Rare Disease Report

Gene Therapy for Localized Scleroderma Gets Rare Pediatric Designation

JUNE 12, 2017
James Radke
Fibrocell Science is on a roll. 
One month after receiving a Rare Pediatric Disease Designation for its gene therapy, FCX-007 to treat epidermolysis bullosa, the U.S. Food and Drug Administration (FDA) has granted a Rare Pediatric Disease Designation for FCX-013 to treat localized scleroderma.
Localized scleroderma is a chronic autoimmune disease characterized by thickening of the skin from excessive collagen deposits.
FCX-013 is a gene-therapy drug candidate in which gene-modified autologous fibroblasts that encode a gene to help breakdown excess collagen. The treatment is being developed in collaboration with Intrexon Corporation.
Details of the treatment are limited, but Fibrocell plans to inject FCX-013 at the location of the fibrosis where the genetically-modified fibroblast cells will produce a protein to break down excess collagen accumulation. The patient will also take an oral compound to facilitate protein expression from FCX-013. Once the fibrosis is resolved, the patient can stop taking the pill and thereby stop further production of the enzyme.
“Moderate to severe forms of localized scleroderma, including the linear subtype, can result in significant morbidity, including pain, restricted motion, disfigurement and developmental issues,” said John Maslowski, Chief Executive Officer of Fibrocell. “With no FDA-approved therapies available, we believe controlled gene therapy through FCX-013 offers promise to address this high unmet medical need of patients suffering from this chronic and often debilitating disease.”
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 and Gilead purchased one from Knight Therapeutics for for $67.5 and $125 million, respectively.

According to 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 application sponsor 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.
At present, FCS-013 is still in preclinical development.

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