Raptor Pharmaceutical Corp., a biopharmaceutical company, focuses on developing and commercializing transformative treatments for people affected by rare and debilitating diseases. Marketed Product PROCYSBI The company’s first commercial product, PROCYSBI (cysteamine bitartrate) delayed-release capsules (PROCYSBI), received marketing approval from the U.S. Food and Drug Administration (FDA) in 2013 for the management of nephropathic cystinosis in adults and children six years and older. 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. In August 2015, the company received FDA approval for the expanded use of PROCYSBI to treat children two to six years of age with nephropathic cystinosis. In Europe, PROCYSBI gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate), received marketing authorization in 2013 from the European Commission as an orphan medicinal product for the management of proven nephropathic cystinosis in the European Union (EU). The EU marketing authorization allows the company to commercialize PROCYSBI in the 28 Member States of the EU plus Norway, Liechtenstein and Iceland (which are not EU Member States but are part of the European Economic Area). PROCYSBI received 7 years of market exclusivity, through 2020 for patients 6 years and older as an orphan drug in the United States and 10 years of market exclusivity, through 2023, as an orphan drug in Europe. PROCYSBI received orphan drug designation for the treatment of patients ages two years to six years, through 2022. The company commenced commercial sales of PROCYSBI in the United States in 2013 and in Europe in 2014. Approved Product in Canada and Europe QUINSAIR QUINSAIR is a formulation of the antibiotic drug levofloxacin, suitable for inhalation through a nebulizer. QUINSAIR, as approved, is administered twice daily in 28-day cycles, using a hand-held nebulizer with a disposable handset known as the Zirela device, manufactured by its partner PARI Pharma GmbH, and configured specifically for use with QUINSAIR. In October 2015, the company acquired various assets and rights related to levofloxacin solution for inhalation, a pharmaceutical product also known as MP-376 and commercially as ‘QUINSAIR’, from Tripex Pharmaceuticals, LLC (Tripex). QUINSAIR received marketing authorization by the EC for treating chronic lung infection caused by the bacteria Pseudomonas aeruginosa in adults who have cystic fibrosis in 2015 and Health Canada in 2015 for the management of cystic fibrosis in patients aged 18 years or older with chronic pulmonary Pseudomonas aeruginosa infections. The company plans to launch QUINSAIR in Europe in the first half of 2016 and Canada later in 2016. The company plans to discuss the path to potential approval in the same indication in the United States with the FDA in 2016. The company would also pursue a clinical program for the development of MP-376 in non-cystic fibrosis related bronchiectasis in 2016 and are planning to do work in preparation to support further clinical development of MP-376 in nontuberculous mycobacteria. Clinical Development RP103 Clinical Development RP103, enteric-coated delayed-release cysteamine bitartrate, is being evaluated as a neuroprotective treatment for Huntington’s disease (HD). Centre Hospitalier Universitaire d’Angers in France, is conducting a Phase 2/3 clinical trial of RP103. This trial comprises an 18-month blinded, placebo-controlled phase, followed by an 18-month open-label phase
raptor pharmaceutical corp
7 Hamilton Landing
Novato, CA 94949
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