FDA Gives Greenlight to Fibrocell to Begin Enrollment of Pediatric Patients in Trial of Experimental Gene Therapy for Rare Skin Disease

Rare Daily Staff

The U.S. Food and Drug Administration gave approval to Fibrocell Science to begin enrolling pediatric patients in a clinical trial of FCX-007, its experimental gene therapy developed in collaboration with Intrexon for the treatment of recessive dystrophic epidermolysis bullosa, a rare, genetic skin disease that causes blistering and carries a high rate of mortality.

FCX-007 is Fibrocell’s experimental gene therapy for the treatment of RDEB, which is caused by the deficiency of the protein type VII collagen (COL7). FCX-007 is a genetically-modified autologous fibroblast that encodes the gene for COL7. By genetically modifying autologous fibroblasts ex vivo to produce COL7, culturing them and then treating wounds locally via injection, Fibrocell said that FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas while avoiding systemic distribution.

The FDA allowance to begin enrollment of pediatric patients in the phase 2 portion of the company’s phase 1/2 clinical trial is based on evidence of safety and potential benefit of FCX-007 in adult patients dosed in the phase 1 portion of the clinical 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, chief medical advisor of Fibrocell and professor of Dermatology and Pediatrics (Emeritus) at the Stanford University School of Medicine. 

In its submission, Fibrocell reported the interim readout of safety data from three adult patients 12 weeks post-administration and one adult patient 25 weeks post-administration, which showed FCX-007 was well-tolerated following a single intradermal injection session in targeted wounds and separate intact skin sites. 

No serious adverse events and no product-related adverse events were reported.  In addition to encouraging interim safety data, positive early trends were noted in wound healing and pharmacology signals to support potential benefit of the product candidate.

Fibrocell plans to enroll six patients ages seven and older in the phase 2 portion of the clinical trial. One RDEB adult patient has been enrolled in Phase 2, and dosing is expected to occur in the second quarter of 2018.

The primary objective of this open-label clinical trial is to evaluate the safety of FCX-007 in RDEB patients. Additionally, the trial is assessing wound healing and pharmacology at 4, 12, 25 and 52 weeks post-administration. 

“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,” said John Maslowski, president and CEO of Fibrocell.

The FDA has granted FCX-007 Orphan Drug, Rare Pediatric Disease and Fast Track Designations by the FDA.

February 1, 2018
Photo: John Maslowski, president and CEO of Fibrocell