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Raptor Pharmaceutical Corp. Announces Presentation Of PROCYSBI(TM) (RP103) Extension Study Data At The ASN Annual Meeting

NOVATO, Calif., Oct. 17, 2012 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. ("Raptor" or the "Company") (Nasdaq:RPTP), announced that Craig B. Langman, M.D., Head of Kidney Diseases, and the Isaac A. Abt, M.D., Professor of Kidney Diseases and Tenured Professor of Pediatrics, Northwestern University Feinberg School of Medicine, will present a Late Breaking Poster session titled "Extended Treatment with RP103 (PROCYSBI™) in Patients with Nephropathic Cystinosis" at Kidney Week 2012, the world's premier nephrology meeting, which is being held October 30-November 4 th at the San Diego Convention Center in San Diego, California. Dr. Langman's presentation is on Saturday, November 3 rd in a session beginning at 10:00 A.M. PT.

About Nephropathic Cystinosis

Nephropathic Cystinosis is a rare, life-threatening metabolic disorder that causes systemic toxic cystine accumulation. Toxic cystine accumulation causes progressive and irreversible tissue damage and organ failure including renal failure, blindness, CNS toxicities, respiratory deficiencies and muscle wasting. 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. 

About Cysteamine and PROCYSBI™ (RP103)

PROCYSBI™ is Raptor's delayed-release oral medication currently in clinical development for several indications. PROCYSBI™ is an enteric-coated, delayed and extended-release formulation of cysteamine bitartrate. PROCYSBI™ was engineered specifically to allow release of micro-spheronized enteric-coated cysteamine bitartrate in the duodenum for optimal absorption while simultaneously enabling administration every 12-hours.

In December 2007, Raptor obtained an exclusive, worldwide license from the University of California, San Diego to intellectual property related to the development of PROCYSBI™ and other forms of cysteamine for the potential treatment of non-alcoholic steatohepatitis ("NASH"), currently in a Phase 2b clinical trial in the U.S., Huntington's Disease, currently in the Phase 2/3 clinical trial in France, and for the development of PROCYSBI™ for nephropathic cystinosis, which Raptor has recently filed for marketing approval in the U.S. and E.U. The U.S. Food and Drug Administration ("FDA") has accepted for filing Raptor's New Drug Application ("NDA") for PROCYSBI™ for the potential treatment of nephropathic cystinosis and assigned the user fee goal date of January 30, 2013.  Raptor's E.U. marketing application of PROCYSBI™ for the potential treatment of nephropathic cystinosis is under review by the EMA, and Raptor expects a decision in the first half of calendar 2013. Raptor has been granted orphan product designation for PROCYSBI™ for the potential treatment for nephropathic cystinosis by the European Medicines Agency and FDA and for the potential treatment of Huntington's Disease by the FDA. Raptor's intellectual property portfolio also includes patents covering the use of transglutaminase inhibitors, a class of molecules chemically similar to cysteamine, in the potential treatment of Huntington's Disease and other neurological disorders from the Weizmann Institute of Science in Israel and Niigata University in Japan; the use of cysteamine and related compounds in the potential treatment of parasitic diseases, including malaria, from McGill University in Montreal, Canada; the use of cysteamine and related compounds in the potential treatment of Parkinson's Disease from Université Laval, Quebec, Canada; and the use of cysteamine and related compounds in the potential treatment of tissue fibrosis from the Seattle Children's Research Institute.

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