Arimoclomol - Orphazyme

Drug Profile

Arimoclomol - Orphazyme

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

Latest Information Update: 22 Jan 2018

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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; Niemann-Pick diseases
  • New Molecular Entity Yes

Highest Development Phases

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

Most Recent Events

  • 17 Apr 2018 Orphazyme plans a phase III trial for Amyotrophic lateral sclerosis in May 2018 (NCT03491462)
  • 06 Feb 2018 Orphazyme plans a phase II trial for Gaucher disease in Q2 2018
  • 24 Jan 2018 Orphazyme plans a phase II/III trial for Amyotrophic lateral sclerosis in USA in H2 2018
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