the unmet need of patients with RDEB. We look forward to the next steps in advancing the RDEB program to the clinic.”

About Fibrocell Science, Inc.

Fibrocell Science, Inc. (NYSE MKT:FCSC) is an autologous cell therapy company primarily focused on developing first-in-class treatments for rare and serious skin and connective tissue diseases with high unmet medical needs. Based on its proprietary autologous fibroblast technology, Fibrocell is pursuing breakthrough medical applications of azficel-T for restrictive burn scarring and vocal cord scarring. The company’s collaboration with Intrexon Corporation (NYSE:XON), a leader in synthetic biology, includes using genetically-modified fibroblasts for treating orphan skin diseases for which there are no currently approved products. For additional information, visit