Either you have JavaScript disabled or your browser does not support Javascript . To work properly, this page requires JavaScript to be enabled.
How to enable JavaScript in your browser?

Arimoclomol - Orphazyme

Drug Profile

Arimoclomol - Orphazyme

Alternative Names: Arimoclomol citrate; BRX-345; OR-01; OR-04

Latest Information Update: 29 Aug 2019

Price : $50 *
  • Adis is an information provider. We do not sell or distribute actual drugs.
  • Final gross price and currency may vary according to local VAT and billing address.
  • Your purchase entitles you to full access to the information contained in our drug profile at the time of purchase.
  • A link to download a PDF version of the drug profile will be included in your email receipt.

At a glance

  • Originator Biorex
  • Developer CytRx Corporation; Food and Drug Administration; Orphazyme; University of Kansas Medical Center
  • Class Hydroxylamines; Oxides; Pyridines; 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 Amyotrophic lateral sclerosis; Inclusion body myositis; Niemann-Pick diseases
  • Phase II Gaucher's disease
  • No development reported Glioblastoma

Most Recent Events

  • 27 Aug 2019 Orphazyme completes enrolment in a phase III trial for Amyotropic lateral sclerosis in USA, UK, Belgium, Canada, France, Germany, Italy, Netherlands, Poland, Spain, Sweden and Switzerland (NCT03491462)
  • 22 Jul 2019 Orphazyme completes enrolment in its phase III trial for Amyotrophic lateral sclerosis in USA, Canada, France, Germany, Italy, Netherlands, Poland, Spain, Sweden, Switzerland, United Kingdom (PO) (NCT03491462) (EudraCT2018-000137-13)
  • 20 May 2019 Phase-III clinical trials in Inclusion body myositis (In adults, In the elderly) in USA (PO) (NCT04049097)
Restricted Access

Oops, it looks like you don’t have a valid subscription to this content. To gain full access to the content and functionality of the AdisInsight database try one of the following.

  • with a username/password associated to an account with a valid subscription
  • Contact your organization’s admin about adding this content to your AdisInsight subscription
  • Request a trial

If you are a subscriber to this content then contact us at AsktheExpert.AdisInsight@springer.com so we can help.

Back to top