17 August 2015 - Raptor Pharmaceutical Corp. today announced that the U.S. Food and Drug Administration (FDA) approved the expanded use of Procysbi (cysteamine bitartrate) delayed-release capsules to treat children two to six years of age with nephropathic cystinosis. The approved supplement was based on efficacy and safety data from an ongoing long-term extension study in which a cohort of children aged 2 to 6 years were enrolled and treated with Procysbi for 12 months. Additionally, data submitted as part of this supplement supported the long-term maintenance of white blood cell cystine levels and renal function in all age groups studied during extended treatment with Procysbi. Procysbi is now approved for the treatment of nephropathic cystinosis in adult and in pediatric patients 2 years of age and older in the U.S.

"Strict adherence to cystine depleting therapy from as early an age as possible is critical to maintaining kidney function and leading longer, healthier lives for patients with cystinosis," said Craig Langman, M.D., The Isaac A. Abt, M.D. Professor of Kidney Diseases, Feinberg School of Medicine and Head of Kidney Diseases at Ann & Robert H. Lurie Children's Hospital of Chicago and the lead investigator on the extension trial. "The data in the two to six year old group confirm the maintenance of stable kidney function over time. Not only will this help these young patients with cystinosis, but the 12 hour dosing schedule for Procysbi could be a significant benefit to the caregivers of these young children."

For more details, go to: http://ir.raptorpharma.com/releasedetail.cfm?ReleaseID=927832