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 Nov 2026
3,310Orphan designations listed
2,201Currently active
114Designated in the last 12 months
1,073Withdrawn or expired
01
Matching designations
6 matches| Substance | Intended use | Status | Designated |
|---|---|---|---|
| allogeneic peripheral blood mononuclear cells incubated ex-vivo with 16, 16-dimethyl prostaglandin E2;dexamethasone EU/3/16/1774 341 clinical trials → |
Treatment in haematopoietic stem cell transplantation | Withdrawn | 18 Nov 2016 |
| Dexamethasone sodium phosphate encapsulated in human autologous erythrocytes EU/3/13/1158 |
Treatment of ataxia telangiectasia | Withdrawn | 17 Jul 2013 |
| dexamethasone EU/3/10/763 341 clinical trials → |
Treatment of non-infectious uveitis affecting the posterior segment of the eye | Withdrawn | 4 Aug 2010 |
| dexamethasone EU/3/10/745 341 clinical trials → |
Treatment of multiple myeloma | Withdrawn | 9 Jun 2010 |
| Dexamethasone phosphate EU/3/09/636 341 clinical trials → |
Treatment of corneal graft rejection | Withdrawn | 15 May 2009 |
| Dexamethasone sodium phosphate encapsulated in human erythrocytes EU/3/04/230 |
Treatment of cystic fibrosis | Withdrawn | 20 Oct 2004 |
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 Nov 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)
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