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: 17 Nov 2017

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

Most Recent Events

  • 13 Nov 2017 The Scottish Medicines Consortium rejects mercaptamine for routine use by NHS Scotland for the treatment of Nephropathic cystinosis
  • 04 Nov 2017 No recent reports of development identified for preclinical development in Malaria in USA
  • 10 Jul 2017 Raptor completes a phase III trial for Nephropathic cystinosis in the Netherlands, France, Italy, United Kingdom and USA (NCT01733316)
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