On 29 May 2019, orphan designation (EU/3/19/2166) was granted by the European Commission to Dipharma B.V., Netherlands, for sodium benzoate, sodium phenylacetate for the treatment of carbamoyl-phosphate synthase-1 deficiency.
Sodium benzoate, sodium phenylacetate
|Disease / condition||
Treatment of carbamoyl-phosphate synthase-1 deficiency
|Date of first decision||
|EU designation number||
Sponsor's contact details
For contact details of patients’ organisations whose activities are targeted at rare diseases, see: