EU/3/14/1420: Orphan designation for the treatment of Aicardi-Goutières syndrome



On 15 January 2015, orphan designation (EU/3/14/1420) was granted by the European Commission to Dr Yanick Crow, France, for emtricitabine for the treatment of Aicardi-Goutières syndrome.

Key facts

Active substance
Intended use
Treatment of Aicardi-Goutières syndrome
Orphan designation status
EU designation number
Date of designation
Dr Yanick Crow
Institute of genetic disease
Laboratory of neurogenetics and neuroinflammation
24 Boulevard du Montparnasse
75015 Paris
Tel. +33 1 42 75 44 51

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:

  • 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.

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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