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
9 matches| Substance | Intended use | Status | Designated |
|---|---|---|---|
| imatinib EU/3/21/2449 35 clinical trials → |
Treatment of pulmonary arterial hypertension | Active (positive) | 21 Jun 2021 |
| imatinib EU/3/14/1357 35 clinical trials → |
Treatment of acute respiratory distress syndrome (ARDS) | Active (positive) | 19 Nov 2014 |
| imatinib mesilate EU/3/05/340 35 clinical trials → |
Treatment of myelodysplastic / myeloproliferative diseases | Expired | 23 Dec 2005 |
| imatinib mesilate EU/3/05/320 35 clinical trials → |
Treatment of chronic eosinophilic leukaemia and the hypereosinophilic syndrome | Expired | 28 Oct 2005 |
| imatinib mesilate EU/3/05/306 35 clinical trials → |
Treatment of mastocytosis | Withdrawn | 26 Aug 2005 |
| imatinib mesilate EU/3/05/305 35 clinical trials → |
Treatment of dermatofibrosarcoma protuberans | Expired | 26 Aug 2005 |
| imatinib mesilate EU/3/05/304 35 clinical trials → |
Treatment of acute lymphoblastic leukaemia | Expired | 26 Aug 2005 |
| imatinib mesilate EU/3/01/061 35 clinical trials → |
Treatment of malignant gastrointestinal stromal tumours | Expired | 20 Nov 2001 |
| imatinib mesilate EU/3/01/021 35 clinical trials → |
Treatment of chronic myeloid leukaemia | Expired | 15 Feb 2001 |
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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This page is general information for life-science and pharmaceutical professionals. It is
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)
and speak to your doctor or pharmacist before acting on anything here. Drugs in the same class are not
automatically interchangeable, and approvals, brand names and indications differ between the US, the
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