Every medicine the European Commission has designated as an orphan medicine for a rare disease, on the EMA's recommendation, with its current status.
| 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 34 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Treatment of dermatomyositis | Withdrawn |
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.
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 27 Sep 2026.