EU/3/06/406: Orphan designation for the treatment of amyotrophic lateral sclerosis


Table of contents


Please note that this product was withdrawn from the Union Register of orphan medicinal products in July 2022 on request of the Sponsor.

On 26 October 2006, orphan designation (EU/3/06/406) was granted by the European Commission to Wainwright Associates Ltd, United Kingdom, for arimoclomol for the treatment of amyotrophic lateral sclerosis.

The sponsorship was transferred to Orphazyme ApS, Denmark, in January 2012.

In October 2020, Orphazyme ApS changed name to Orphazyme A/S.

Key facts

Active substance
Intended use
Treatment of amyotrophic lateral sclerosis
Orphan designation status
EU designation number
Date of designation

Orphazyme A/S
Ole Maaløes Vej 3
2200 Copenhagen N
Tel. +45 39 17 82 72

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

EU register of orphan medicines

The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

How useful was this page?

Add your rating