Cytonet receives FDA orphan drug designation for liver cell infusion to treat urea cycle disorders

May 18 2011

International biotechnology firm Cytonet, which is investigating a liver cell infusion of donated human liver cells for the treatment of urea cycle disorders (UCD) in young children, announced it has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA).

"This designation recognizes our work to advance the landscape of UCD treatment for young children and offer a much-needed new option for a very serious condition where the only current cure is liver transplantation," says Dr. Wolfgang Rudinger, CEO and CSO of Cytonet Germany. "Orphan drug designation enhances our ability to execute the SELICA III trials on this novel treatment."

The Orphan Drug Act was enacted to allow the FDA to grant special orphan status to treatments for rare diseases or conditions – those that affect fewer than 200,000 people in the United States. It is intended to facilitate drug development and provides potential benefits including funding for certain clinical studies, study design assistance, tax incentives and seven years of market exclusivity for the product upon regulatory approval.