Rare Disease Report

Gene Therapy for Epidermolysis Bullosa Gets Rare Pediatric Designation

MAY 12, 2015
James Radke, PhD

Fibrocell Science can hopefully add a priority review voucher to their portfolio following the announcement that the FDA has granted their orphan designated drug FCX-007 a rare pediatric disease designation.

FCX-007 is being investigated as a treatment for patients with recessive dystrophic epidermolysis bullosa (RDEB). The drug is currently in late stage pre-clinical development with an IND filing targeted for mid-2015.

Vouchers Can Fetch $67-$125 Million

If the drug gets approved, the company would be given a priority review voucher that could be worth millions of dollars. Last year, Regeneron purchased a priority review voucher from Biomarin for $67.5 million and Gilead purchased one from Knight Therapeutics for $125 million.

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 the 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 can also be sold or transferred an unlimited number of times.

"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," said David Pernock, Chairman and Chief Executive Officer of Fibrocell. "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.

FCX-007 and RDEB

FCX-007 is Fibrocell's first-in-class gene-therapy drug candidate for the treatment of RDEB, a congenital and progressive orphan skin disease caused by the deficiency of the protein type VII collagen (COL7).  FCX-007 is a gene-modified autologous fibroblast that encodes COL7 and is being developed in collaboration with Intrexon Corporation.

RDEB is a severe form of epidermolysis bullosa (EB) a congenital, progressive, devastatingly painful and debilitating genetic disorder that leads to death. There are approximately 1,100 – 2,500 people with RDEB in the United States. There are many genetic and symptomatic variations of EB with RDEB being one of the more serious forms of condition. All forms of EB however, share the common symptom of fragile skin that blisters and tears from the slightest friction or trauma. Current standard of care consists of bandaging and bathing the open wounds to prevent infection and trying to manage patients’ pain.


Fibrocell Receives Rare Pediatric Disease Designation From FDA for FCX-007 for the Treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB) [press release]. Exton, PA: Fibrocell Science; May 12, 2015. http://ir.fibrocellscience.com/phoenix.zhtml?c=253777&p=irol-newsArticle&ID=2047210

Image "Tratamiento epidermolisis bullosa" by Yovanna.Gonzalez - Own work. Licensed under GFDL via Wikimedia Commons - https://commons.wikimedia.org/wiki/File:Tratamiento_epidermolisis_bullosa.jpg#/media/File:Tratamiento_epidermolisis_bullosa.jpg

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