EU/3/19/2157: Orphan designation for the treatment of ornithine transcarbamylase deficiency

Sodium benzoate / Sodium phenylacetate


On 24 April 2019, orphan designation (EU/3/19/2157) was granted by the European Commission to Dipharma B.V., the Netherlands, for sodium benzoate, sodium phenylacetate for the treatment of ornithine transcarbamylase deficiency.

Key facts

Active substance
  • Sodium benzoate
  • Sodium phenylacetate
Intended use
Treatment of ornithine transcarbamylase deficiency
Orphan designation status
EU designation number
Date of designation

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

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:

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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