Medicines and regulation
EMA’s 2025 review records 16 confirmed orphan designations
EMA’s annual review reports that 16 medicines recommended for marketing authorisation in 2025 had their orphan designation confirmed by the end of that year.
Overall, the agency recommended 104 human medicines for marketing authorisation in 2025, including 38 containing new active substances.
Why the categories matter
EMA reviews orphan status at the approval stage to assess whether the criteria for maintaining it are met. Its report also records five medicines that lost orphan status before receiving marketing authorisation: losing that status did not itself mean that the products were not authorised.
This is a retrospective regulatory overview for 2025, not an announcement of 16 new approvals in October 2026. Orphan designation and marketing authorisation should not be used as interchangeable terms.
Primary sources
Sources checked: 31 October 2026.
