Orphan drug status granted for Cytonet’s biotech liver treatment

An investigational liver treatment from German biotechnology company Cytonet has received orphan drug status in the United States.

Cytonet, which has its U.S. headquarters in Durham, North Carolina, is currently in a phase 2 clinical trial studying its liver cell therapy in children with congenital and severe urea cycle disorders. A urea cycle disorder, or UCD, is a genetic disorder in the urea cycle, which is responsible for removing ammonia from the bloodstream. The disorder is caused by a deficiency in one of the enzymes needed in the urea cycle. The resulting buildup of ammonia in the body can cause brain damage, coma and even death.

The National Urea Cycle Disorders Foundation has no firm figures on how many children have UCD. But the group says that in April 2000, research experts at a urea cycle conference estimated the incidence of UCD was 1 in every 10,000 births.

Cytonet’s experimental treatment consists of an infusion of a liver cell preparation into the patient. The liver cell preparation comes from cells extracted from livers that are not suitable for transplant. The first American child to receive this liver therapy had the treatment performed in December at the Yale University School of Medicine, one of the clinical trial sites.

Orphan drug status granted by the U.S. Food and Drug Administration gives companies incentives to create treatments for rare diseases that have no cure. With the designation, a company that successfully develops a new orphan drug will receive a period of market exclusivity as well as tax breaks. Cytonet’s liver treatment received orphan drug status from European regulators 2006.