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 |
|---|---|---|---|
| teriparatide EU/3/16/1689 4 clinical trials → |
Treatment of hypoparathyroidism | Active (positive) | 27 Jun 2016 |
| setmelanotide EU/3/16/1688 4 clinical trials → |
Treatment of Prader-Willi syndrome | Active (positive) | 27 Jun 2016 |
| recombinant protein derived from the saliva of the Ornithodoros moubata tick (nomacopan) EU/3/16/1687 |
Treatment of Guillain-Barrรฉ syndrome | Active (positive) | 27 Jun 2016 |
| recombinant humanised monoclonal IgG2 lambda antibody against human sclerostin EU/3/16/1686 |
Treatment of osteogenesis imperfecta | Active (positive) | 27 Jun 2016 |
| molgramostim EU/3/16/1685 3 clinical trials → |
Treatment of acute respiratory distress syndrome (ARDS) | Active (positive) | 27 Jun 2016 |
| modified mRNA encoding the UGT1A1 protein EU/3/16/1684 |
Treatment of Crigler-Najjar syndrome | Active (positive) | 27 Jun 2016 |
| melatonin EU/3/16/1683 23 clinical trials → |
Treatment of neonatal sepsis | Active (positive) | 27 Jun 2016 |
| Humanised monoclonal antibody targeting interleukin-15 EU/3/16/1681 |
Treatment of eosinophilic oesophagitis | Active (positive) | 27 Jun 2016 |
| Humanised anti-IL-6 receptor monoclonal antibody (satralizumab) EU/3/16/1680 · now authorised as Enspryng |
Treatment of neuromyelitis optica spectrum disorders | Active (positive) | 27 Jun 2016 |
| eflornithine EU/3/16/1679 6 clinical trials → |
Treatment of glioma | Active (positive) | 27 Jun 2016 |
| Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment- EU/3/16/1678 · now authorised as Luxceptar |
Treatment in haematopoietic stem cell transplantation | Withdrawn | 27 Jun 2016 |
| diclofenamide EU/3/16/1677 |
Treatment of periodic paralysis | Active (positive) | 27 Jun 2016 |
| Cyclocreatine EU/3/16/1676 |
Treatment of creatine deficiency syndromes | Withdrawn | 27 Jun 2016 |
| citric acid monohydrate EU/3/16/1675 |
Treatment of acute liver failure | Active (positive) | 27 Jun 2016 |
| allogeneic donor-derived ex-vivo expanded T lymphocytes transduced with a retroviral vector containing inducible caspase 9 and truncated CD19 EU/3/16/1674 |
Treatment in haematopoietic stem cell transplantation | Withdrawn | 27 Jun 2016 |
| pyridoxine;l-pyroglutamic acid EU/3/16/1673 |
Treatment of fragile X syndrome | Active (positive) | 27 Jun 2016 |
| 3-(5-Amino-2-methyl-4-oxoquinazolin-3(4H)-yl)piperidine-2,6-dione hydrochloride EU/3/16/1672 |
Treatment of diffuse large B-cell lymphoma | Withdrawn | 27 Jun 2016 |
| 2'-O-(2-Methoxyethyl) phosphorothioate antisense oligonucleotide targeting the growth hormone receptor EU/3/16/1671 |
Treatment of acromegaly | Withdrawn | 27 Jun 2016 |
| vemurafenib EU/3/16/1670 11 clinical trials → |
Treatment of Langerhans' cell histiocytosis | Active (positive) | 30 May 2016 |
| temsirolimus EU/3/16/1669 4 clinical trials → |
Treatment of adrenoleukodystrophy | Active (positive) | 30 May 2016 |
| sodium nitrite;ethylenediaminetetraacetic acid EU/3/16/1668 |
Treatment of cystic fibrosis | Active (positive) | 30 May 2016 |
| rovalpituzumab tesirine EU/3/16/1667 |
Treatment of small cell lung cancer | Withdrawn | 30 May 2016 |
| rimiducid EU/3/16/1666 8 clinical trials → |
Treatment of graft-versus-host disease | Withdrawn | 30 May 2016 |
| recombinant adeno-associated viral vector containing the human RPGR gene EU/3/16/1665 |
Treatment of retinitis pigmentosa caused by mutations in the RPGR gene | Active (positive) | 30 May 2016 |
| polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase (pegtibatinase) EU/3/16/1664 |
Treatment of homocystinuria | Active (positive) | 30 May 2016 |
| pentosan polysulfate sodium EU/3/16/1663 3 clinical trials → |
Treatment of interstitial cystitis | Active (positive) | 30 May 2016 |
| H-Phe-Ser-Arg-Tyr-Ala-Arg-OH acetate (Alirinetide) EU/3/16/1662 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 30 May 2016 |
| Fc- and CDR-modified humanised monoclonal antibody against C5 (ravulizumab) EU/3/16/1661 |
Treatment of paroxysmal nocturnal haemoglobinuria | Withdrawn | 30 May 2016 |
| autologous CD34+ cells transduced with lentiviral vector encoding the human beta globin gene EU/3/16/1660 |
Treatment of beta thalassaemia intermedia and major | Active (positive) | 30 May 2016 |
| 4-[(2E)-1-oxo-3-(2,6,6-trimethyl-1-cyclohexen-1-yl)-2-propen-1-yl]-1-piperazinecarboxamide EU/3/16/1658 |
Treatment of retinitis pigmentosa | Withdrawn | 30 May 2016 |
| (R)-6-(2-fluorophenyl)-N-(3-(2-((2-methoxyethyl)amino)ethyl)phenyl)-5,6-dihydrobenzo[h]quinazolin-2-amine dihydrochloride (derazantinib) EU/3/16/1657 |
Treatment of biliary tract cancer | Active (positive) | 30 May 2016 |
| (S)-ethyl 2-amino-3-(4-(2-amino-6((R)-1-(4-chloro-2-(3-methyl-1H-pyrazol-1-yl)phenyl)-2,2,2-trifluoroethoxy)pyrimidin-4-yl)phenyl)propanoate EU/3/09/661 · now authorised as Xermelo |
Treatment of carcinoid syndrome | Active (positive) | 30 May 2016 |
| Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu, Ser-Gly-Gln-Ala-Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu-Pro-Ser-Cys-Leu-Glu-Ser, Arg-Ser-Asp-Glu-Leu-Val-Arg-His-His-Asn-Met-Hโฆ EU/3/16/1656 |
Treatment of malignant mesothelioma | Active (positive) | 28 Apr 2016 |
| Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu, Ser-Gly-Gln-Ala-Tyr-Met-Phe-Pro-Asn-Ala-Pro-Tyr-Leu-Pro-Ser-Cys-Leu-Glu-Ser, Arg-Ser-Asp-Glu-Leu-Val-Arg-His-His-Asn-Met-Hโฆ EU/3/16/1655 |
Treatment of acute myeloid leukaemia | Active (positive) | 28 Apr 2016 |
| S-acetyl-(S)-4'-phosphopantetheine, calcium salt EU/3/16/1654 |
Treatment of pantothenate-kinase-associated neurodegeneration | Active (positive) | 28 Apr 2016 |
| resiquimod EU/3/16/1653 |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 28 Apr 2016 |
| recombinant human cerebral dopamine neurotrophic factor EU/3/16/1652 |
Treatment of amyotrophic lateral sclerosis | Active (positive) | 28 Apr 2016 |
| recombinant adeno-associated viral vector serotype 9 carrying the gene for the human E6-AP ubiquitin protein ligase EU/3/16/1651 |
Treatment of Angelman syndrome | Active (positive) | 28 Apr 2016 |
| N-carboxymethyl-glycyl-L-threonyl-L-histidyl-L-3,3-diphenylalanyl-L-piperidincarboxy-3-yl-L-arginyl-L-S-methylthio-cystyl-L-arginyl-L-tryptophyl-aminohexanyl-Nโฆ EU/3/16/1650 |
Treatment of beta thalassaemia intermedia and major | Withdrawn | 28 Apr 2016 |
| Humanised recombinant IgG4 anti-human tau antibody (tilavonemab) EU/3/16/1649 |
Treatment of progressive supranuclear palsy | Withdrawn | 28 Apr 2016 |
| Human/murine chimeric monoclonal antibody against endoglin (carotuximab) EU/3/16/1648 |
Treatment of soft tissue sarcoma | Withdrawn | 28 Apr 2016 |
| Fluocinolone acetonide EU/3/16/1647 |
Treatment of non-infectious uveitis | Withdrawn | 28 Apr 2016 |
| 4-hydroxyandrostenedione;serenoa serrulata;alpha lipoic acid EU/3/16/1646 9 clinical trials → |
Treatment of multiple symmetric lipomatosis | Active (positive) | 28 Apr 2016 |
| cannabidiol EU/3/16/1645 16 clinical trials → |
Prevention of graft-versus-host disease | Active (positive) | 28 Apr 2016 |
| brincidofovir EU/3/16/1644 |
Prevention of cytomegalovirus disease | Active (positive) | 28 Apr 2016 |
| autologous stromal vascular cell fraction from adipose tissue EU/3/16/1643 |
Treatment of systemic sclerosis | Withdrawn | 28 Apr 2016 |
| autologous dermal fibroblasts genetically modified ex vivo with a lentiviral vector containing the human COL7A1 gene EU/3/16/1642 |
Treatment of epidermolysis bullosa | Active (positive) | 28 Apr 2016 |
| antisense oligonucleotide complementary to the exonic splicer enhancer sequence at intron 26 of the centrosomal protein 290 pre-mRNA EU/3/16/1641 |
Treatment of Leber's congenital amaurosis | Active (positive) | 28 Apr 2016 |
| 2-methyl-1-[(4-[6-(trifluoromethyl)pyridin-2-yl]-6-{[2-(trifluoromethyl)pyridin-4-yl]amino}-1,3,5-triazin-2-yl)amino]propan-2-ol methanesulfonate (enasidenib) EU/3/16/1640 |
Treatment of acute myeloid leukaemia | Active (positive) | 28 Apr 2016 |
| (1E,6E)-1,7-bis(3,4-dimethoxyphenyl)-4-cyclobutylmethyl-1,6-heptadiene-3,5-dione EU/3/16/1639 |
Treatment of X-linked spinal and bulbar muscular atrophy (Kennedy's disease) | Active (positive) | 28 Apr 2016 |
| ubenimex EU/3/16/1638 |
Treatment of pulmonary arterial hypertension | Active (positive) | 21 Mar 2016 |
| synthetic double-stranded siRNA oligonucleotide directed against hydroxyacid oxidase 1 mRNA and covalently linked to a ligand containing three N-acetylgalactosโฆ EU/3/16/1637 · now authorised as Oxlumo |
Treatment of primary hyperoxaluria | Active (positive) | 21 Mar 2016 |
| sindbis virus envelope pseudotyped lentiviral vector encoding New York oesophageal squamous cell carcinoma-1 protein EU/3/16/1636 |
Treatment of soft tissue sarcoma | Withdrawn | 21 Mar 2016 |
| N-acetyl-D-mannosamine monohydrate EU/3/16/1635 |
Treatment of GNE myopathy | Active (positive) | 21 Mar 2016 |
| Glucopyranosyl lipid A stable emulsion and recombinant New York oesophageal squamous cell carcinoma-1 protein EU/3/16/1634 |
Treatment of soft tissue sarcoma | Withdrawn | 21 Mar 2016 |
| Fosbretabulin tromethamine EU/3/16/1633 |
Treatment of gastro-entero-pancreatic neuroendocrine tumours | Withdrawn | 21 Mar 2016 |
| Florilglutamic acid (18F) EU/3/16/1632 |
Diagnosis of hepatocellular carcinoma | Withdrawn | 21 Mar 2016 |
| Florilglutamic acid (18F) EU/3/16/1631 |
Diagnosis of glioma | Withdrawn | 21 Mar 2016 |
| Fenretinide EU/3/16/1630 2 clinical trials → |
Treatment of cutaneous T-cell lymphoma | Withdrawn | 21 Mar 2016 |
| exenatide EU/3/16/1629 2 clinical trials → |
Treatment of idiopathic intracranial hypertension | Active (positive) | 21 Mar 2016 |
| Diaspirin cross-linked haemoglobin EU/3/16/1628 |
Treatment of oesophageal cancer | Active (positive) | 21 Mar 2016 |
| allogeneic Epstein-Barr virus specific cytotoxic T lymphocytes (Tabelecleucel) EU/3/16/1627 · now authorised as Ebvallo |
Treatment of post-transplantation lymphoproliferative disorders | Active (positive) | 21 Mar 2016 |
| acalabrutinib EU/3/16/1626 48 clinical trials → |
Treatment of lymphoplasmacytic lymphoma | Active (positive) | 21 Mar 2016 |
| acalabrutinib EU/3/16/1625 48 clinical trials → |
Treatment of mantle cell lymphoma | Withdrawn | 21 Mar 2016 |
| acalabrutinib EU/3/16/1624 48 clinical trials → |
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma | Withdrawn | 21 Mar 2016 |
| adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase (avalotcagene ontaparvovec) EU/3/16/1623 |
Treatment of ornithine transcarbamylase deficiency | Active (positive) | 21 Mar 2016 |
| adeno-associated viral vector serotype 5 containing a B-domain deleted variant of human coagulation factor VIII gene EU/3/16/1622 · now authorised as Roctavian; Roctavian |
Treatment of haemophilia A | Active (positive) | 21 Mar 2016 |
| Delta-9-tetrahydrocannabinol and cannabidiol from extracts of the Cannabis sativa L. plant EU/3/16/1621 |
Treatment of glioma | Active (positive) | 17 Feb 2016 |
| allogeneic fetal human retinal progenitor cells expanded ex vivo EU/3/16/1620 |
Treatment of retinitis pigmentosa | Active (positive) | 17 Feb 2016 |
| DNA plasmid encoding a recombinant fusion protein consisting of the extracellular domain of human TNFฮฑ p55 receptor linked to the human IgG1 Fc domain EU/3/16/1619 |
Treatment of non-infectious uveitis | Active (positive) | 17 Feb 2016 |
| venetoclax EU/3/16/1617 204 clinical trials → |
Treatment of acute myeloid leukaemia | Withdrawn | 17 Feb 2016 |
| diclofenamide EU/3/16/1616 |
Treatment of periodic paralysis | Active (positive) | 17 Feb 2016 |
| 2-Ethylbutyl (2S)-2-{[(S)-{[(2R,3S,4R,5R)-5-(4-aminopyrrolo[2,1-f][1,2,4]triazin-7-yl)-5-cyano-3,4-dihydroxytetrahydrofuran-2-yl]methoxy}(phenoxy)phosphoryl]amโฆ EU/3/16/1615 |
Treatment of Ebola virus disease | Active (positive) | 17 Feb 2016 |
| tolfenamic acid EU/3/16/1614 |
treatment of behavioural variant frontotemporal dementia | Active (positive) | 17 Feb 2016 |
| tolfenamic acid EU/3/16/1613 |
Treatment of progressive supranuclear palsy | Active (positive) | 17 Feb 2016 |
| Methyl 3-((2R)-2-hydroxy-4-(((((S)-1-methoxy-1-oxopropan-2-yl) amino)(phenoxy)phosphoryl)oxy)-3,3-dimethylbutanamido)propanoate EU/3/16/1612 |
Treatment of pantothenate-kinase-associated neurodegeneration | Withdrawn | 17 Feb 2016 |
| ex-vivo-expanded autologous fibroblasts transduced with lentiviral vector containing the COL7A1 gene EU/3/16/1611 |
Treatment of epidermolysis bullosa | Active (positive) | 17 Feb 2016 |
| arsenic trioxide EU/3/16/1610 11 clinical trials → |
Treatment of acute myeloid leukaemia | Active (positive) | 17 Feb 2016 |
| S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-L-tryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-L-argiโฆ EU/3/16/1608 |
Treatment of C3 glomerulopathy | Active (positive) | 17 Feb 2016 |
| N-(4-Methoxyphenyl)-N,2,6-trimethylfuro[2,3-d]pyrimidin-4-amine EU/3/16/1618 |
Treatment of glioma | Active (positive) | 16 Feb 2016 |
| glibenclamide EU/3/15/1589 · now authorised as Amglidia |
Treatment of neonatal diabetes | Active (positive) | 15 Jan 2016 |
| Entolimod EU/3/15/1607 |
Treatment of acute radiation syndrome | Withdrawn | 11 Jan 2016 |
| (S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfateย (larotrectinib) EU/3/15/1606 |
Treatment of soft tissue sarcoma | Withdrawn | 11 Jan 2016 |
| synthetic double-stranded oligomer specific to the SERPINA1 gene and containing a cholesterol-conjugated, acyclic nucleobase analogue EU/3/15/1605 |
Treatment of congenital alpha-1 antitrypsin deficiency | Withdrawn | 11 Jan 2016 |
| two allogeneic irradiated pancreatic tumour cell lines EU/3/15/1604 |
Treatment of pancreatic cancer | Withdrawn | 11 Jan 2016 |
| Live attenuated Listeria monocytogenes delta actA/delta inlB strain expressing human mesothelin EU/3/15/1603 |
Treatment of pancreatic cancer | Withdrawn | 11 Jan 2016 |
| Live attenuated Listeria monocytogenes transfected with plasmids encoding the HPV-16E7 protein fused to a truncated fragment of the Lm protein listeriolysin O EU/3/15/1602 |
Treatment of anal cancer | Withdrawn | 11 Jan 2016 |
| sodium benzoate EU/3/15/1601 |
Treatment of argininosuccinic aciduria | Active (positive) | 11 Jan 2016 |
| sodium benzoate EU/3/15/1600 |
Treatment of hyperargininaemia | Active (positive) | 11 Jan 2016 |
| Adeno-associated viral vector serotype rh10 containing the human factor IX gene for the treatment of haemophilia B- EU/3/15/1599 |
Treatment of haemophilia B | Withdrawn | 14 Dec 2015 |
| 2-(2-Chlorobenzylidene)hydrazinecarboximidamide acetate EU/3/15/1598 |
Treatment of Charcot-Marie-Tooth disease | Active (positive) | 14 Dec 2015 |
| sodium (2R,3S,5R)-5-(4-amino-2-oxo-1,3,5-triazin-1(2H)-yl)-2-(hydroxymethyl)tetrahydrofuran-3-yl ((2R,3S,5R)-5-(2-amino-6-oxo-1H-purin-9(6H)-yl)-3-hydroxytetraโฆ EU/3/15/1597 |
Treatment of acute myeloid leukaemia | Withdrawn | 14 Dec 2015 |
| bilayer engineered collagen hydrogel-based skin graft composed of autologous keratinocytes and fibroblasts EU/3/15/1596 |
Treatment of partial deep dermal and full-thickness burns | Active (positive) | 14 Dec 2015 |
| live attenuated Listeria monocytogenes bioengineered with a chimeric human epidermal growth factor receptor 2 fused to a truncated form of the Lm protein listeโฆ EU/3/15/1595 |
Treatment of osteosarcoma | Active (positive) | 14 Dec 2015 |
| Live attenuated Listeria monocytogenes delta actA/delta inlB strain expressing human mesothelin EU/3/15/1594 |
Treatment of malignant mesothelioma | Withdrawn | 14 Dec 2015 |
| imetelstat sodium EU/3/15/1593 4 clinical trials → |
Treatment of myelofibrosis | Active (positive) | 14 Dec 2015 |
| -[4-Aminobutanoic acid-glycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutamyl-L-alanyl-L-lysโฆ EU/3/15/1592 |
Treatment of pseudohypoaldosteronism 1B | Active (positive) | 14 Dec 2015 |
| synthetic peptide L-cysteine, L-cysteinylglycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutaโฆ EU/3/15/1591 |
Treatment of pseudohypoaldosteronism 1B | Active (positive) | 14 Dec 2015 |
| recombinant human monoclonal IgG1 antibody against programmed death ligand-1 (avelumab) EU/3/15/1590 |
Treatment of Merkel cell carcinoma | Withdrawn | 14 Dec 2015 |
| (R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea (ceclazepide) EU/3/15/1588 |
Treatment of gastro-entero-pancreatic neuroendocrine tumours | Withdrawn | 14 Dec 2015 |
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.