Raptor Pharmaceutical Corp. (NASDAQ: RPTP) is an emerging global biopharmaceutical company focused on developing and commercializing life-altering therapeutics that treat rare, debilitating and often fatal diseases.
Raptor’s first product, PROCYSBI® (cysteamine bitartrate) delayed-release capsules, received FDA approval in April 2013 for the management of nephropathic cystinosis in adults and children 6 years and older. In Europe, PROCYSBI® gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate) received European Commission approval in September 2013 for the treatment of proven nephropathic cystinosis.
The company has active clinical development programs in multiple therapeutic areas such as nephropathic cystinosis, nonalcoholic fatty liver disease, Huntington’s disease (HD), and Leigh syndrome and other mitochondrial disease. Raptor holds several orphan drug designations, including U.S. orphan drug designation for RP103 in Huntington’s disease. Raptor holds intellectual property for the use of cysteamine in HD and other neurodegenerative disorders including Parkinson’s disease, Rett and the treatment of MeCP2-associated disorders. For additional information, please visit Products & Pipeline.
Clinical Development Pipeline
Raptor is developing its RP103 for the potential treatment of:
- Huntington’s disease (Phase 2/3 topline data announced Feb. 2014)
- Nonalcoholic Fatty Liver Disease (NAFLD) in Children (Phase 2b fully enrolled Jan 2014)
- Leigh Syndrome and Other Mitochondrial Diseases (IND submitted for Phase 2b in Dec. 2013)
Raptor’s other clinical development program:
- Convivia™(oral 4-methylpyrazole) for the potential management of acetaldehyde toxicity due to ALDH2 deficiency, an inherited metabolic disorder (Phase 2, licensed to Uni Pharma Ltd., Taiwan)
Raptor’s preclinical pipeline includes novel drug candidates to potentially treat cancers and other diseases. These programs include: