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NOVATO, Calif., Dec. 21, 2012 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), today announced that the U.S. Food and Drug Administration ("FDA") will require additional time to complete its review of the New Drug Application ("NDA") for RP103 (PROCYSBI™), for the potential treatment of nephropathic cystinosis. In a notice received today from the FDA, the initial Prescription Drug User Fee Act ("PDUFA") goal date has been extended from January 30, 2013 to April 30, 2013. The three month extension is a standard extension period.
The FDA exercised its option to extend the PDUFA goal date to provide more time to complete the review of information submitted within three months of the PDUFA date. The agency has not asked for additional studies.
About Cystinosis and RP103
Cystinosis is a rare, life-threatening metabolic disorder that causes systemic toxic cystine accumulation. Toxic cystine accumulation leads to progressive and irreversible tissue damage and multi-organ failure including renal failure, blindness, CNS damage, respiratory deficiencies, muscle wasting, and premature death. Cystinosis is usually diagnosed in the first years of life and requires lifelong therapy. Left untreated, the disease is fatal by the end of the first decade of life. Daily cystine depletion is the primary treatment strategy for cystinosis. Despite a life-long requirement for cystine-depleting agent use to help preserve multi-organ function, a cumulative inability to maintain adequate cystine control results in the rapid deterioration of kidney function and ensuing need for kidney transplantation.
RP103 is an oral, delayed and extended-release medication containing enteric-coated spheronized micro-beads of cysteamine bitartrate. PROCYSBI™ is the branded name of RP103 for the potential treatment of cystinosis. The NDA and Marketing Authorization Application for RP103 for the potential treatment of nephropathic cystinosis have been submitted to the FDA and the European Medicines Agency, respectively, and the NDA has been assigned a PDUFA date of April 30, 2013. RP103 is also in clinical development for the potential treatment of Huntington's disease and non-alcoholic steatohepatitis ("NASH"). In cystinosis patients, RP103 may reduce cellular toxicity by continuously removing cystine from the lysosome. RP103, which can also cross the blood-brain barrier, was engineered specifically to allow release of cysteamine bitartrate micro-spheres in the duodenum for optimal absorption while simultaneously enabling administration every 12-hours for the potential treatment of cystinosis.