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Raptor Pharmaceutical Receives FDA Approval

Expanded label for Procysbi to treat children aged 2-6 years with nephropathic cystinosis

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By: Tim Wright

Editor-in-Chief, Contract Pharma

Raptor Pharmaceutical Corp. received approval from the U.S. Food and Drug Administration (FDA) for 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...

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