Rare Disease Report

Fibrocell Submits IND Application for Scleroderma Treatment

FEBRUARY 05, 2018
Mathew Shanley
This morning, Fibrocell Science, Inc. announced the submission of an Investigational New Drug (IND) Application with the U.S. Food and Drug Administration (FDA) for FCX-013, a gene therapy candidate for the treatment of moderate to severe localized scleroderma.

In June, the company was granted a Rare Pediatric Disease designation for the potential therapy.

Localized scleroderma, a chronic autoimmune disease, is characterized by a thickening of the skin as the result of excessive collagen deposits. Moderate to severe forms of the condition, including the linear subtype, can lead to significant morbidity, including pain, hindered motion, disfigurement, and developmental concerns.

FCX-013 is in development to express matrix metalloproteinase 1 (MMP-1), the protein responsible for breaking down superfluous collagen.

“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,” said John Maslowski, President and Chief Executive Officer of Fibrocell 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.”

The drug is being developed in conjunction with Intrexon Corporation and uses its proprietary RheoSwitch Therapeutic System, a biologic switch activated by an orally administered compound to control protein expression once the initial fibrosis has been resolved.

FCX-013 is intended to be injected under the skin at the location of the fibrotic lesions where the genetically-modified fibroblast cells will produce MMP-1. With the therapy, patients will be permitted to also take an oral compound to facilitate protein expression, and once fibrosis is resolved, the patient will cease taking the oral compound which will halt further MMP-1 production.

Current standard-of-care for localized scleroderma only operates for an alleviation of symptoms, and includes systemic or topical corticosteroids, UVA light therapy and physical therapy. Per the Scleroderma Foundation, an estimated 100,000 Americans have systemic form of scleroderma.

In addition to the Rare Pediatric Disease designation, the FDA has also previously granted FCX-013 an orphan drug designation.

Because of the Rare Pediatric Disease designation, if the drug is approved, the company would receive a priority review voucher that could be worth millions of dollars. Last year, Regeneron purchased a priority review voucher from BioMarin for $67.5 million.

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