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 |
|---|---|---|---|
| nanatinostat;valganciclovir EU/3/22/2673 5 clinical trials → |
Treatment of peripheral T-cell lymphoma | Withdrawn | 10 Aug 2022 |
| toll-like receptor 4 agonist EU/3/22/2672 |
Treatment of osteosarcoma | Active (positive) | 10 Aug 2022 |
| autologous CD34+ haematopoietic stem and progenitor cells genetically modified with a lentiviral vector encoding for the N-acetylgalactosamine 6-sulfatase cDNA EU/3/22/2671 |
Treatment of mucopolysaccharidosis type IV A (Morquio A syndrome) | Active (positive) | 10 Aug 2022 |
| efgartigimod alfa EU/3/18/1992 · now authorised as Vyvgart |
Treatment of myasthenia gravis | Active (positive) | 10 Aug 2022 |
| teclistamab EU/3/20/2331 34 clinical trials → |
Treatment of multiple myeloma | Withdrawn | 19 Jul 2022 |
| moxetumomab pasudotox EU/3/13/1150 |
Treatment of B-lymphoblastic leukaemia/lymphoma | Withdrawn | 19 Jul 2022 |
| (R)-3-(2,3-dihydroxypropyl)-6-fluoro-5-(2-fluoro-4-iodophenylamino)-8-methylpyrido[2,3-d]pyrimidine-4,7(3H,8H)-dione EU/3/22/2670 |
Treatment of familial adenomatous polyposis (FAP) | Active (positive) | 18 Jul 2022 |
| toripalimab EU/3/22/2669 168 clinical trials → |
Treatment of nasopharyngeal cancer | Withdrawn | 18 Jul 2022 |
| erlotinib EU/3/22/2668 14 clinical trials → |
Treatment of pachyonychia congenita | Active (positive) | 18 Jul 2022 |
| liraglutide EU/3/22/2667 6 clinical trials → |
Treatment of Wolfram syndrome | Active (positive) | 18 Jul 2022 |
| W253R/R275S tissue plasminogen activator EU/3/22/2666 |
Treatment of non-traumatic spontaneous intracerebral haemorrhage | Active (positive) | 18 Jul 2022 |
| pyridoxal 5'-phosphate EU/3/22/2665 |
Treatment of pyridoxal 5'‐phosphate homeostasis protein deficiency | Active (positive) | 18 Jul 2022 |
| human decorin fused to the truncated homing peptide CRK EU/3/22/2664 |
Treatment of epidermolysis bullosa | Active (positive) | 18 Jul 2022 |
| sodium phenylbutyrate EU/3/22/2663 |
Treatment of maple syrup urine disease | Active (positive) | 18 Jul 2022 |
| vilobelimab EU/3/22/2662 2 clinical trials → |
Treatment of pyoderma gangrenosum | Active (positive) | 18 Jul 2022 |
| 2,4,6,7,8,9-hexahydro-4-((2-methylphenyl)methyl)-7-(phenylmethyl)imidazo(1,2-a)pyrido(3,4-e)pyrimidin-5(1H)-one EU/3/22/2661 |
Treatment of glioma | Active (positive) | 18 Jul 2022 |
| panobinostat EU/3/22/2660 |
Treatment of glioma | Withdrawn | 18 Jul 2022 |
| Parsaclisib EU/3/22/2659 2 clinical trials → |
Treatment of autoimmune haemolytic anaemia | Active (positive) | 18 Jul 2022 |
| heterologous swine glyco-humanised polyclonal antibody against T lymphocytes EU/3/22/2658 |
Treatment in solid organ transplantation | Active (positive) | 18 Jul 2022 |
| Salmonella enterica, subsp. enterica, serovar Typhimurium, strain YS1646, live EU/3/22/2657 |
Treatment of schwannoma | Active (positive) | 18 Jul 2022 |
| odronextamab EU/3/22/2656 · now authorised as Ordspono 10 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 18 Jul 2022 |
| berzosertib EU/3/22/2655 3 clinical trials → |
Treatment of small cell lung cancer | Withdrawn | 18 Jul 2022 |
| (S)-1-(4-(1-(3,4,5-trimethoxyphenyl)-1H-imidazol-4-ylamino)thieno[2,3-d]pyrimidin-2-yl)pyrrolidine-2-carboxamide EU/3/22/2654 |
Treatment of fibrodysplasia ossificans progressiva | Withdrawn | 18 Jul 2022 |
| efgartigimod alfa EU/3/22/2653 |
Treatment of pemphigus | Withdrawn | 18 Jul 2022 |
| 3-(1,3-benzodioxol-5-yl)-5-(3-bromophenyl)-1H-pyrazole EU/3/22/2652 |
Treatment of multiple system atrophy | Active (positive) | 18 Jul 2022 |
| hydroquinidine hydrochloride EU/3/22/2651 |
Treatment of Brugada syndrome | Active (positive) | 18 Jul 2022 |
| zilucoplan EU/3/22/2650 4 clinical trials → |
Treatment of myasthenia gravis | Withdrawn | 18 Jul 2022 |
| odronextamab EU/3/22/2649 · now authorised as Ordspono 10 clinical trials → |
Treatment of follicular lymphoma | Withdrawn | 18 Jul 2022 |
| thiostrepton EU/3/22/2648 |
Treatment of malignant mesothelioma | Active (positive) | 18 Jul 2022 |
| autologous naive regulatory T cells transduced with a lentiviral vector encoding for a chimeric antigen receptor to recognise the HLA-A*02 antigen EU/3/22/2647 |
Treatment in solid organ transplantation | Active (positive) | 18 Jul 2022 |
| mRNA encoding modified human ornithine transcarbamylase EU/3/22/2646 |
Treatment of ornithine transcarbamylase deficiency | Active (positive) | 18 Jul 2022 |
| Fosmanogepix EU/3/22/2645 2 clinical trials → |
Treatment of invasive aspergillosis | Active (positive) | 18 Jul 2022 |
| Fosmanogepix EU/3/22/2644 2 clinical trials → |
Treatment of invasive candidiasis | Active (positive) | 18 Jul 2022 |
| Humanised IgG4 monoclonal antibody against active complement component 1, subcomponent s EU/3/22/2643 |
Treatment of autoimmune haemolytic anaemia | Active (positive) | 18 Jul 2022 |
| arimoclomol citrate EU/3/16/1659 |
Treatment of inclusion body myositis | Withdrawn | 18 Jul 2022 |
| Humanised anti-CD37 monoclonal antibody conjugated to maytansinoid DM1 (naratuximab emtansine) EU/3/15/1494 |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 18 Jul 2022 |
| arimoclomol EU/3/06/406 |
Treatment of amyotrophic lateral sclerosis | Withdrawn | 18 Jul 2022 |
| Pirfenidone EU/3/22/2630 13 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 24 Jun 2022 |
| recombinant human acid sphingomyelinase (also known as olipudase alfa) EU/3/01/056 · now authorised as Xenpozyme |
Treatment of Niemann-Pick disease | Active (positive) | 24 Jun 2022 |
| govorestat EU/3/22/2642 · now authorised as Nugalviq |
Treatment of galactosaemia | Active (positive) | 21 Jun 2022 |
| eltanexor EU/3/22/2641 |
Treatment of myelodysplastic syndromes | Active (positive) | 21 Jun 2022 |
| diflunisal EU/3/22/2640 · now authorised as Attrogy |
Treatment of ATTR amyloidosis | Withdrawn | 21 Jun 2022 |
| 2'-O-(2-methoxyethyl)-5-methyl-P-thiocytidylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouridylyl-(3'-O->5'-O)-2'-O-(2-methoxyethyl)-5-methyl-P-thiouri… EU/3/22/2639 |
Treatment of cystic fibrosis | Active (positive) | 21 Jun 2022 |
| modified mRNA encoding human methylmalonyl-coenzyme A mutase containing a polymorphism at position 671 EU/3/22/2638 |
Treatment of methylmalonic acidaemia | Active (positive) | 21 Jun 2022 |
| human allogeneic keratinocytes EU/3/22/2637 |
Treatment of partial deep dermal and full thickness burns | Active (positive) | 21 Jun 2022 |
| 2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting UBE3A antisense transcript RNA EU/3/22/2636 |
Treatment of Angelman syndrome | Active (positive) | 21 Jun 2022 |
| epcoritamab EU/3/22/2634 26 clinical trials → |
Treatment of follicular lymphoma | Withdrawn | 21 Jun 2022 |
| sirolimus EU/3/22/2633 32 clinical trials → |
Treatment of lymphatic malformations | Active (positive) | 21 Jun 2022 |
| Lithium carbonate EU/3/22/2632 4 clinical trials → |
treatment of TBR1-related disorder | Active (positive) | 21 Jun 2022 |
| sodium (4Z,7Z,10R,11E,13E,15Z,17S,19Z)10,17-dihydroxy-docosa-4,7,11,13,15,19-hexaenoate EU/3/22/2631 |
Prevention of retinopathy of prematurity | Active (positive) | 21 Jun 2022 |
| 4-[[(3S)-1-benzylpyrrolidin-3-yl]-methylamino]-2-fluoro-5-methyl-N-(1,3-thiazol-4-yl)benzenesulfonamide EU/3/22/2629 |
Treatment of SCN8A developmental and epileptic encephalopathy | Active (positive) | 21 Jun 2022 |
| 3-(1-(2',3'-dimethoxy-[1,1'-biphenyl]-4-yl)-1H-1,2,3-triazol-4-yl)benzoic acid EU/3/22/2628 |
Treatment of gain-of-function mutations of STIM1 and ORAI1 related diseases | Active (positive) | 21 Jun 2022 |
| ropeginterferon alfa-2b EU/3/22/2627 |
Treatment of chronic myeloid leukaemia | Active (positive) | 21 Jun 2022 |
| amitriptyline EU/3/22/2626 2 clinical trials → |
Treatment of erythromelalgia | Withdrawn | 21 Jun 2022 |
| sebetralstat EU/3/22/2625 · now authorised as Ekterly |
Treatment of hereditary angioedema | Active (positive) | 21 Jun 2022 |
| Doxorubicin EU/3/22/2624 126 clinical trials → |
Treatment of soft tissue sarcoma | Active (positive) | 21 Jun 2022 |
| Humanised IgG4 monoclonal antibody against A proliferation-inducing ligand EU/3/22/2623 |
Treatment of primary IgA nephropathy | Active (positive) | 21 Jun 2022 |
| losartan EU/3/22/2622 10 clinical trials → |
Treatment of osteogenesis imperfecta | Active (positive) | 21 Jun 2022 |
| Escherichia coli, strain Nissle 1917, expressing high affinity phenylalanine transporter, phenylalanine ammonia lyase and L-amino acid deaminase EU/3/22/2621 |
Treatment of hyperphenylalaninaemia | Withdrawn | 21 Jun 2022 |
| pasireotide EU/3/22/2620 |
Treatment of noninsulinoma pancreatogenous hypoglycemia syndrome | Active (positive) | 16 May 2022 |
| chimeric peptide of human glucagon-like peptide-1, glucagon and gastric inhibitory polypeptide analogues linked to a human immunoglobulin Fc fragment EU/3/22/2619 2 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 16 May 2022 |
| Icerguastat acetate EU/3/22/2618 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 16 May 2022 |
| autologous peripheral blood-derived CD4 T-cells CRISPR-edited at the CD40LG locus EU/3/22/2617 |
Treatment of hyper IgM syndromes | Active (positive) | 16 May 2022 |
| adeno-associated virus serotype 8 expressing the human gamma-sarcoglycan gene EU/3/22/2616 |
Treatment of limb-girdle muscular dystrophy | Active (positive) | 16 May 2022 |
| Ibudilast EU/3/22/2615 |
Treatment of fragile X syndrome | Active (positive) | 16 May 2022 |
| elamipretide EU/3/22/2614 2 clinical trials → |
Treatment of myopathic mitochondrial DNA depletion syndrome | Active (positive) | 16 May 2022 |
| streptococcus pyogenes, group A, type 3, strain Su, inactivated EU/3/22/2613 |
Treatment of lymphatic malformations | Active (positive) | 16 May 2022 |
| Elezanumab EU/3/22/2612 |
Treatment of spinal cord injury | Active (positive) | 16 May 2022 |
| Fusion protein composed of the first 2 immunoglobulin-like domains of the human Roundabout guidance receptor 2 fused to a human IgG1 crystallised fragment EU/3/22/2611 |
Treatment of focal segmental glomerulosclerosis | Withdrawn | 16 May 2022 |
| autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD123 EU/3/22/2610 |
Treatment of blastic plasmacytoid dendritic cell neoplasm | Active (positive) | 16 May 2022 |
| cannabidiol EU/3/22/2609 16 clinical trials → |
Treatment of epidermolysis bullosa | Active (positive) | 16 May 2022 |
| adeno-associated virus serotype R100 containing the human RPGRorf15 gene isoform EU/3/22/2608 |
Treatment of inherited retinal dystrophies due to defects in the RPGR gene | Active (positive) | 16 May 2022 |
| adeno-associated virus serotype C102 containing the human GLA gene EU/3/22/2607 |
Treatment of Fabry disease | Withdrawn | 13 Apr 2022 |
| adeno-associated viral vector serotype 8 encoding B-domain deleted liver specific codon optimized bioengineered chimeric human porcine factor VIII, under a syn… EU/3/22/2606 |
Treatment of haemophilia A | Active (positive) | 13 Apr 2022 |
| obecabtagene autoleucel EU/3/22/2605 · now authorised as Aucatzyl 3 clinical trials → |
Treatment of acute lymphoblastic leukaemia | Withdrawn | 13 Apr 2022 |
| (2S)‑4‑[2‑methoxyethyl‑[4‑(5,6,7,8‑tetrahydro‑1,8‑naphthyridin‑2‑yl)butyl]amino]‑2‑(quinazolin‑4‑ylamino)butanoic acid EU/3/22/2604 |
Treatment of primary sclerosing cholangitis | Withdrawn | 13 Apr 2022 |
| tiratricol EU/3/22/2603 |
Treatment of resistance to thyroid hormone type beta | Active (positive) | 13 Apr 2022 |
| Devimistat EU/3/22/2602 |
Treatment of biliary tract cancer | Active (positive) | 13 Apr 2022 |
| N-(methoxypolyethylene glycol 5000 carbamoyl)-1,2-dipalmitoyl-sn-glycero-3-phosphatidylethanolamine EU/3/22/2601 |
Treatment in solid organ transplantation | Active (positive) | 13 Apr 2022 |
| cannabidiol EU/3/22/2600 16 clinical trials → |
Treatment of epilepsy with myoclonic-atonic seizures | Active (positive) | 13 Apr 2022 |
| belzupacap sarotalocan EU/3/22/2599 |
Treatment of uveal melanoma | Active (positive) | 13 Apr 2022 |
| adeno-associated virus vector serotype 9 encoding human gigaxonin gene EU/3/22/2598 |
Treatment of giant axonal neuropathy | Withdrawn | 13 Apr 2022 |
| norucholic acid EU/3/22/2597 |
Treatment of primary biliary cholangitis | Active (positive) | 13 Apr 2022 |
| adeno-associated virus serotype 9 containing human MYBPC3 gene EU/3/22/2596 |
Treatment of Hypertrophic cardiomyopathy due to mutations in the MYBPC3 gene encoding cardiac myosin-binding protein C | Active (positive) | 13 Apr 2022 |
| Glofitamab EU/3/22/2595 46 clinical trials → |
Treatment of mantle cell lymphoma | Active (positive) | 13 Apr 2022 |
| heterologous human adult liver-derived stem cells EU/3/22/2594 |
Treatment of argininosuccinic aciduria | Active (positive) | 16 Mar 2022 |
| adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase EU/3/22/2593 |
Treatment of phenylalanine hydroxylase deficiency | Withdrawn | 16 Mar 2022 |
| adeno-associated virus serotype HSC 15 expressing human iduronate 2-sulfatase (Bevufenogene nofeparvovec) EU/3/22/2592 |
Treatment of mucopolysaccharidosis type II (Hunter Syndrome) | Withdrawn | 16 Mar 2022 |
| emactuzumab EU/3/22/2591 2 clinical trials → |
Treatment of tenosynovial giant cell tumour, localised and diffuse type | Active (positive) | 16 Mar 2022 |
| alisporivir EU/3/22/2590 |
Treatment of collagen VI-related myopathies | Active (positive) | 16 Mar 2022 |
| (S)-12-fluoro-4-(2-methylpyridin-3-yl)-7a,8,13,14-tetrahydro-7H-[1,2,4]triazolo[4',3':1,6]pyrido[3,2-b]benzofuro[4,3-fg][1,4]oxazonine EU/3/22/2589 |
Treatment of sickle cell disease | Withdrawn | 16 Mar 2022 |
| treprostinil sodium EU/3/22/2588 4 clinical trials → |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 16 Mar 2022 |
| tropatepine hydrochloride EU/3/22/2587 |
Treatment of narcolepsy | Active (positive) | 16 Mar 2022 |
| codergocrine mesilate;oxitriptan EU/3/22/2586 |
Treatment of fragile X syndrome | Active (positive) | 16 Mar 2022 |
| dersimelagon EU/3/22/2585 |
Treatment of erythropoietic protoporphyria | Active (positive) | 16 Mar 2022 |
| Hymecromone EU/3/22/2584 |
Treatment of spinal cord injury | Active (positive) | 16 Mar 2022 |
| cannabidiol EU/3/22/2583 16 clinical trials → |
Treatment of fragile X syndrome (FXS) | Active (positive) | 25 Feb 2022 |
| enzastaurin hydrochloride EU/3/22/2582 |
Treatment of Ehlers-Danlos syndrome | Withdrawn | 24 Feb 2022 |
| Epcoritamab EU/3/22/2581 26 clinical trials → |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 24 Feb 2022 |
| autologous CD34+ cells edited with CRISPR/Cas9 and transduced with an adeno-associated virus vector serotype 6 containing the codon-optimized version of PKLR g… EU/3/22/2580 |
Treatment of pyruvate kinase deficiency | Active (positive) | 24 Feb 2022 |
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.