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Mercaptamine bitartrate controlled-release - Chiesi Farmaceutici/Horizon Therapeutics

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Drug Profile

Mercaptamine bitartrate controlled-release - Chiesi Farmaceutici/Horizon Therapeutics

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

Latest Information Update: 06 Nov 2023

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At a glance

  • Originator University of California, San Diego
  • Developer Horizon Pharma; Horizon Therapeutics plc; National Institute of Diabetes and Digestive and Kidney Diseases; Raptor Pharmaceutical Corp
  • Class Mercaptoethylamines; Small molecules; Sulfhydryl compounds
  • Mechanism of Action Metabolism-modulators
  • 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

  • 28 Apr 2020 Launched for Nephropathic cystinosis (In adolescents, In adults, In children, In infants) in USA (PO, Granules)
  • 19 Feb 2020 Registered for Nephropathic cystinosis (In adolescents, In adults, In children, In infants) in USA (PO, Granules)
  • 18 Feb 2020 Horizon Therapeutics plans to launch PROCYSBI® oral granules (75 mg and 300 mg) in the first half of 2020

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