Every medicine the European Commission has designated as an orphan medicine for a rare disease, on the EMA's recommendation, with its current status.
The EMA lists 3,310 EU orphan designations, 2,205 of them currently active, and 125 were granted in the last 12 months. An orphan designation is for a medicine intended to diagnose, prevent or treat a life-threatening or chronically debilitating condition affecting no more than 5 in 10,000 people in the EU. It gives incentives such as fee reductions and, once the medicine is authorised, market exclusivity. It is not a marketing authorisation.
| Substance | Intended use | Status | Designated |
|---|---|---|---|
| autologous CD34+ haematopoietic stem and progenitor cells transduced with a lentiviral vector containing the human DCLRE1C gene EU/3/26/3274 |
Treatment of severe combined immunodeficiency (SCID) due to DCLRE1C (Artemis) deficiency | Active (positive) | 28 Jul 2026 |
| exaluren sulfate EU/3/26/3273 |
Treatment of autosomal dominant polycystic kidney disease | Active (positive) | 28 Jul 2026 |
| cisatracurium besilate EU/3/26/3272 3 clinical trials → |
Treatment of osteogenesis imperfecta | Active (positive) | 28 Jul 2026 |
| Lyophilized consortium of Faecalibacterium sp., strain PD09; Anaerostipes sp., strain PD10; Akkermansia sp., strain PD11; Butyricicoccus sp., strain PD12; Rose… EU/3/26/3271 |
Treatment of pouchitis | Active (positive) | 28 Jul 2026 |
| single stranded RNA editing oligonucleotide against GLUL mRNA EU/3/26/3270 |
Treatment of urea cycle disorders | Active (positive) | 28 Jul 2026 |
| 1-({(5S,7S,8R)-8-Fluoro-3-[5-(2-hydroxypropan-2-yl)pyrazin-2-yl]-7-methyl-2-oxo-1-oxa-3-azaspiro[4.5]decan-7-yl}methyl)-1H-1,3-benzimidazole-6-carbonitrile EU/3/26/3269 |
Treatment of TRPV4-related neuromuscular disease | Active (positive) | 28 Jul 2026 |
| sonesitatug vedotin EU/3/26/3268 6 clinical trials → |
Treatment of gastric cancer | Active (positive) | 28 Jul 2026 |
| allogeneic human induced pluripotent stem cell-derived photoreceptor precursor cells EU/3/26/3267 |
Treatment of non-syndromic inherited retinal dystrophies of the cone-dominant phenotype | Active (positive) | 28 Jul 2026 |
| guide RNA against the human SERPINA1 gene, mRNA encoding a CRISPR-associated endonuclease Cas9 against the human SERPINA1 gene EU/3/26/3266 |
Treatment of alpha-1 antitrypsin deficiency | Active (positive) | 28 Jul 2026 |
| lead (212Pb) bamzireotide navoxetan EU/3/26/3265 |
Treatment of gastro-entero-pancreatic neuroendocrine tumours | Active (positive) | 28 Jul 2026 |
| (2R)-2-[[6-[Ethyl(phenylmethyl)amino]-9-(1-methylethyl)-9H-purin-2-yl]amino]butyl L-valine ester hydrochloride (1:1) EU/3/26/3264 |
Treatment of neonatal encephalopathy | Active (positive) | 28 Jul 2026 |
| olorofim EU/3/26/3263 |
Treatment of coccidioidomycosis | Active (positive) | 28 Jul 2026 |
| selcodebart EU/3/26/3262 |
Treatment of myelofibrosis | Active (positive) | 28 Jul 2026 |
| serabelisib EU/3/26/3261 2 clinical trials → |
Treatment of venous malformations | Active (positive) | 28 Jul 2026 |
| serabelisib EU/3/26/3260 2 clinical trials → |
Treatment of lymphatic malformations | Active (positive) | 28 Jul 2026 |
| rozanolixizumab EU/3/26/3259 6 clinical trials → |
Treatment of myelin oligodendrocyte glycoprotein antibody-associated disease | Active (positive) | 28 Jul 2026 |
| cabazitaxel EU/3/26/3258 11 clinical trials → |
Treatment of ovarian cancer | Active (positive) | 28 Jul 2026 |
| modified mRNA encoding human cystic fibrosis transmembrane conductance regulator EU/3/26/3256 |
Treatment of cystic fibrosis | Active (positive) | 23 Jun 2026 |
| short hairpin RNA against NANOG mRNA EU/3/26/3252 |
Treatment of glioma | Active (positive) | 23 Jun 2026 |
| dinutuximab beta EU/3/26/3251 4 clinical trials → |
Treatment of Ewing sarcoma | Active (positive) | 23 Jun 2026 |
| guide RNA against the human HAO1 gene, mRNA encoding an engineered variant of the Cas12 nuclease EU/3/26/3257 |
Treatment of primary hyperoxaluria | Active (positive) | 22 Jun 2026 |
| adeno-associated viral vector serotype 8 containing the human CASQ2 gene EU/3/26/3255 |
Treatment of catecholaminergic polymorphic ventricular tachycardia | Active (positive) | 22 Jun 2026 |
| 3-(Aminocarbonyl)-1-(2,3,5-tri-O-acetyl-beta-D-ribofuranosyl)-pyridinium chloride (1:1) EU/3/26/3254 |
Treatment of ataxia telangiectasia | Active (positive) | 22 Jun 2026 |
| cyclo(arginyl-glycyl-aspartyl-D-tyrosyl-lysyl)-nizaracianine-1 EU/3/26/3253 |
Treatment of head and neck cancer | Active (positive) | 22 Jun 2026 |
| herpes simplex virus, type 1, containing the human NGF gene EU/3/26/3250 |
Treatment of neurotrophic keratitis | Active (positive) | 22 Jun 2026 |
| satralizumab EU/3/26/3249 8 clinical trials → |
Treatment of myelin oligodendrocyte glycoprotein antibody-associated disease | Active (positive) | 22 Jun 2026 |
| (S)-Quinuclidin-3-yl((R)-5-(3-chloro-4-isopropoxyphenyl)-2,2-dimethyl-2,3-dihydro-1H-inden-1-yl)carbamate ((1S,4R)-7,7-dimethyl-2-oxobicyclo[2.2.1]heptan-1-yl)… EU/3/26/3248 |
Treatment of Gaucher disease | Active (positive) | 22 Jun 2026 |
| humanised IgG4 monoclonal antibody against activated protein C EU/3/26/3247 |
Treatment of factor VII deficiency | Active (positive) | 22 Jun 2026 |
| dasatinib EU/3/26/3246 26 clinical trials → |
Treatment of Diamond-Blackfan anaemia | Active (positive) | 22 Jun 2026 |
| human IgG1 monoclonal antibody against JC polyomavirus, major capsid protein VP1 EU/3/26/3245 |
Treatment of progressive multifocal leukoencephalopathy | Active (positive) | 22 Jun 2026 |
| (S)-N-(2-Methoxy-5-((5-(trifluoromethyl)pyridin-2-yl)oxy)phenyl)-3-methyl-2-oxoimidazolidine-4-carboxamide EU/3/26/3244 |
Treatment of malignant mesothelioma | Active (positive) | 22 Jun 2026 |
| allogeneic human induced pluripotent stem cell-derived photoreceptor precursor cells EU/3/26/3243 |
Treatment of syndromic inherited retinal dystrophies of the rod-dominant phenotype | Active (positive) | 20 May 2026 |
| allogeneic peripheral blood mononuclear cells incubated in vitro with mitomycin C EU/3/26/3242 |
Treatment of in solid organ transplantation | Active (positive) | 20 May 2026 |
| quemliclustat EU/3/26/3241 3 clinical trials → |
Treatment of pancreatic cancer | Active (positive) | 20 May 2026 |
| igermetostat maleate EU/3/26/3240 |
Treatment of peripheral T-cell lymphoma | Active (positive) | 20 May 2026 |
| serabelisib EU/3/26/3239 2 clinical trials → |
Treatment of PIK3CA related overgrowth spectrum | Active (positive) | 20 May 2026 |
| allogeneic human induced pluripotent stem cell-derived photoreceptor precursor cells EU/3/26/3238 |
Treatment of non-syndromic inherited retinal dystrophies of the rod-dominant phenotype | Active (positive) | 20 May 2026 |
| allogeneic human Wharton's jelly-derived mesenchymal stem cells EU/3/26/3237 |
Treatment of Charcot-Marie-Tooth disease | Active (positive) | 20 May 2026 |
| infliximab EU/3/26/3236 39 clinical trials → |
Treatment of Kawasaki disease | Active (positive) | 20 May 2026 |
| antisense oligonucleotide against USH2A pre-mRNA EU/3/26/3235 |
Treatment of inherited retinal disease due to dysfunction in the USH2A gene | Active (positive) | 20 May 2026 |
| humanised IgG1 monoclonal antibody against delta-like ligand 3, octakis(thioether) with N-[6-(2-mercapto-5-pyrimidinyl)-1-oxo-5-hexyn-1-yl]-L-valyl-N6,N6-dipro… EU/3/26/3234 |
Treatment of pulmonary neuroendocrine carcinoma | Active (positive) | 20 May 2026 |
| (S)-N-(2-(2-Cyclopropylpyridin-4-yl)-4,5,6,7-tetrahydrobenzo[d]oxazol-4-yl)-1-methyl-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide EU/3/26/3233 |
Treatment of KCNT1-related epilepsy | Active (positive) | 20 May 2026 |
| copper metabolism disorder EU/3/26/3232 |
Treatment of Wilson's disease | Active (positive) | 20 May 2026 |
| alixorexton EU/3/26/3231 3 clinical trials → |
Treatment of narcolepsy | Active (positive) | 20 May 2026 |
| peptide-oligonucleotide conjugate against CNOT3 mRNA EU/3/26/3230 |
Treatment of inherited retinal dystrophies due to mutation in the PRPF31 gene | Active (positive) | 20 May 2026 |
| antisense oligonucleotide against SYNGAP1 regulatory RNA EU/3/26/3229 |
Treatment of SYNGAP1-related disorder | Active (positive) | 20 Apr 2026 |
| coagulation factor V EU/3/26/3228 |
Treatment of congenital factor V deficiency | Active (positive) | 20 Apr 2026 |
| daraxonrasib EU/3/26/3227 |
Treatment of pancreatic cancer | Active (positive) | 20 Apr 2026 |
| adeno-associated virus serotype SLB101 containing the human microdystrophin gene EU/3/26/3226 |
Treatment of Duchenne muscular dystrophy | Active (positive) | 20 Apr 2026 |
| aganirsen EU/3/26/3225 |
Prevention of corneal graft rejection | Active (positive) | 20 Apr 2026 |
| surovatamig EU/3/26/3224 9 clinical trials → |
Treatment of large B-cell lymphoma | Active (positive) | 20 Apr 2026 |
| Momelotinib dihydrochloride monohydrate EU/3/26/3223 |
Treatment of VEXAS syndrome | Active (positive) | 20 Apr 2026 |
| 3-(4-((7-Cyano-1-methyl-2-((1-methyl-2-oxo-5-(trifluoromethyl)-1,2-dihydropyridin-3-yl)amino)-1H-imidazo[4,5-b]pyridin-6-yl)oxy)pyridin-2-yl)-1,1-dimethylurea EU/3/26/3222 |
Treatment of myelofibrosis | Active (positive) | 20 Apr 2026 |
| oremepermin alfa EU/3/26/3221 |
Treatment of spinal cord injury | Active (positive) | 20 Apr 2026 |
| upadacitinib EU/3/26/3220 40 clinical trials → |
Treatment of systemic sclerosis | Active (positive) | 20 Apr 2026 |
| lisaftoclax EU/3/26/3218 21 clinical trials → |
Treatment of myelodysplastic syndromes | Active (positive) | 13 Apr 2026 |
| dusquetide EU/3/26/3219 |
Treatment of Behçet's Disease | Active (positive) | 25 Mar 2026 |
| irpagratinib tosilate monohydrate EU/3/26/3217 |
Treatment of hepatocellular carcinoma | Active (positive) | 25 Mar 2026 |
| exaluren sulfate EU/3/26/3216 |
Treatment of Alport syndrome | Active (positive) | 25 Mar 2026 |
| atigotatug;nivolumab EU/3/26/3215 268 clinical trials → |
Treatment of pulmonary neuroendocrine carcinoma | Active (positive) | 25 Mar 2026 |
| acetylleucine EU/3/26/3214 |
Treatment of CACNA1A disorders | Active (positive) | 25 Mar 2026 |
| telisotuzumab adizutecan EU/3/26/3213 16 clinical trials → |
Treatment of pancreatic cancer | Active (positive) | 25 Mar 2026 |
| dibotatug EU/3/26/3212 3 clinical trials → |
Treatment of large granular lymphocytic leukaemia | Active (positive) | 25 Mar 2026 |
| hydroxocobalamin acetate EU/3/26/3211 |
Treatment of homocystinuria and/or methylmalonic acidaemia due to genetic defects of intracellular cobalamin processing | Active (positive) | 25 Mar 2026 |
| tacrolimus monohydrate EU/3/26/3210 40 clinical trials → |
Treatment of vernal keratoconjunctivitis | Active (positive) | 25 Mar 2026 |
| humanised IgG1 monoclonal antibody against SEZ6, conjugated to (2S)-2-(2-bromoacetamido)-N-[(2S)-1-({3-[(7S)-7-ethyl-7-hydroxy-8,11-dioxo-7,8,11,13-tetrahydro-… EU/3/26/3209 |
Treatment of pulmonary neuroendocrine carcinoma | Active (positive) | 25 Mar 2026 |
| adeno-associated viral vector serotype 44.9 containing the human HRS1 gene EU/3/26/3208 |
Treatment of inherited retinal dystrophy due to dysfunction in the human retinoschisin (RS1) gene | Active (positive) | 23 Feb 2026 |
| non-replicating adeno-associated virus serotype 5 containing the human retinal guanylate cyclase 1 gene EU/3/26/3207 |
Treatment of inherited retinal dystrophy due to biallelic autosomal recessive mutations in the human guanylate cyclase 1 (GUCY2D) gene | Active (positive) | 23 Feb 2026 |
| povetacicept EU/3/26/3205 4 clinical trials → |
Treatment of primary membranous nephropathy | Active (positive) | 23 Feb 2026 |
| N-[(1S)-1-(4-Tert-butylphenyl) ethyl]-2-(6,7-difluoro-1H-benzimidazol-1-yl)acetamide EU/3/26/3204 |
Treatment of erythromelalgia | Active (positive) | 23 Feb 2026 |
| spironolactone EU/3/26/3203 15 clinical trials → |
Treatment of limbal stem cell deficiency | Active (positive) | 23 Feb 2026 |
| Measles virus Schwarz strain (live, attenuated) EU/3/26/3201 |
Treatment of mesothelioma | Active (positive) | 23 Feb 2026 |
| aviptadil EU/3/26/3200 |
Treatment of acute respiratory distress syndrome (ARDS) | Active (positive) | 23 Feb 2026 |
| (4Z,7R,8E,10E,12Z,14S,16Z,19Z)-7,14-Dihydroxydocosa-4,8,10,12,16,19-hexaenoic acid EU/3/26/3197 |
Treatment of spinal cord injury | Active (positive) | 23 Feb 2026 |
| Humanised IgG1 kappa monoclonal antibody against erythropoietin isoform EV-3 EU/3/26/3196 |
Treatment of glioma | Active (positive) | 23 Feb 2026 |
| sefaxersen EU/3/26/3195 |
Treatment of primary IgA nephropathy | Active (positive) | 23 Feb 2026 |
| saracatinib EU/3/25/3194 |
Treatment of fibrodysplasia ossificans progressiva | Active (positive) | 9 Jan 2026 |
| spironolactone EU/3/25/3193 15 clinical trials → |
Treatment of central serous chorioretinopathy | Active (positive) | 9 Jan 2026 |
| opamtistomig EU/3/25/3192 |
Treatment of extrapulmonary neuroendocrine carcinoma | Active (positive) | 9 Jan 2026 |
| Adeno-associated virus serotype ATC-0187 vector containing the human BAG3 gene EU/3/25/3191 |
Treatment of BAG3 dilated cardiomyopathy | Active (positive) | 9 Jan 2026 |
| domatinostat EU/3/25/3190 |
Treatment of osteosarcoma | Active (positive) | 9 Jan 2026 |
| sirolimus EU/3/25/3189 32 clinical trials → |
Treatment of osteosarcoma | Active (positive) | 9 Jan 2026 |
| Phe-Thr-Thr-Phe-Thr-Val-Thr EU/3/25/3188 |
Treatment of idiopathic pulmonary fibrosis | Active (positive) | 9 Jan 2026 |
| 2-[[(4-Methoxy-3,5-dimethyl-2-pyridinyl)methyl]sulfinyl]-1H-benzimidazol-5-ol EU/3/25/3187 |
Treatment of multiple system atrophy | Active (positive) | 9 Jan 2026 |
| N-[5-(2H-1,3-Benzodioxol-5-yl)-6-{2-[(5-bromopyrimidin-2-yl)oxy]ethoxy}pyrimidin-4-yl]-N'-(2-methoxyethyl)sulfuric diamide EU/3/25/3186 |
Treatment of primary IgA nephropathy | Active (positive) | 9 Jan 2026 |
| human IgG1 kappa monoclonal antibody against pappalysin-1 EU/3/25/3185 |
Treatment of autosomal dominant polycystic kidney disease | Active (positive) | 9 Jan 2026 |
| surovatamig EU/3/25/3184 9 clinical trials → |
Treatment of follicular lymphoma | Active (positive) | 9 Jan 2026 |
| deupirfenidone EU/3/25/3183 2 clinical trials → |
Treatment idiopathic pulmonary fibrosis | Active (positive) | 9 Jan 2026 |
| canvuparatide EU/3/25/3182 |
Treatment of hypoparathyroidism | Active (positive) | 9 Jan 2026 |
| autologous human T cells transduced with a lentiviral vector encoding a chimeric antigen receptor against the ERBB family EU/3/25/3181 |
Treatment of Fanconi anaemia | Active (positive) | 9 Jan 2026 |
| aminolevulinic acid hydrochloride EU/3/25/3180 |
Diagnosis of ovarian cancer | Active (positive) | 9 Jan 2026 |
| lanthanum carbonate octahydrate EU/3/25/3179 |
Treatment of primary hyperoxaluria | Active (positive) | 9 Jan 2026 |
| navtemadlin EU/3/25/3178 5 clinical trials → |
Treatment of myelofibrosis | Active (positive) | 9 Jan 2026 |
| potravitug EU/3/25/3176 |
Treatment in solid organ transplantation | Active (positive) | 15 Dec 2025 |
| rezatapopt EU/3/25/3177 2 clinical trials → |
Treatment of ovarian cancer | Active (positive) | 9 Dec 2025 |
| amsulostat EU/3/25/3174 |
Treatment of myelofibrosis | Active (positive) | 9 Dec 2025 |
| efdoralprin alfa EU/3/25/3170 |
Treatment of alpha-1 antitrypsin deficiency | Active (positive) | 9 Dec 2025 |
| N-[[(2S)-4-[(4-Methyl-1H-imidazol-5-yl)methyl]-3-oxo-2-(phenylmethyl)-1-piperazinyl]carbonyl]-L-leucine trihydrate EU/3/25/3169 |
Treatment of cutaneous T-cell lymphoma | Active (positive) | 9 Dec 2025 |
| [4-(6-aminopyridazin-3-yl)piperidin-1-yl][5-(4-fluorophenoxy)-4-methoxypyridin-2-yl]methanone EU/3/25/3166 |
Treatment of focal segmental glomerulosclerosis | Active (positive) | 9 Dec 2025 |
| domvanalimab EU/3/25/3162 11 clinical trials → |
Treatment of gastric cancer | Active (positive) | 9 Dec 2025 |
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.