EU/3/00/007: Orphan designation for the treatment of N-acetylglutamate synthetase (NAGS) deficiency

N-carbamyl-L-glutamic acid


Please note that this product was withdrawn from the Community Register of designated Orphan Medicinal Products in January 2013 on request of the Sponsor.

On 18 October 2000, orphan designation (EU/3/00/007) was granted by the European Commission to Orphan Europe SARL, France, for n-carbamyl-l-glutamic acid for the treatment of n-acetylglutamate synthetase (NAGS) deficiency.

N-carbamyl-l-glutamic acid treatment of N-acetylglutamate synthetase (NAGS) deficiency has been authorised in the EU as Carbaglu since 24 January 2003.

For a list of the administrative updates to this public summary of opinion please refer to the PDF document below.

Key facts

Active substance
N-carbamyl-L-glutamic acid
Medicine name
Intended use
Treatment of N-acetylglutamate synthetase (NAGS) deficiency
Orphan designation status
EU designation number
Date of designation
Orphan Europe SARL
Immeuble Le Wilson
70 Avenue du Général de Gaulle
F-92800 Puteaux
Telephone: +33 1 47 73 64 58
Telefax: +33 1 49 06 00 04

Review of designation

Please note that this product (marketed as Carbaglu) was withdrawn from the Community register of designated orphan medicinal products in September 2013 on request of the sponsor, after the granting of a marketing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:

  • 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.

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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