Arimoclomol - Orphazyme

Drug Profile

Arimoclomol - Orphazyme

Alternative Names: BRX-345; OR-01; OR-04; Orphazyme OR-01

Latest Information Update: 09 Nov 2017

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At a glance

  • Originator Biorex
  • Developer CytRx Corporation; Food and Drug Administration; Orphazyme; University of Kansas Medical Center
  • Class Hydroxylamines; Small molecules
  • Mechanism of Action Molecular chaperone modulators; Protein synthesis stimulants
  • Orphan Drug Status

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

    Yes - Inclusion body myositis; Amyotrophic lateral sclerosis
  • New Molecular Entity Yes
  • Available For Licensing Yes - Amyotrophic lateral sclerosis; Stroke

Highest Development Phases

  • Phase III Niemann-Pick diseases
  • Phase II/III Amyotrophic lateral sclerosis
  • Phase II Inclusion body myositis
  • Preclinical Gaucher's disease
  • No development reported Glioblastoma

Most Recent Events

  • 07 Nov 2017 Arimoclomol - Orphazyme receives Orphan Drug status for Inclusion body myositis in USA
  • 04 Nov 2017 No recent reports of development identified for preclinical development in Glioblastoma in USA (PO)
  • 01 Aug 2017 Phase-II clinical trials in Inclusion body myositis in USA (PO) (NCT02753530)
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