Potential Efficacy in Huntington's Disease Already Demonstrated
NOVATO, Calif., June 9 /PRNewswire-FirstCall/ -- Raptor Pharmaceuticals Corp. ("Raptor" or the "Company") (OTC Bulletin Board: RPTP), today announced that the U.S. Food and Drug Administration ("FDA") has granted orphan drug designation for cysteamine bitartrate ("cysteamine") for the treatment of Huntington's disease ("HD"). Cysteamine is currently approved by the FDA and European Medicines Agency ("EMEA") to treat nephropathic cystinosis ("cystinosis"), a rare lysosomal storage disease. Preclinical results suggest that cysteamine has neuroprotective effects that could potentially help treat HD. Raptor's clinical development subsidiary, Bennu Pharmaceuticals Inc. ("Bennu"), plans to evaluate cysteamine in patients with HD.
HD is a rare and hereditary neurological disease thought to occur in approximately 1 out of every 20,000 people resulting in around 20,000 patients in the U.S. presently. There is currently no drug available that targets the unique molecular defect that is believed to cause HD, a progressive disease that often leads to death within 15 to 20 years after diagnosis. HD is caused by a defective gene resulting in the degeneration of certain nerve cells in the brain. The disease is characterized by uncontrollable movements and mood swings or depression, followed by dementia. Preclinical findings on cysteamine's mechanism of action suggest that it has neuroprotective effects by increasing the levels of BDNF, a neuroprotective neurotrophic factor, in the brain of HD mice. BDNF levels may also be a potential biomarker of efficacy for cysteamine in HD, an important development issue when a therapy is used to prevent or slow clinical manifestation of a disorder.
The 1983 Orphan Drug Act provides important economic incentives to
encourage companies to develop potential therapies for the diagnosis,
prevention and/or treatment of rare, serious diseases affecting 200,0
|SOURCE Raptor Pharmaceuticals Corp.|
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