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.
|Disease / condition||
Treatment myasthenia gravis
|Date of first decision||
|EU designation number||
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.
Sponsor's contact details
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.