On 7 November 2008, orphan designation (EU/3/08/575) was granted by the European Commission to Orphan Europe SARL, France, for carglumic acid for the treatment of isovaleric acidaemia.

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

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

Key facts

Active substance
carglumic acid
Medicine name
Disease / condition
Treatment of isovaleric acidaemia
Date of first decision
EU designation number

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.

Sponsor's contact details

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

Patients' organisations

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

  • Orphanet, a database containing information on rare diseases, which includes a directory of patients’ organisations registered in Europe.

  • European Organisation for Rare Diseases (EURORDIS), a non-governmental alliance of patient organisations and individuals active in the field of rare diseases.

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