Orphan designation: marketing authorisation
Rare diseases
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The European Medicines Agency (EMA) is responsible for the scientific evaluation of marketing authorisation applications for all orphan medicines in the European Economic Area (EEA), as they fall under the mandatory scope of the centralised procedure.
In this section
In other sections
Sponsors of designated orphan medicines at the marketing authorisation stage should also consider the regulatory information in the sections below:
- Authorisation of medicines
- Conditional marketing authorisation
- Orphan incentives
- Activities after orphan designation
- Submitting annual reports on medicine development
- Changing the name or address of a sponsor
- Transferring an orphan designation
- Applying for orphan designation
- Rewards and incentives for paediatric medicines
- Market exclusivity: orphan medicines