Raptor Pharmaceutical Corp., a biopharmaceutical company, focuses on developing and commercializing life-altering therapeutics that treat debilitating and often fatal diseases. Products The company’s first product, PROCYSBI (cysteamine bitartrate) delayed-release capsules received marketing approval from the U.S. Food and Drug Administration (FDA) in April 2013 for the management of nephropathic cystinosis in adults and children six years and older. In September 2013, the European equivalent, PROCYSBI gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate), received a Community or EU marketing authorization from the European Commission (EC), as an orphan medicinal product for the management of proven nephropathic cystinosis. The company commenced commercial sales of PROCYSBI in the U.S. in June 2013. PROCYSBI is an approved therapy for the management of nephropathic cystinosis, a rare, life-threatening metabolic lysosomal storage disorder that causes the rapid, toxic accumulation of cystine in all cells, tissues and organs in the body. PROCYSBI capsules contain cysteamine bitartrate in the form of microspheronized beads that are individually coated to create delayed and extended-release properties, allowing patients to maintain consistent therapeutic systemic drug levels over a 12-hour dosing period. Randomized controlled clinical trials and extended treatment with PROCYSBI therapy demonstrated consistent cystine depletion as monitored by levels of the biomarker (and surrogate marker), white blood cell cystine. RP103 as a treatment for Huntington's disease (HD) RP103, enteric-coated delayed-release cysteamine bitartrate, is being evaluated as a potentially disease modifying treatment for HD. Other Clinical-Stage Product Candidates Convivia for aldehyde dehydrogenase Deficiency: The company is in the process of developing Convivia, its proprietary oral formulation of 4-methylpyrazole, or 4-MP, for the potential treatment of acetaldehyde toxicity resulting from aldehyde dehydrogenase deficiency. The company owns the intellectual property portfolio pertaining to Convivia, including method of use and formulation patents. In 2010, the company granted a license to commercialize Convivia in Taiwan to Uni Pharma Co., Ltd. Under this agreement, Uni Pharma Co., Ltd. is responsible for clinical development, registration and commercialization of Convivia in Taiwan. Preclinical Product Candidates The company’s preclinical programs include its cysteamine dioxygenase, or ADO, program, to improve treatment of diseases for which cysteamine is therapeutic and its HepTide program to treat hepatocellular carcinoma and other cancers susceptible to induced lysosomal storage. Trademarks The trademark ‘Raptor’ is registered in the U.S. and the EU, and applied for in certain other countries. The company also owns applications and registrations for various other marks in the U.S. and certain countries throughout the world. Research and Development As of December 31, 2013, the company incurred approximately $29.2 million on research and development expenses. History Raptor Pharmaceutical Corp. was founded in 2006. The company was incorporated under the laws of the state of Delaware.
raptor pharmaceutical corp
(RPTP:Consolidated Issue listed on NASDAQ Global Market)
5 Hamilton Landing
Novato, CA 94949
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