Drug Profile


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

Latest Information Update: 18 Jul 2016

Price : $50

At a glance

  • Originator Biorex
  • Developer CytRx Corporation; Orphazyme
  • 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 - Amyotrophic lateral sclerosis
  • New Molecular Entity Yes
  • Available For Licensing Yes - Amyotrophic lateral sclerosis; Stroke

Highest Development Phases

  • Phase II/III Amyotrophic lateral sclerosis; Niemann-Pick diseases
  • Preclinical Glioblastoma

Most Recent Events

  • 01 Jun 2016 Phase-II/III clinical trials in Niemann-Pick disease (Adjunctive treatment, In adolescents, In children) in Denmark (PO) (NCT02612129)
  • 27 Apr 2016 University of Kansas Medical Center and FDA Office of Orphan Products Development plan a phase II trial for Inclusion body myositis in USA (PO) (NCT02753530)
  • 26 Nov 2015 Orphazyme plans a phase II/III trial for Niemann-Pick disease (Adjunctive treatment, In children, In adolescents) in Denmark, United Kingdom, France, Germany, Spain, Italy, Poland and Switzerland (PO) (NCT02612129; CT-ORZY-NPC-002)
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