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.
|Disease / condition||
Treatment of amyotrophic lateral sclerosis
|Date of first decision||
|EU designation number||
Review of designation
Sponsor's contact details
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.