EU/3/14/1242: Orphan designation for the treatment of Rett syndrome

3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl) amino]methyl}piperidin-1-yl]methanone


On 19 February 2014, orphan designation (EU/3/14/1242) was granted by the European Commission to Neurolixis UK Ltd., United Kingdom, for 3-chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl) amino]methyl}piperidin-1-yl]methanone for the treatment of Rett syndrome.

The sponsorship was transferred to Neurolixis SAS, France, in September 2018.

Key facts

Active substance
3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl) amino]methyl}piperidin-1-yl]methanone
Intended use
Treatment of Rett syndrome
Orphan designation status
EU designation number
Date of designation

Neurolixis SAS
2 rue Georges Charpak l'Arobase
le Causse Espace d'Entreprise
81290 Labruguiere
Tel. +33-563-59-60-37

Review of designation

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

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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