FDA Grants Orphan Drug Status to iBio’s Scleroderma Product Candidate

iBio recently announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to iBio-CFB03 for the treatment of systemic sclerosis.

iBio is a leading provider of plant-based biotechnology for developing and manufacturing biological products for a range of fibrotic diseases (where is scar tissue has formed), including idiopathic pulmonary fibrosis (IPF) and systemic sclerosis.

Systemic sclerosis, or scleroderma, is a fibrotic disease that affects the connective tissue in the skin, internal organs and walls of blood vessels. Treatment is currently limited to symptom management, but iBio-CFB03 could make a difference.

Pre-clinical data for the drug candidate, published in 2012 by Dr. Carol Feghali-Bostwick, indicated that specific endostatin-derived peptides are useful for both inhibition and reversal of fibrosis in mouse models of the disease, as well as in human skin.

iBio-CFB03 is a recombinant, plant-made protein that was designed as an anti-fibrotic agent. Its development is based on the involvement of endostatin in fibrotic disease, specifically on endostatin’s anti-fibrotic activity.

“This is an important step toward our goal for a family of iBio proprietary products against fibrotic diseases like systemic sclerosis and idiopathic pulmonary fibrosis,” Robert B. Kay, iBio’s executive chairman, said in a press release.

“We expect to apply for additional orphan drug designations for products against additional fibrotic diseases. Viewed together and across the major geographic markets, effective therapies against fibrotic diseases are among the most significant unmet medical needs in the world,” Kay said.

The investigational biotherapeutic product was developed using the company’s gene expression technology. iBio-CFB03 was designed based on Feghali-Bostwick’s research. iBio licensed exclusive global rights to her intellectual property covering not only iBio-CFB03 but also all related products, and established a research agreement with the Medical University of South Carolina, where Feghali-Bostwick is now the Kitty Trask Holt and SmartState SC Centers of Economic Excellence Endowed Professor.

The FDA’s Office of Orphan Products Development explains that the designation qualifies the drug for benefits specified in the Orphan Drug Act during its development. FDA guidelines require that orphan drugs must meet the same standard regulatory requirements as other drug candidates, such as establishing safety and efficacy through well-controlled clinical studies.

Recently, iBio obtained a U.S. patent protecting the company’s drug development attempts using endostatin-related peptides, including iBio-CFB03, for the treatment of fibrotic diseases.