On 12 October 2017, orphan designation (EU/3/17/1934) was granted by the European Commission to Edison Orphan Pharma BV, the Netherlands, for (R)-troloxamide quinone (also known as EPI-589) for the treatment of amyotrophic lateral sclerosis.

The sponsorship was transferred to PTC Therapeutics International Limited, Ireland, in November 2019.

Key facts

Active substance
(R)-troloxamide quinone
Disease / condition
Treatment of amyotrophic lateral sclerosis
Date of first decision
EU designation number

Review of designation

The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.

Sponsor's contact details

PTC Therapeutics International Limited
5th floor, 3 Grand Canal Plaza
Grand Canal Street Upper
Dublin 4, D04 EE70
Tel. +353 1 906 87 37

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.

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