Mercaptamine bitartrate controlled-release - Chiesi Farmaceutici/Horizon Pharma

Drug Profile

Mercaptamine bitartrate controlled-release - Chiesi Farmaceutici/Horizon Pharma

Alternative Names: Cysteamine bitartrate - Chiesi Farmaceutici/Horizon Pharma; Cysteamine bitartrate controlled release - Chiesi Farmaceutici/Horizon Pharma; PROCYSBI; RP-103; RP-104; RP103-Q12H

Latest Information Update: 22 May 2018

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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
  • Phase II Non-alcoholic steatohepatitis
  • No development reported Malaria
  • Discontinued Leigh disease; Mitochondrial disorders; Neuronal ceroid lipofuscinosis

Most Recent Events

  • 13 Apr 2018 Discontinued - Phase-II/III for Leigh disease (In adolescents, In children) in USA (PO)
  • 13 Apr 2018 Discontinued - Phase-II/III for Mitochondrial disorders (In adolescents, In children) in USA (PO) (NCT02473445)
  • 13 Apr 2018 Raptor Pharmaceuticals terminates a phase II extension trial based upon sponsor decision to end development of mercaptamine bitartrate for Mitochondrial disorders (In children, In adolescents) in USA (PO) (NCT02473445)
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