The FDA has granted rare pediatric disease designation to FCX-007 for the treatment of recessive dystrophic epidermolysis bullosa, according to the drug’s manufacturer.
A congenital genetic disorder caused by the deficiency of the protein type VII collagen, recessive dystrophic epidermolysis bullosa (RDEB) weakens the adherence between the epidermis and the underlying dermis causing severe blistering, open wounds and scarring in response to any kind of friction, including ordinary daily activities such as rubbing or scratching.
Children who inherit the condition are often called “butterfly children” as their skin is seen to be as delicate and fragile as that of a butterfly. Currently, there is no cure for the approximately 2,500 pediatric patients with RDEB in the US, with treatments including daily bandaging, hydrogel dressings, antibiotics, feeding tubes and surgeries.
FCX-007 (Fibrocell Science), a gene-therapy drug candidate for the treatment of RDEB, may help address the underlying cause of the disease by genetically modifying autologous fibroblasts to produce and culture type VII collagen. Delivered via injection, FCX-007 could potentially address the root cause of RDEB by providing high levels of type VII collagen directly to the affected areas, thus bypassing the need for systematic treatment.
“We are pleased that the FDA has granted our request to designate FCX-007 for the treatment of RDEB as a drug for a rare pediatric disease,” David Pernock, chairman and CEO of Fibrocell, said in a press release. “FCX-007 may offer RDEB patients and their families the first therapy to treat the underlying cause of the disease, bringing hope and relief to what is today a painful, disabling and often fatal congenital disorder.”
The FDA designation was based on results from ongoing late stage pre-clinical development, with an investigational new drug filing targeted for mid-2015.