Mercaptamine bitartrate controlled-release

Drug Profile

Mercaptamine bitartrate controlled-release

Alternative Names: Cysteamine bitartrate delayed-release; Delayed-release cysteamine; DR cysteamine; DR mercaptamine bitartrate; EC cysteamine; EC cysteamine bitartrate; EC mercaptamine bitartrate; Gastro-resistant cysteamine; Mercaptamine bitartrate delayed-release; PROCYSBI; RP-103; RP-104

Latest Information Update: 23 Jun 2017

Price : $50

At a glance

  • Originator University of California, San Diego
  • Developer Horizon Pharma; National Institute of Diabetes and Digestive and Kidney Diseases; Raptor Pharmaceutical Corp
  • Class Mercaptoethylamines; Small molecules; Sulfhydryl compounds
  • Mechanism of Action Antimetabolites; Glutathione synthase stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Huntington's disease; Cystinosis; Neuronal ceroid lipofuscinosis
  • New Molecular Entity No
  • Available For Licensing Yes - Non-alcoholic steatohepatitis

Highest Development Phases

  • Marketed Nephropathic cystinosis
  • Phase II/III Huntington's disease; Leigh disease; Mitochondrial disorders
  • Phase II Non-alcoholic steatohepatitis
  • Preclinical Malaria
  • Discontinued Neuronal ceroid lipofuscinosis

Most Recent Events

  • 19 Jun 2017 Registered for Nephropathic cystinosis in Canada (PO)
  • 12 Jun 2017 Mercaptamine bitartrate controlled-release (PROCYSBI®) licensed to Chiesi Farmaceutici SpA in Europe, the Middle East and Africa (EMEA) regions
  • 06 Mar 2017 Raptor Pharmaceuticals completes a phase II extension trial for Mitochondrial disorders (In children, In adolescents) in USA (NCT02473445)
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