Raptor Pharmaceutical Announces Positive Data on RP103 Extension Study

Raptor Pharmaceutical RPTP, announced that Craig B. Langman, M.D., Head of Kidney Diseases, and the Isaac A. Abt, M.D., Professor of Kidney Diseases and Tenured Professor of Pediatrics, Northwestern University Feinberg School of Medicine, presented on Saturday, November 3rd a "Late Breaking" poster entitled "Extended Treatment with RP103 in Patients with Nephropathic Cystinosis" at Kidney Week 2012. The poster presented data from Raptor's extension study in which 40 patients completing the Phase 3 clinical trial elected to continue on RP103 (cysteamine bitartrate delayed-release) treatment and to be monitored for white blood cell ("WBC") cystine levels, plasma cysteamine levels, estimated glomerular filtration rate ("eGFR", an indicator of kidney function), quality of life (PedsQL) and use of antacids. The data from the extension study demonstrated that patients with nephropathic cystinosis using RP103 for up to 24 months, maintained WBC cystine levels continuously below the therapeutic target. After 20 months of treatment, the total daily dose of RP103 given every 12 hours needed to achieve optimal WBC cystine levels was on average 72% of the initial immediate release cysteamine bitartrate dose. Kidney function was well preserved over the length of the See full press release
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