Mercaptamine bitartrate controlled-release

Drug Profile

Mercaptamine bitartrate controlled-release

Alternative Names: PROCYSBI; RP-103; RP-104; RP103-Q12H

Latest Information Update: 17 Aug 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
  • Preclinical Malaria
  • Discontinued Neuronal ceroid lipofuscinosis

Most Recent Events

  • 10 Jul 2017 Raptor completes a phase III trial for Nephropathic cystinosis in the Netherlands, France, Italy, United Kingdom and USA (NCT01733316)
  • 26 Jun 2017 Horizon Pharma completes a phase III trial in Nephropathic cystinosis (In adolescent, In adult, In children) in USA, France, Netherlands (NCT01197378)
  • 19 Jun 2017 Registered for Nephropathic cystinosis in Canada (PO)
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