http://www.raredr.com/news/fcx-007-pediatric-trial
FDA Allows Fibrocell to Initiate Pediatric Enrollment in Epidermolysis Bullosa Trial

Mathew Shanley

This morning, Fibrocell Science, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted the company allowance to begin enrollment in the Phase 2 portion of its Phase 1/2 clinical trial of FCX-007.

The gene therapy candidate is in development in collaboration with Intrexon Corporation for the treatment of pediatric patients with recessive dystrophic epidermolysis bullosa (RDEB). In May 2015, it was granted Rare Pediatric Disease Designation for the patient population.

RDEB is a hereditary, progressive, excruciatingly painful and debilitating genetic disorder and the most severe variation of dystrophic epidermolysis bullosa (DEB). Today’s news is based on the evidence of safety and potential clinical benefit of FCX-007 in adult patients dosed in the Phase 1 portion of the trial.

“Pediatric RDEB patients are among the most vulnerable to this devastating blistering skin disease because of the scarring that develops in childhood and progresses into adulthood,” said Alfred Lane, MD, Chief Medical Advisor of Fibrocell and Professor of Dermatology and Pediatrics (Emeritus) at the Stanford University School of Medicine in a press release.  “I am pleased that we now have the opportunity to evaluate FCX-007 in children with the goal of improving wound outcomes for these patients.”

RDEB is the result of a deficiency of the type VII collagen (COL7) protein, and FCX-007 is a genetically-modified autologous fibroblast that encodes COL7. In the Phase 1 portion of the study, FCX-007 was well-tolerated following a single intradermal injection session in targeted wounds and separate intact skin sites in 3 adult patients 12 weeks post-administration and 1 adult patient 25 weeks post-administration.

“Receiving allowance from the FDA to include pediatric patients in the Phase 2 portion of our clinical trial of FCX-007 signifies important clinical progress, moving us closer to our goal of transforming the lives of RDEB patients,” said John Maslowski, President and CEO of Fibrocell. “We believe our distinctive approach to treatment—targeting the underlying origin of the disease—holds promise to relieve the chronic symptoms and suffering associated with RDEB.”

In addition to evaluating the safety and efficacy of FCX-007 in RDEB patients, the trial is observing wound healing and pharmacology at 4, 12, 25 and 52 weeks post-administration. The Phase 1 portion of the trial didn’t report any product-related adverse events (AEs), and the Phase 2 portion is looking to enroll 6 patients ages 7 years and older.

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