The rare skin blistering disorder is caused by defects in a gene that produces type VII collagen protein. COL7 protein is a major component of anchoring fibrils, which are responsible for binding layers of skin together.
How FCX-007 works
FCX-007 consists of dermal fibroblasts, or skin cells that produce extracellular matrix proteins, including collagen. Fibrocell collects fibroblasts from a patient, then modifies them. The alteration involves using a healthy gene to supersede the defective one that produces faulty COL7 protein. Thus, each patient is treated with their own cells.
Doctors inject FCX-007 directly into blisters and wounds. The correct version of the COL7 protein helps create anchoring fibrils that are able to hold layers of skin together. The way that FCX-007 works means that it can address the underlying cause of RDEB by delivering high levels of COL7 directly to blistered areas. This avoids systemic treatments that can affect the body beyond the blistered regions.
Since doctors administer the modified fibroblasts locally, FCX-007 avoids side effects associated with systemic therapy. And because the fibroblasts are compatible with the patients’ body, they are not rejected and do not cause immune reactions.
FCX-007 in clinical trials
Fibrocell has started a Phase 1/2 clinical trial (NCT02810951) that is recruiting RDEB patients. The main aim of the open-label trial is to evaluate the safety of FCX-007 injections. It will also assess the therapy’s ability to generate COL7 protein and promote the growth of anchoring fibrils. Researchers will measure effectiveness by assessing how FCX-007 heals wounds.
Initially, the Phase 1 portion of the trial will involve six adults ages 18 or older. Additional patients are expected to be dosed after a four-week waiting period and health assessment.
Fibrocell expects to have results of the Phase 1 component of the trial 12 weeks after patients are treated. The findings will cover its safety, mechanism of action, and effectiveness. Researchers will evaluate the treatment’s ability to heal wounds at four, 12, 25, and 52 weeks.
In the Phase 2 portion of the trial, the research team will administer FCX-007 to six people ages 7 or older. The therapy will be delivered to one or more of each patient’s wounds. Researchers will use photographs and biopsies to monitor healing.
Before Fibrocell can conduct clinical trials with children, it must obtain U. S. Food and Drug Administration approval. It will do this by submitting evidence of FCX-007’s activity in adults, and results from a preclinical-trial toxicology study.
In January 2017, the FDA granted FCX-007 fast track designation as a treatment for dystrophic epidermolysis bullosa, which includes RDEB. The agency had previously granted the therapy orphan drug designation and rare pediatric disease designation as a treatment for RDEB.
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