Arimoclomol - Orphazyme

Drug Profile

Arimoclomol - Orphazyme

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

Latest Information Update: 05 Oct 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 - Niemann-Pick diseases; Amyotrophic lateral sclerosis; Inclusion body myositis
  • New Molecular Entity Yes

Highest Development Phases

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

Most Recent Events

  • 28 Sep 2018 Top-line safety and efficacy data from a phase II/III trial in Niemann-Pick disease released by Orphzyme
  • 13 Sep 2018 Orphazyme intends to submit MAA and NDA to EMA and US FDA, respectively, for Niemann-Pick disease in the second half of 2019
  • 13 Sep 2018 Orphazyme plans to launch arimoclomol for Niemann-Pick disease in 2020
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