EU/3/08/576: Orphan designation for the treatment of methylmalonic acidaemia

carglumic acid


On 7 November 2008, orphan designation (EU/3/08/576) was granted by the European Commission to Orphan Europe SARL, France, for carglumic acid for the treatment of methylmalonic acidaemia.

Carglumic acid for treatment of methylmalonic acidaemia has been authorised in the EU as Carbaglu since 27 May 2011.

Please note that this product was withdrawn from the Community Register of designated orphan medicinal products in June 2021 at the end of the 10-year period of market exclusivity.

Key facts

Active substance
carglumic acid
Medicine name
Intended use
Treatment of methylmalonic acidaemia
Orphan designation status
EU designation number
Date of designation
Orphan Europe SARL
Immeuble Le Wilson
70 Avenue du Général de Gaulle
F-92800 Puteaux
Tel. +33 1 4773 6458
Fax +33 1 49 00 18 00

Review of designation

During its meeting of 4-5 May 2011, the Committee for Orphan Medicinal Products (COMP) reviewed the designations for Carbaglu (carglumic acid) as an orphan medicinal product for the treatment of isovaleric acidaemia, methylmalonic acidaemia and propionic acidaemia.

The COMP assessed whether, at the time of addition of these new indications to the marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the conditions, and the existence of other satisfactory methods of treatment.

As other satisfactory methods of treatment for patients with these conditions are authorised in the European Union (EU), the COMP also looked at the significant benefit of the product over existing treatments. The COMP recommended that the orphan designations of the medicine be maintained.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of addition of new indications to the marketing authorisation: Carbaglu (carglumic acid) for the treatment of isovaleric acidaemia, methylmalonic acidaemia... (PDF/141.26 KB)


    First published: 10/10/2011
    Last updated: 09/06/2021

  • 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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