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
All orphan designations, newest first
3,310 designations · page 34 of 34| Substance | Intended use | Status | Designated |
|---|---|---|---|
| alpibectir;ethionamide EU/3/25/3116 |
Treatment of tuberculosis | Active (positive) | |
| IgG1 trispecific monoclonal antibody against T-cell receptor CD3, B-cell maturation antigen and G protein-coupled receptor class C group 5 member D EU/3/25/3106 |
Treatment of multiple myeloma | Active (positive) | |
| (R)-3-(5-dimethylcarbamoyl-pent-1-enyl)-N-(2-hydroxy-1methylethyl)benzamide EU/3/25/3074 |
Treatment of fragile X syndrome | Active (positive) | |
| nipocalimab EU/3/25/3045 12 clinical trials → |
Prevention of fetal and neonatal alloimmune thrombocytopenia | Active (positive) | |
| adeno-associated viral vector serotype 3B encoding human CYP27A1 EU/3/24/2971 |
Treatment of inborn errors in primary bile acid synthesis | Active (positive) | |
| Humanised IgG1 monoclonal antibody against misfolded immunoglobulin G, fused with pan-amyloid-reactive peptide p5R EU/3/24/2969 |
Treatment of AL amyloidosis | Active (positive) | |
| Interleukin 4 - interleukin 10 fusion protein EU/3/24/2960 |
Treatment of complex regional pain syndrome | Active (positive) | |
| ulefnersen EU/3/23/2839 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | |
| tamoxifen citrate EU/3/22/2635 39 clinical trials → |
Treatment of neuronal ceroid lipofuscinosis | Active (positive) | |
| (6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid EU/3/18/2070 |
Treatment of dermatomyositis | Withdrawn |
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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