Five Patents for PROCYSBI Now Listed in FDA Orange Book
Orphan Drug Exclusivity Extended in Nephropathic Cystinosis Patients 2-6 Years Old
NOVATO, Calif., Jan. 25, 2016 (GLOBE NEWSWIRE) -- Raptor Pharmaceutical Corp. (Nasdaq:RPTP) today announced that a U.S. PROCYSBI patent, number 9,233,077, is now listed in the FDA publication Approved Drug Products with Therapeutic Equivalence Evaluations, commonly known as the Orange Book. The patent, entitled “Delayed Release Cysteamine Bead Formulation, and Methods of Making and Using Same,” covers the enteric coated micro-bead formulation of PROCYSBI as well as other related formulations. This patent expires in June 2034. Raptor Pharmaceutical now has five patents for PROCYSBI listed in the Orange Book, including four that issued in the last three months.
In addition, the U.S. Food and Drug Administration (FDA) granted additional orphan drug designation for PROCYSBI® (cysteamine bitartrate) delayed-release capsules to treat nephropathic cystinosis in children aged 2 to 6 years old. The FDA approved the expanded use of PROCYSBI in this patient population in August 2015 after initially approving PROCYSBI for the treatment of nephropathic cystinosis in adults and children 6 years and older in April 2013.
Orphan drug designation for PROCYSBI has been granted post-approval, conferring the benefit of seven years of orphan drug exclusivity for the treatment of nephropathic cystinosis in pediatric patients 2 to 6 years of age. The seven-year term of orphan exclusivity for this recently approved pediatric indication will expire in August 2022. The FDA's Orphan Drug Designation program grants orphan status to drugs intended for the treatment of rare disorders affecting fewer than 200,000 people in the U.S. Orphan drug designation, if requested and subsequently awarded, is typically granted prior to a New Drug Application (NDA) submission, making potential drugs eligible for benefits including tax credits for clinical research costs and a waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
About PROCYSBI® (cysteamine bitartrate) delayed-release capsules
PROCYSBI is a cystine depleting agent that is approved in the U.S. for the management of nephropathic cystinosis in adults and children ages 2 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 in at least one other potential indication of bronchiectasis or lung infections associated with nontuberculous mycobacteria. In addition, Raptor is developing RP103, known commercially as PROCYSBI, in multiple therapeutic areas such as nephropathic cystinosis, Huntington’s disease, and mitochondrial diseases including Leigh syndrome. Raptor holds several orphan drug designations, including orphan drug exclusivity for nephropathic cystinosis in the U.S. and EU. For additional information, please visit www.raptorpharma.com.
This document 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, but not limited to, statements regarding: PROCYSBI as a treatment option for patients with nephropathic cystinosis, orphan drug exclusivity and patent protection for the therapy in the U.S., Raptor's plans to develop MP-376 in cystic fibrosis and at least one of bronchiectasis or nontuberculous mycobacteria 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; 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, 2014 filed with the SEC on March 2, 2015, 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.
Kimberly Lee, D.O.
Vice President, Corporate Strategy and Communications
Raptor Pharmaceutical Corp.
Westwicke Partners, LLC
Robert H. Uhl
Source:Raptor Pharmaceutical Inc