Exton’s Fibrocell has been granted the Rare Pediatric Disease Designation by the Food and Drug Administration for its gene therapy candidate to treat children with localized scleroderma, writes Maria Verissimo for Scleroderma News.
Fibrocell is hoping that its new drug will help patients suffering from this painful disease by eliminating the excessive creation of collagen, the disorder’s underlying cause.
As a chronic autoimmune skin disorder, localized scleroderma causes skin to thicken in localized areas and can sometimes spread to underlying tissue and muscle.
Fibrocell CEO John Maslowski estimates that more than 40,000 patients nationwide have some form of linear subtype of localized scleroderma. This can result in severe pain, restricted motion, and disfigurement, as well as 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,” he said.