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

Price : *
* Final gross price and currency may vary according to local VAT and billing address.

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)
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at so we can help.

Back to top