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 9 Nov 2026
3,310Orphan designations listed
2,201Currently active
114Designated in the last 12 months
1,073Withdrawn or expired
01
Matching designations
13 matches| Substance | Intended use | Status | Designated |
|---|---|---|---|
| Doxorubicin, liposomal, pegylated EU/3/25/3056 156 clinical trials → |
Treatment of soft-tissue sarcoma | Active (positive) | 22 May 2025 |
| Doxorubicin EU/3/22/2624 156 clinical trials → |
Treatment of soft tissue sarcoma | Active (positive) | 21 Jun 2022 |
| doxorubicin hydrochloride in a lipid-based pegylated nanoparticle modified with a 31-aminoacid peptide targeting nucleolin EU/3/16/1814 |
Treatment of malignant mesothelioma | Active (positive) | 12 Jan 2017 |
| Doxorubicin EU/3/15/1513 156 clinical trials → |
Treatment of hepatoblastoma | Active (positive) | 28 Jul 2015 |
| Doxorubicin(6-maleimidocaproyl)hydrazone (aldoxorubicin) EU/3/14/1258 |
Treatment of soft tissue sarcoma | Withdrawn | 26 Mar 2014 |
| Doxorubicin EU/3/12/1006 156 clinical trials → |
Treatment of glioma | Withdrawn | 28 Nov 2012 |
| doxorubicin hydrochloride EU/3/10/833 156 clinical trials → |
Treatment of hepatocellular carcinoma | Active (positive) | 23 Feb 2011 |
| glutathione-pegylated liposomal doxorubicin hydrochloride EU/3/10/781 |
Treatment of glioma | Withdrawn | 20 Sep 2010 |
| Pyr-His-Trp-Ser-Tyr-D-Lys(doxorubicinylglutarate)-Leu-Arg-Pro-Gly-NH2, acetate salt EU/3/10/766 |
Treatment of ovarian cancer | Withdrawn | 4 Aug 2010 |
| doxorubicin hydrochloride EU/3/07/507 156 clinical trials → |
Treatment of glioma | Withdrawn | 30 Nov 2007 |
| doxorubicin hydrochloride EU/3/06/410 156 clinical trials → |
Treatment of soft tissue sarcoma | Active (positive) | 27 Oct 2006 |
| Doxorubicine polyisohexylcyanoacrylate nanoparticles EU/3/04/229 |
Treatment of hepatocellular carcinoma | Withdrawn | 21 Oct 2004 |
| Doxorubicin carbon;iron magnetically targeted microparticles EU/3/02/112 |
Treatment of hepatocellular carcinoma | Withdrawn | 11 Sep 2002 |
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 9 Nov 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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