Raptor Pharmaceuticals announces publication of PROCYSBI data Raptor Pharmaceutical announced that data from its open label extension study of delayed-release cysteamine bitartrate, or PROCYSBI, has been published in the Journal of Pediatrics. The study demonstrated that patients with nephropathic cystinosis who took PROCYSBI for two years were able to sustain optimal cystine control in their white blood cells and preserve kidney function over the long term, and had significant improvements in social, school and total functioning based on validated quality of life measurement scales. There were no unexpected or serious safety concerns attributable to PROCYSBI. All 40 patients experienced one or more treatment-emergent adverse events.
Raptor Pharmaceuticals receives FDA approval of expanded label for Procysbi Raptor Pharmaceutical announced that the FDA approved the expanded use of Procysbi 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.