NOVATO, Calif., March 21, 2016 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. (Nasdaq:RPTP) today announced that Health Canada has accepted for review its New Drug Submission (NDS) for PROCYSBI® (cysteamine bitartrate) delayed-release capsules for the treatment of nephropathic cystinosis (NC), with Priority Review status. Priority Review provides for a shortened review process of 180 days compared to a standard 300 days. Raptor estimates, based on information provided by physicians, that there are approximately 100 individuals affected by nephropathic cystinosis in Canada. PROCYSBI, a cystine depleting agent, is approved in the U.S. for the treatment of NC in adults and children ages two years and older and in all patients with NC in Europe.
“I am pleased that PROCYSBI has been accepted for review in Canada and that the review process will be expedited,” said Krishna Polu, M.D., Chief Medical Officer of Raptor Pharmaceutical Corp. “There are currently no approved cystine depleting agents in Canada and the Priority Review status by Health Canada acknowledges the urgent unmet need Canadian patients with Cystinosis have for accessing PROCYSBI.”
About PROCYSBI® (cysteamine bitartrate) delayed-release capsules
PROCYSBI is a cystine depleting agent that is approved in the U.S. for the treatment of nephropathic cystinosis in adults and children ages two years and older. It is contraindicated in patients with a hypersensitivity to penicillamine. The most commonly reported side effects are vomiting, abdominal pain/discomfort, headaches, nausea, diarrhea, anorexia/decreased appetite, breath odor, fatigue, dizziness, skin odor and rash. For additional information on PROCYSBI, including full prescribing information, please visit www.procysbi.com.
About Raptor Pharmaceutical
Raptor Pharmaceutical Corp. is a global biopharmaceutical company focused on the development and commercialization of transformative therapeutics for rare, debilitating and often fatal diseases. With its recent acquisition of QUINSAIR, Raptor plans to develop MP-376, the pharmaceutical product known commercially as QUINSAIR, in cystic fibrosis and intends to pursue clinical programs in 2016 in bronchiectasis not associated with cystic fibrosis and to do work in preparation to support further clinical development in lung infections associated with nontuberculous mycobacteria. In addition, Raptor is developing RP103, known commercially as PROCYSBI, in additional therapeutic areas such Huntington’s disease and mitochondrial diseases including Leigh syndrome. Raptor holds several orphan drug designations, including orphan drug exclusivity for PROCYSBI for nephropathic cystinosis in the U.S. and EU. For additional information, please visit www.raptorpharma.com.
Forward-Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995. These statements are indicated by words or phrases such as "believes," "expects," "anticipates," "estimates," "plans," "continuing," "ongoing," "projected" and similar words or phrases and relate to future events, including statements regarding: PROCYSBI as a treatment option for patients with nephropathic cystinosis, Huntington's Disease and mitochondrial disorders, including Leigh Syndrome, orphan drug exclusivity for the therapy in the U.S., the number of patients with nephropathic cystinosis in Canada, the ability to obtain approval for marketing of PROCYSBI in Canada or the timing of such potential approval, Raptor's plans to develop MP-376 in cystic fibrosis, bronchiectasis not associated with cystic fibrosis and potentially also nontuberculous mycobacteria and the timing of those and Raptor's other development programs. 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. Raptor cautions readers not to place undue reliance on any such forward-looking statements, which speak only as of the date they were made. Factors which may contribute to differences in actual results include, among others: Raptor's ability to market and sell QUINSAIR; continued and increased market acceptance and sales of PROCYSBI in the U.S. and other territories; Raptor's ability to expand the use of RP103 and MP-376 and to receive regulatory approval for other indications; Raptor's reliance on single active pharmaceutical ingredient suppliers for PROCYSBI and QUINSAIR and other third parties in connection with drug product development; compliance with healthcare regulations, ongoing regulatory requirements and potential penalties; any serious adverse side effects associated with PROCYSBI, QUINSAIR or any other future products; any product liability claims; third-party payor coverage, reimbursement and pricing for PROCYSBI, QUINSAIR and future products; enacted and future healthcare legislation; Raptor's ability to obtain and maintain orphan drug or other regulatory exclusivity for PROCYSBI, QUINSAIR or any other future products; the integration of European operations with U.S. operations; relationships with key scientific and medical collaborators; intellectual property protection and claims and continued license rights; and Raptor's ability to fund its operations and make required payments on its debt. Certain of these risks, uncertainties and other factors are described in greater detail in the company's filings from time to time with the Securities and Exchange Commission (SEC), which Raptor strongly urges you to read and consider, including: Raptor's annual report on Form 10-K for the twelve months ended December 31, 2015 filed with the SEC on February 26, 2016, Raptor's quarterly reports on Form 10-Q for the quarterly periods ended March 31, 2015, June 30, 2015 and September 30, 2015 filed with the SEC on May 7, 2015, August 6, 2015 and November 5, 2015, respectively, and Raptor's other periodic reports filed with SEC, all of which are available free of charge on the SEC's web site at http://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 except as may be required by law.