European Union · EMA · Rare diseases
EU orphan designations
Every medicine the European Commission has designated as an orphan medicine for a rare disease, on the EMA's recommendation, with its current status.
Source: European Medicines Agency, orphan designations data (reproduced with acknowledgement of the source).
Synced 10 Oct 2026
3,310Orphan designations listed
2,204Currently active
125Designated in the last 12 months
1,070Withdrawn or expired
01
Matching designations
7 matches| Substance | Intended use | Status | Designated |
|---|---|---|---|
| Ibrutinib EU/3/16/1780 44 clinical trials → |
Treatment of graft-versus-host disease | Withdrawn | 18 Nov 2016 |
| Ibrutinib EU/3/15/1541 44 clinical trials → |
Treatment of marginal zone lymphoma | Withdrawn | 10 Aug 2015 |
| Ibrutinib EU/3/14/1264 44 clinical trials → |
Treatment of lymphoplasmacytic lymphoma | Withdrawn | 29 Apr 2014 |
| Ibrutinib EU/3/13/1212 44 clinical trials → |
Treatment of follicular lymphoma | Withdrawn | 16 Jan 2014 |
| Ibrutinib EU/3/13/1203 44 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 13 Nov 2013 |
| 1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one (ibrutinib) EU/3/13/1115 |
Treatment of mantle cell lymphoma | Withdrawn | 12 Mar 2013 |
| 1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H-pyrazolo[3,4 d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one (ibrutinib) EU/3/12/984 |
Treatment of chronic lymphocytic leukaemia | Withdrawn | 26 Apr 2012 |
How this page is built
Every row is copied from the EMA's orphan designations data, reproduced with acknowledgement of the source. Each substance links to its official EMA page.
- EMA orphan designations data (JSON report) ↗ active substance, intended use, EU designation number, date of designation, status and the link to each designation page
A daily job downloads the EMA's orphan designations JSON report and lists every designation, newest first. No AI drafting and no editorial pass. See our editorial standards. The the EMA data on this page was last retrieved on 10 Oct 2026. How every register is built: methodology · fixes we have made: corrections log.
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not medical advice, and it has not been reviewed by a clinician — our editorial
team holds life-science qualifications, not clinical ones. It is not exhaustive and may not reflect the most
recent label change. Always check the official prescribing information (US Prescribing Information or EU SmPC)
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