NASH is believed to affect 2% to 5% of the U.S. population. While commonly diagnosed in overweight adults and children with and without insulin-resistant diabetes and abnormal serum lipid profiles, NASH can also occur in persons of average weight. Though most people with NASH feel healthy and show no outward signs of liver disease, NASH causes liver scarring and potentially leads to cirrhosis and liver failure. In more severe cases, the progressive nature of NASH may require liver transplantation in affected patients.
Ranjan Dohil, Professor of Pediatrics at UCSD, commented, "The opportunity for DR Cysteamine in NASH could become a potentially significant breakthrough for these patients. Our collaboration with Raptor allows us to run this clinical trial by leveraging the University's resources and scientific capabilities with Raptor's instrumental support in funding, providing drug supply, and offering formulation expertise."
About Cysteamine and DR Cysteamine
Cysteamine is approved for sale by the FDA and European Medicines Agency to treat nephropathic cystinosis, a rare, genetic lysosomal storage disease. DR Cysteamine is designed as an improved, enterically coated, oral formulation of Cysteamine. Compared to the currently marketed formulation, DR Cysteamine could allow less frequent dosing, improving compliance and potentially reducing gastrointestinal side effects, such as nausea and vomiting.
Raptor obtained an exclusive, worldwide license to DR Cysteamine, as
well as orphan drug designation from the FDA for DR Cysteamine for the
treatment of nephropathic cystinosis, through its December 2007
|SOURCE Raptor Pharmaceuticals Corp.|
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