Fibrocell’s scleroderma gene therapy has been granted Rare Pediatric Disease Designation by the Food and Drug Administration (FDA). The company will thus be able to expedite the review process for future Biologics License Applications (BLA) submissions. This is due to the Rare Pediatric Disease Priority Review Voucher program that is part of the designation.
Sclerodema is a chronic autoimmune disease that is characterized by the excess production of extracellular matrix. This can impair growth, development, and movement as well as result in severe joint pain from lesions. Fibrocell’s gene candidate for this condition, FCX-013, comprises of genetically modified fibroblast cells that are injected at fibrosis sites. FCX-013 has a biologic switch incorporated within it (Intrexon’s proprietary RheoSwitch Therapeutic System® ), this is activated by an orally administered compound which initiates the expression of a collagen that breaks down excess collagen accumulation. Halting the oral compound stops protein production once the fibrosis has been sufficiently treated.
“We are pleased the FDA has awarded Rare Pediatric Disease Designation to FCX-013, which in addition to its Orphan Drug Designation provides important incentives to Fibrocell for developing therapies for rare pediatric diseases,” commented CEO John Maslowski .
“Moderate to severe forms of localized scleroderma, including the linear subtype, can result in significant morbidity, including pain, restricted motion, disfigurement and developmental issues. 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.”
Source: Fibrocell Receives Rare Pediatric Disease Designation from FDA for FCX-013 for Treatment of Localized Scleroderma. Press Release