EU/3/14/1336: Orphan designation for the treatment of Leigh syndrome

(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride


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.

Key facts

Active substance
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride
Intended use
Treatment of Leigh syndrome
Orphan designation status
EU designation number
Date of designation

Khondrion BV
Transistorweg 5
6534 Nijmegen
The Netherlands
Tel. +31 243617505

Review of designation

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

Update history

March 2023The sponsor's address was updated.

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:

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