Cystine depletion is the primary treatment strategy for nephropathic cystinosis. However, poor adherence to therapy has been a major challenge resulting in poor sustained control of cystine levels, and patients consequently experience poor clinical outcomes, including kidney insufficiency leading to dialysis and kidney transplantation, muscle wasting and in some cases, premature death. Even brief interruptions in daily therapy can permit toxic accumulation of cystine, exposing tissues to renewed, progressive deterioration.
About Raptor Pharmaceutical
Raptor Pharmaceutical Corp. is a biopharmaceutical company focused on developing and commercializing life-altering therapeutics that treat rare, debilitating and often fatal diseases. The company's first product, PROCYSBI™ (cysteamine bitartrate) delayed-release capsules, has been approved by the FDA, and recommended for marketing authorization by the European Committee for Medicinal Products for Human Use (CHMP) for the management of nephropathic cystinosis, a rare metabolic lysosomal storage disease. Raptor's pipeline also includes RP103 in a Phase 2/3 trial for Huntington's disease and a Phase 2 trial in nonalcoholic fatty liver disease in children. PROCYSBI has received orphan drug exclusivity for nephropathic cystinosis in the U.S. RP103 has received U.S. orphan drug designation for Huntington's disease. For additional information, please visit www.raptorpharma.com .
Forward Looking Statements This document contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements relate to future events or our future results of operations or future financial performance, including, but not limited to statements regarding anticipated EU approval of PROCYSBI and the timing of that anticipated approval. These statements are only predictions and involve known and unknown risks, uncertainties and other factors, which may cause the Company's actual results to be materially different from these forward-looking statements. Factors which may significantly change or prevent the Company's forward looking statements from fruition are described in greater detail in the Company's filings from time to time with the Securities and Exchange Commission (the "SEC"), which Raptor strongly urges you to read and consider, including: Raptor's transition report for the four months ended December 31, 2012 on Form 10-KT filed with the SEC on March 14, 2013, as amended by the Form 10-KT/A filed with the SEC on June 19, 2013,and Raptor's Quarterly Report on Form 10-Q filed with the SEC on May 8, 2013, as amended by the Form 10-Q/A filed with the SEC on June 19, 2013, which are available free of charge on the SEC's website at www.sec.gov . Subsequent written and oral forward-looking statements attributable to Raptor or to persons acting on its behalf are expressly qualified in their entirety by the cautionary statements set forth in Raptor's reports filed with the SEC. Raptor expressly disclaims any intent or obligation to update any forward-looking statements.
CONTACT: Georgia Erbez Chief Financial Officer Raptor Pharmaceutical Corp. (415) 408-6231 email@example.com INVESTOR CONTACTS: Westwicke Partners, LLC Stefan Loren, Ph.D. Managing Director (443) 213-0507 firstname.lastname@example.org Robert H. Uhl Managing Director (858) 356-5932 email@example.com MEDIA CONTACT: Carolyn Hawley Canale Communications (619) 849-5375 firstname.lastname@example.org
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