EU/3/19/2166

Table of contents

About

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.

Key facts

Active substance
Sodium benzoate, sodium phenylacetate
Disease / condition
Treatment of carbamoyl-phosphate synthase-1 deficiency
Date of first decision
29/05/2019
Outcome
Positive
EU designation number
EU/3/19/2166

Sponsor's contact details

Dipharma B.V.
Prins Bernhardplein 200
1097 JB Amsterdam
Netherlands
Tel. +41 9160 11713
E-mail: regulatory@dipharma-group.com

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.

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