EU/3/14/1304: Orphan designation for the treatment myasthenia gravis



On 29 July 2014, orphan designation (EU/3/14/1304) was granted by the European Commission to Alexion Europe SAS, France, for eculizumab for the treatment of myasthenia gravis.

Eculizumab in treatment myasthenia gravis in has been authorised in the EU as Soliris since 14 August 2017.

The sponsor’s address was updated in November 2019.

Key facts

Active substance
Medicine name
Intended use
Treatment myasthenia gravis
Orphan designation status
EU designation number
Date of designation

Alexion Europe S.A.S.
103-105 Rue Anatole France
92300 Levallois Perret

Review of designation

On 3 July 2017, the Committee for Orphan Medicinal Products (COMP) concluded its review of the designation EU/3/14/1304 for Soliris (eculizumab) as an orphan medicinal product for the treatment of myasthenia gravis. The COMP assessed whether, at the time of addition of a new indication to the marketing authorisation, the medicinal product still met the criteria for orphan designation. The Committee looked at the seriousness and prevalence of the condition, and the existence of other methods of treatment. As other methods of treatment are authorised in the European Union (EU), the COMP also considered whether the medicine is of significant benefit to patients with myasthenia gravis. The COMP recommended that the orphan designation of the medicine be maintained.1

1The maintenance of the orphan designation at time of marketing authorisation would, except in specific situations, give an orphan medicinal product 10 years of market exclusivity in the EU. This means that in the 10 years after its authorisation similar products with the same therapeutic indication cannot be placed on the market.

Documents related to this orphan designation evaluation

  • List item

    Recommendation for maintenance of orphan designation at the time of addition of a new indication to the marketing authorisation: Soliris (eculizumab) for the treatment of myasthenia gravis (PDF/80.71 KB)


    First published: 26/09/2017
    Last updated: 26/09/2017

  • 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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