FDA Grants Systemic Sclerosis Drug Orphan Designation

The FDA has granted Orphan Drug Designation to iBio-CFB03, an investigational biotherapeutic product intended to treat systemic sclerosis.

Systemic sclerosis is categorized as a fibrotic disorder that impacts the connective tissue of the skin and internal organs, as well as the walls of blood vessels. No approved drug has been proven to address the processes that drive the progression of the illness.

iBio-CFB03, manufactured by iBio, Inc., is an anti-fibrotic agent based on a plant-made protein. At the moment, iBio is working to increase the production of iBio-CFB03 for clinical trials.

"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," said Robert B. Kay, executive chairman of iBio. "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."

In 2014 the FDA approved pirfenidone (Esbriet) and nintedanib (Ofev) for the treatment of idiopathic pulmonary fibrosis (IPF), a progressive and fatal lung disease that makes breathing difficult and causes irreversible lung damage.

Orphan Drug Designation is given to drugs that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, specifically those that impact less than 200,000 people in the United States.