EU/3/14/1336: Orphan designation for the treatment of Leigh syndrome
Table of contents
On 15 October 2014, orphan designation (EU/3/14/1336) was granted by the European Commission to Khondrion BV, the Netherlands, for (S)-6-Hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3‑yl)chroman-2-carboxamide hydrochloride for the treatment of Leigh syndrome.
Treatment of Leigh syndrome
|Orphan designation status||
|EU designation number||
|Date of designation||
Review of designation
The Committee for Orphan Medicinal Products reviews the orphan designation of a product if it is approved for marketing authorisation.
|March 2023||The sponsor's address was updated.|
Documents related to this orphan designation evaluation
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: