Drug Profile


Alternative Names: Betaine-anhydrous; Cystadan; Cystadane; RM-003

Latest Information Update: 17 Apr 2017

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At a glance

  • Originator Orphan Medical
  • Developer Recordati; ReqMed
  • Class Quaternary ammonium compounds; Small molecules; Trimethyl ammonium compounds
  • Mechanism of Action Methyltransferase stimulants
  • Orphan Drug Status

    Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.

    Yes - Homocystinuria
  • New Molecular Entity Yes

Highest Development Phases

  • Marketed Homocystinuria
  • No development reported Infantile Refsum disease; Peroxisomal disorders; Zellweger syndrome

Most Recent Events

  • 13 Apr 2017 Recordati Rare Diseases acquires North American marketing rights to Betaine anhydrous from Recordati
  • 15 Dec 2016 Biomarkers information updated
  • 03 Jun 2016 No development reported - Phase-III for Zellweger syndrome, Peroxisomal disorders and Infantile Refsum disease in Canada (PO)
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