EU/3/19/2165: Orphan designation for the treatment of citrullinaemia type 1

Sodium phenylacetate / Sodium benzoate

Table of contents


On 29 May 2019, orphan designation (EU/3/19/2165) was granted by the European Commission to Dipharma B.V., the Netherlands, for sodium benzoate, sodium phenylacetate for the treatment of citrullinaemia type 1.

Key facts

Active substance
  • Sodium phenylacetate
  • Sodium benzoate
Intended use
Treatment of citrullinaemia type 1
Orphan designation status
EU designation number
Date of designation

Dipharma B.V.
Prins Bernhardplein 200
1097 JB Amsterdam
Tel. +41 9160 11713

Documents related to this orphan designation evaluation

Patients' organisations

For contact details of patients’ organisations whose activities are targeted at rare diseases, see:

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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