Fibrocell Science has filed an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA) for FCX-013, its gene therapy candidate for moderate to severe localized scleroderma.
An IND is a request for FDA authorization to administer an investigational therapy to humans. It is generally the result of a successful preclinical development program and is the vehicle through which a sponsor advances an investigational therapy to clinical trials.
Scleroderma results from the excess production of collagen, the body’s main connective-tissue protein. This leads to the thickening of the skin, or fibrosis. FCX-013 is an autologous fibroblast that has been genetically engineered to express a protein called matrix metalloproteinase 1 (MMP-1), which is responsible for breaking down collagen. Fibroblast cells are the most common cells of connective tissue and are responsible for the production of collagen.
With FCX-013, fibroblast cells are collected from a patient, cultured in a lab, and genetically modified to carry the MMP-1 gene. The treatment is then injected under the skin at the site of fibrotic lesions. There, the fibroblasts begin to produce MMP-1, which ultimately destroys the excess collagen and reduces fibrosis.
FCX-013 gene therapy uses Intrexon’s proprietary RheoSwitch Therapeutic System, which acts as a biological switch to turn on the expression of MMP-1. It works by having patients take an oral compound that switches on the expression of MMP-1. Once fibrosis is resolved, the patient stops taking the oral compound, thereby turning off the expression of the protein.
“We are pleased to announce the submission of the IND as it represents a significant milestone in advancing development of FCX-013 for the treatment of moderate to severe localized scleroderma,” John Maslowski, Fibrocell’s president and chief executive officer, said in a press release.
“Adequate treatment options do not exist for this vulnerable patient population. We are committed to developing our novel gene therapy candidate to address this important unmet medical need of patients suffering from this chronic, painful and debilitating disorder,” he added.
FCX-013 is currently in preclinical development. The FDA granted the gene therapy orphan drug designation for the treatment of localized scleroderma in 2016. The following year, the agency granted FCX-013 rare pediatric disease designation for the treatment of moderate to severe localized scleroderma in children.
The FDA’s rare pediatric disease designation provides incentives to support pharmaceutical companies that are developing therapies and biologics for rare diseases. The federal agency defines a rare pediatric disease as a serious/life-threatening disease that primarily affects individuals younger than 18 and that impacts fewer than 200,000 individuals in the U.S.
We are sorry that this post was not useful for you!
Let us improve this post!
Tell us how we can improve this post?