Gene Therapy for Localized Scleroderma Gets FDA’s Fast Track Designation

Fibrocell Science announced that the Food and Drug Administration (FDA) has granted Fast Track Designation to FCX-013 for the treatment of moderate to severe localized scleroderma. 

FCX-013, an autologous fibroblast genetically modified using lentivirus and encoded for matrix metalloproteinase 1 (MMP-1), utilizes the proprietary RheoSwitch Therapeutic System, a biologic switch that controls protein expression at the site of localized scleroderma lesions. The treatment is intended for injection under the skin at the location of the lesions where the genetically-modified fibroblast cells will produce MMP-1 to break down excess collagen accumulation. 

Clinical enrollment for an open-label, single-arm Phase 1/2 trial of FCX-013 was initiated in August 2018; the primary objective of the trial will be to investigate the safety of the treatment. The Phase 1 portion of the trial will evaluate FCX-013 in adult patients; the Company plans on including pediatric patients in the Phase 2 portion.

“We are pleased the FDA has awarded this designation to FCX-013 which, we believe, has the potential to be the first gene therapy to treat excessive collagen accumulation in the skin and soft tissue at the site of localized scleroderma lesions and to bring relief from the severe pain and functional disability associated with the disorder,” said John Maslowski, President and Chief Executive Officer of Fibrocell.

For more information visit Fibrocell.com.