EU/3/16/1706: Orphan designation for the treatment of carbamoyl-phosphate synthase-1 deficiency
Table of contents
On 14 July 2016, orphan designation (EU/3/16/1706) was granted by the European Commission to Lucane Pharma SA, France, for sodium benzoate for the treatment of carbamoyl-phosphate synthase-1 deficiency.
Treatment of carbamoyl-phosphate synthase-1 deficiency
|Orphan designation status||
|EU designation number||
|Date of designation||
Lucane Pharma SA
172 rue de Charonne
Tel. +33 1 53 86 87 53
Fax +33 1 47 34 56 72
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
For contact details of patients’ organisations whose activities are targeted at rare diseases, see:
Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.
European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.
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: