WEINHEIM, Germany, May 17, 2011 /PRNewswire/ -- 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.
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 5 with UCD.
For more information, please visit Cytonet's website at http://www.cytonetllc.com/
About the SELICA (Safety and Efficacy of Liver Cell Application) Study
For the past several years Cytonet has worked with internationally-leading metabolism and neonatal centers to study its liver cell treatment. This therapy uses healthy and metabolically functional human hepatocytes for infusion to treat the metabolism disorder. Healthy cells from donated livers not suitable for transplantation (obtained from U.S. organ procurement organizations) are isolated and undergo complex processing. These cells are infused into the hepatic portal vein over six days.
In July 2010, the FDA permitted clinical testing to proceed in the SELICA III trial. The FDA was provided with an analysis of interim results of an ongoing trial (SELICA V) in Germany among newborns with UCDs. Cytonet would like to acknowledge the National Urea Cycle Disorders Foundation for its assistance during the development of the program. For more information about urea cycle disorders, visit the National Urea Cycle Disorders Foundation at www.nucdf.org
About Urea Cycle Disorders
According to the National Urea Cycle Disorders Foundation (NUCDF), a urea cycle disorder is a genetic disorder caused by a deficiency of one of the six enzymes in the urea cycle which is responsible for removing ammonia from the blood stream. These include carbamoyl phosphate synthetase I (CPS I) deficiency, N-acetylglutamate synthetase (NAGS) deficiency, ornithine transcarbamylase (OTC) deficiency, argininosuccinate synthetase (ASS) deficiency (which is also known as citrullinemia), argininosuccinate lyase (ASL) deficiency and arginase 1 deficiency (hyperargininemia). The urea cycle involves a series of biochemical steps in which nitrogen, a waste product of protein metabolism, is removed from the blood and converted to urea. Normally, the urea is transferred into the urine and removed from the body. In urea cycle disorders, the nitrogen accumulates in the form of ammonia, a highly toxic substance, and is not removed from the body resulting in hyperammonemia (elevated blood ammonia). Ammonia then reaches the brain through the blood, where it causes irreversible brain damage, coma and/or death.
Urea cycle disorders are included in the category of inborn errors of metabolism. Inborn errors of metabolism represent a substantial cause of brain damage and death among newborns and infants. Because many cases of urea cycle disorders remain undiagnosed and/or infants born with the disorders die without a definitive diagnosis, the exact incidence of these cases is unknown and underestimated. It is believed that up to 20 percent of Sudden Infant Death Syndrome cases may be attributed to an undiagnosed inborn error of metabolism such a urea cycle disorder. Experts estimate the incidence of the disorders at 1 in 10,000 births. This represents a significant increase in case identification and diagnosis in the last few years. Research studies have now been initiated to more accurately determine the incidence and prevalence of UCDs. For more information, please visit NUCDF at www.nucdf.org
The Cytonet Group is an internationally active biotechnology company which is located in Weinheim and Heidelberg in Germany and in Durham, North Carolina in the U.S. The company develops, produces and markets cell therapeutic products. By using specially prepared human cells, it is possible to provide alternatives to existing therapies for many diseases. In addition, Cytonet provides blood stem cells and bone marrow preparations for the treatment of leukemia and other tumor diseases. The managing directors are Dr. Wolfgang Rudinger and Michael J. Deissner. Cytonet was founded as a result of the demerger of the cell therapy division from the firm of Roche in April 2000. The controlling interest is held by the family of Dietmar Hopp. The project is supported by the German Federal Ministry for Education and Research (BMBF) as part of the Leading Edge Cluster Competition (Spitzencluster Wettbewerb). Cytonet is an active partner in the Biotech-Cluster-Rhein-Neckar (BioRN) - the Rhine Neckar biotechnology cluster, which was designated in 2008 as the leading edge cluster in "cell-based and molecular medicine."
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