Cytonet’s liver cell therapy granted orphan drug designation
International biotechnology firm Cytonet’s investigative liver cell infusion of donated human liver cells for the treatment of urea cycle disorders (UCD) in young children has been granted orphan drug designation by the 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 SELICA III multicenter clinical trial is designed to evaluate the safety and efficacy of liver cell therapy in infants to children up to age five with UCD.