On 9 December 2011, orphan designation (EU/3/11/937) was granted by the European Commission to Edison Europe Holding BV, the Netherlands, for alpha-tocotrienol quinone for the treatment of Leigh syndrome.
The sponsorship was transferred to PTC Therapeutics International Limited, Ireland, in November 2019.
|Disease / condition||
Treatment of Leigh syndrome
|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.