Latest marketing authorisations and orphan drug designations
List of the latest marketing authorisations and medicinal products designations
Detailed information on European orphan medicinal products designation applications is available on theEMA website. A full list of designated and authorised orphan medicinal products in Europe available at:ec.europa.eu.
Learn more about the Orphan designation process in Europe
Recent orphan drug designations
May 2015
Treatment of plasma cell myeloma
Reduced oxydised N-acetyl heparin
Treatment of ovarian cancer
{2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl]-(3H-benzo[f]azepin-4-yl)}-N,N-dipropylcarboxamide
Treatment of myasthenia gravis
Fusion proteins composed by a genetically modified cholera toxin subunit A1, peptides from the acetylcholine receptor alpha chain and a dimer of the D fragment from Staphylococcus aureus protein A
Treatment of non-infectious uveitis
Triamcinolone acetonide
Treatment of mucopolysaccharidosis IIIC (Sanfilippo C syndrome)
Adeno-associated viral vector serotype 9 containing the human HGSNAT gene
Treatment of Huntington’s disease
AASSGVSTPGSAGHDIITEQPRS
Treatment of perinatal asphyxia
Allopurinol sodium
Treatment of diffuse large B-cell lymphoma
Humanised anti-CD37 monoclonal antibody conjugated to maytansinoid DM1
Treatment of glucose transporter type-1 deficiency syndrome
Triheptanoin
Treatment of oculopharyngeal muscular dystrophy
Trehalose
Treatment of Huntington’s disease
5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride
Treatment of cystic fibrosis
2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl)acetamide
Treatment of thromboangiitis obliterans (Buerger's disease)
Adult human bone-marrow-derived, ex-vivo-expanded, pooled allogeneic mesenchymal stromal cells
April 2015
Treatment of retinitis pigmentosa
Recombinant human mesencephalic astrocyte-derived neurotrophic factor
Treatment of neuroblastoma
Sodium 2-hydroxylinoleate
Treatment of cystic fibrosis
Nitric oxide
Treatment of perinatal asphyxia
Xenon
Treatment of choroideremia
Adeno-associated viral vector serotype 5 containing the human CHM gene
Treatment of Huntington’s disease
Phenol, 4-[2-(aminomethyl)-4-thiazolyl]-2,6-bis(1,1-dimethylethyl)monohydrochloride
Treatment of Ebola virus disease
Rintatolimod
Prevention of graft rejection following solid organ transplantation
Recombinant monoclonal IgG1 antibody against T-cell immune response cDNA 7
Treatment of Duchenne muscular dystrophy
Rimeporide
Treatment of pancreatic cancer
Human reovirus type 3 Dearing strain
Treatment of tularaemia
1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride
Treatment of cryptococcosis
1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride
Treatment of Stargardt's disease
Ecothiophate iodide
Treatment of marginal zone lymphoma
Lenalidomide
Diagnosis of glioma
Fluciclovine (18F)
March 2015
Treatment of Wilson’s disease
Trientine tetrahydrochloride
Treatment of biliary tract cancer
5,10,15,20-tetrakis(2,6-difluoro-3-Nmethylsulfamoylphenyl)bacteriochlorin
Treatment of ovarian cancer
Human reovirus type 3 Dearing strain
Treatment of Gaucher disease
Adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene
Treatment of epidermolysis bullosa
Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with aCOL17A1-encoding retroviral vector
Treatment of epidermolysis bullosa
Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with aCOL7A1-encoding retroviral vector
Treatment of epidermolysis bullosa
Ex-vivo-expanded autologous human keratinocytes containing
epidermal stem cells transduced with a LAMB3-encoding retroviral vector
Treatment of systemic sclerosis
Autologous adipose tissue-derived stromal vascular fraction cells
Treatment of plasma cell myeloma
Melphalan flufenamide
Treatment of retinitis pigmentosa
Sodium 3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo[3,2-d][1,3,2]dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5Himidazo[1,2-a]purin-9-one
Treatment of hepatocellular carcinoma
Lenvatinib
Treatment of amyotrophic lateral sclerosis
Enoxacin
Treatment of ovarian cancer
Humanised anti-folate receptor 1 monoclonal antibody conjugated to maytansinoid DM4
Treatment of tenosynovial giant cell tumour, localised and diffuse type
[5-(5-chloro-1H-pyrrolo[2,3-b]pyridin-3-ylmethyl)-pyridin-2-yl]-(6-trifluoromethyl-pyridin-3-ylmethyl)-amine hydrochloride
Prevention of bronchopulmonary dysplasia
Recombinant human club cell 10 KDa protein
Treatment of graft-versus-host disease
Human plasma-derived alpha-1 proteinase inhibitor
Treatment of Netherton syndrome
6-ethoxy-7-methoxy-2-(2-methylsulfanylphenyl)-3,1-benzoxazin-4-one
Treatment of Huntington’s disease
Chimeric 2'-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA
Treatment of fragile X syndrome
Tideglusib
Treatment of Alport syndrome
5'-ASCSASTSCSASGSTSCSTSGSASUSASASGSCSTSA-3'
Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
Gallium (68Ga)-edotreotide
February 2015
Treatment of facioscapulohumeral muscular dystrophy
505 amino acid protein, corresponding to amino acids 2-506 of the wild type human histidyl-tRNA synthetase
Treatment of soft tissue sarcoma
Olaratumab
Treatment of progressive supranuclear palsy
N-(3-(4-(3-(diisobutylamino)propyl)piperazin-1-yl)propyl)-1Hbenzo[d]imidazol-2-amine disulphate salt
Treatment of acute myeloid leukaemia
Ulocuplumab
Treatment of narcolepsy
Mazindol
Treatment of glioma
Recombinant human glutamate oxaloacetate transaminase 1
Treatment of Ebola virus disease
Fibrinogen-coated albumin spheres
Treatment of sickle cell disease
5-hydroxymethyl-2-furfural
Treatment of cytomegalovirus infection following haematopoietic stem cell transplantation
Allogeneic CD4+ and CD8+ T lymphocytes ex vivo incubated with synthetic peptides of the viral antigens of cytomegalovirus, adenovirus and Epstein-Barr virus
Treatment of acute myeloid leukaemia
Alvocidib
Prevention of necrotising enterocolitis
“Lactobacillus reuteri”
Treatment of systemic sclerosis
Nitroglycerin
Treatment of ovarian cancer
Chimeric group B adenovirus (11p/3) with deletions in the E3 and E4 Regions
Treatment of sickle cell disease
Sevuparin sodium
Treatment of hereditary angioedema
3-[2-(4-carbamimidoyl-phenylcarbamoyl)-5-methoxy-4-vinyl-phenyl]-6-(cyclopropylmethyl-carbamoyl)-pyridine-2-carboxylic acid
January 2015
Treatment of congenital factor VII deficiency
Adeno-associated viral vector serotype 8 containing the human factor VII gene
Treatment of ovarian cancer
N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride
Treatment of Wolfram syndrome
Sodium valproate
Treatment of hypophosphatasia
Recombinant human alkaline phosphatase
Prevention of graft-versus-host disease
Allogeneic peripheral blood mononuclear cells induced to an early apoptotic state
Treatment of spinocerebellar ataxia
Ceftriaxone
Treatment of diffuse large B-cell lymphoma
Humanised Fc engineered monoclonal antibody against CD19
Treatment of plasma cell myeloma
Synthetic signal peptide of human mucin-1 (amino acids 1-21)
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Chimeric fusion protein of recombinant human alpha-Nacetylglucosaminidase and human insulin-like growth factor 2
Treatment of gastrointestinal stromal tumours
Ponatinib hydrochloride
Treatment of Aicardi-Goutières syndrome
Emtricitabine
Treatment of Aicardi-Goutières syndrome
Tenofovir disoproxil fumarate
Treatment of adult T-cell leukaemia/lymphoma
A lentiviral vector pseudotyped by the New-Jersey serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and p30II HTLV-1 proteins
Treatment of adult T-cell leukaemia/lymphoma
A lentiviral vector pseudotyped by the Indiana serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and p30II HTLV-1 proteins
Treatment of neuroblastoma
Chimeric monoclonal antibody to O-acetyl-GD2 antigen
Treatment of placental insufficiency
Adenoviral vector serotype 5 containing the vascular endothelial growth factor D isoform (preprocessed short form) from a CMV promoter
Treatment for calciphylaxis
Sodium thiosulfate
Treatment of acute myeloid leukaemia
Allogeneic, umbilical cord blood-derived, ex vivo-expanded, haematopoietic CD133+ cells / allogeneic, umbilical cord blood-derived, non-expanded, haematopoietic CD133- cells
Treatment of pancreatic cancer
Herpes simplex type 1 virus containing cellular B-myb gene as tumour specific promoter
Treatment of interstitial cystitis
Pentosan polysulfate sodium
Treatment of aspartylglucosaminuria
Recombinant human aspartylglucosaminidase
Treatment of malignant mesothelioma
Pegylated recombinant arginine deiminase
December 2014
Treatment of glioma
5,5'-(4-(trifluromethyl)benzylazanediyl)bis(methylene)diquinolin-8-ol
Treatment of epidermolysis bullosa
Allogeneic adipose-derived adult mesenchymal stem cells contained in a fibrin-based bioengineered dermis
Treatment of inborn errors of primary bile acid synthesis
Chenodeoxycholic acid
Treatment of non-infectious uveitis
Autologous collagen type II-specific regulatory T cells
Treatment of multiple system atrophy
1-(2-isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d]pyrimidin-4-one
Treatment of WHIM syndrome
Plerixafor
Treatment of beta-thalassaemia intermedia and major
Benserazide hydrochloride
Treatment of traumatic spinal cord injury
Riluzole
Treatment of neurotrophic keratitis
(1S,4R,5R,7S)-3,4-dibenzyl-2-oxo-6,8-dioxa-3-azabyciclo[3.2.1]octane-7-carboxylic acid-L-lysine
Treatment of amyotrophic lateral sclerosis
Edaravone
Treatment of malignant mesothelioma
Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor
Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Amikacin sulfate
Treatment of glioma
Adenovirus serotype 5 containing partial E1A deletion and an integrin-binding domain
Treatment of acute myeloid leukaemia
Allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells
Treatment of pancreatic cancer
Pegylated recombinant human hyaluronidase PH20
Treatment of diffuse large B cell lymphoma
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor
Treatment of Huntington’s disease
5-bromo-N-(prop-2-yn-1-yl)-2-(1H-1,2,4-triazol-1-yl)pyrimidine-4,6-diamine
Treatment of malignant mesothelioma
5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine
Treatment of hereditary haemorrhagic telangiectasia
Bevacizumab
Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)
Adeno-associated viral vector serotype 10 carrying the human Nsulfoglucosamine sulfohydrolase cDNA
Prevention of graft-versus-host disease
Allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media
Treatment of familial cerebral cavernous malformations
Exisulind
Treatment of ovarian cancer
2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one
Treatment of pancreatic cancer
Heat-killed Mycobacterium obuense (whole cell)
Treatment of WHIM syndrome
((E)-1-(4'-chlorophenyl)-3-(4-hydroxy-3-metoxyphenyl)prop-2-en-1-one)
Treatment of pleural empyema
Single-chain urokinase plasminogen activator
Treatment of acute lymphoblastic leukaemia
Allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonist
November 2014
Treatment of Duchenne muscular dystrophy
Adeno-associated viral vector serotype 8 containing the human MD1 gene
Treatment of mucopolysaccharidosis type I
Ataluren
Treatment of malignant hyperthermia
Dantrolene sodium
Treatment of hepatocellular carcinoma
Diaspirin cross-linked haemoglobin
Treatment of Niemann-Pick disease, type C
Arimoclomol citrate
Treatment of xeroderma pigmentosum
Pro-Pro-Thr-Val-Pro-Thr-Arg
Treatment of Gaucher disease
(3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate
Treatment of granulomatosis with polyangiitis
(2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide
Treatment of microscopic polyangiitis
(2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide
Treatment of systemic sclerosis
Humanised IgG1 monoclonal antibody against human eotaxin-2
Treatment of polymyositis
Siponimod
Treatment of dermatomyositis
Siponimod
Treatment of fibrodysplasia ossificans progressiva
Palovarotene
Treatment of hereditary haemorrhagic telangiectasia
Bazedoxifene acetate
Treatment of primary myelofibrosis
Recombinant human pentraxin-2
Treatment of post-polycythaemia vera myelofibrosis
Recombinant human pentraxin-2
Treatment of glioma
Olaptesed pegol
Treatment of idiopathic pulmonary fibrosis
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid
Treatment of systemic sclerosis
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid
Treatment of haemophilia A
A combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH
Treatment of mucopolysaccharidosis type I
Pentosan polysulfate sodium
Treatment of post-essential thrombocythaemia myelofibrosis
Recombinant human pentraxin-2
Treatment of acute respiratory distress syndrome
Imatinib
Treatment of acute myeloid leukaemia
Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment
Treatment of plasma cell myeloma
Selinexor
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Selinexor
Treatment of myotonic disorders
Mexiletine hydrochloride
October 2014
Treatment of inhalation anthrax disease
Raxibacumab
Treatment of X-linked hypophosphataemia
Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23
Treatment of tenosynovial giant cell tumour, localised and diffused type
Recombinant human monoclonal antibody of the IgG1 kappa class against human macrophage colony-stimulating factor
Treatment of mucopolysaccharidosis type I
Recombinant human insulin receptor monoclonal antibody-fused-α-Liduronidase
Treatment of glioma
Recombinant human bone morphogenetic protein 4
Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Pyridoxal 5'-phosphate
Treatment of short bowel syndrome
Oxalobacter formigenes strain HC-1
Treatment of Cushing's syndrome
Osilodrostat
Treatment of cystic fibrosis
Nitric oxide
Treatment of pancreatic cancer
Immunoglobulin G1, anti-(human tumour-associated calcium signaltransducer 2)(human-Mus musculus monoclonal hRS7 heavy chain), disulfide with human-Musmusculus monoclonal hRS7 k-chain, dimer, hexakis(thioether) with (4S)-4-[[[[4-[[(2S)-2-(4-aminobutyl)-2-[[2-[2-[[26-[4-[[[[4-[(3-mercapto-2,5-dioxo-1-pyrrolidinyl)methyl]cyclohexyl]carbonyl]amino]methyl]-1H-1,2,3-triazol-1-yl]-3,6,9,12,15,18,21,24-octaoxahexacos-1-yl]amino]-2-oxoethoxy]acetyl]amino]-1-oxoethyl]amino]phenyl]methoxy]carbonyl]oxy]-4,11-diethyl-9-hydroxy-1Hpyrano[3',4':6,7]indolizino[1,2-b]quinoline-3,14(4H,12H)-dione
Treatment of congenital hyperinsulinism
Glucagon
Treatment of cystinosis
Cysteamine hydrochloride
Treatment of limbal stem cell deficiency
Cultured allogeneic corneal limbal stem cells
Treatment of Dravet syndrome
Cannabidiol
Treatment of Crigler-Najjar syndrome
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene
Treatment of fragile X syndrome
Acamprosate calcium
Treatment of Leigh syndrome
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride
Treatment of pemphigus
(S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl-3,4-dihydropyrrolo[2,1-f][1,2,4]triazine-5-carbonitrile
August 2014
Treatment of cystic fibrosis
Lumacaftor / ivacaftor
Prevention of ischaemia reperfusion injury associated with solid organ transplantation
Macromolecular conjugate of heparin sodium on a polymer backbone
Treatment of myelodysplastic syndromes
Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain
Treatment of primary biliary cirrhosis
Vector lentiviral que conteniene el gen piruvato quinasa humano de hígado y eritroide (PKLR)
Treatment of acute myeloid leukaemia
4-{[(2R,3S,4R,5S)-4-(4-chloro-2-fluoro-phenyl)-3-(3-chloro-2-fluorophenyl)-4-cyano-5-(2,2-dimethyl-propyl)-pyrrolidine-2-carbonyl]-amino}-3-methoxy-benzoic acid
Treatment of paroxysmal nocturnal haemoglobinuria
S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-Ltryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-Larginyl-L-cysteinyl-N-methyl-L-isoleucinamide)
Treatment of mitochondrial neurogastrointestinal encephalomyopathy
Vector based on an adeno-associated virus serotype 2 backbone, pseudoserotyped with a type 8 capsid, which carries the coding sequence of the human TYMP gene under the control of the human thyroxine binding globulin promoter
Treatment of diffuse large B-cell lymphoma
Obinutuzumab
Treatment of small cell lung cancer
2-(2-methyl-5-nitro-1H-imidazol-1-yl)ethylsulfamide
Treatment of pancreatic cancer
[5-amino-1-(4-fluoro-phenyl)-1H-pyrazol-4-yl]-[3-(2,3-dihydroxypropoxy)-phenyl]-methanone
Treatment of cutaneous T-cell lymphoma
Humanised IgG1 monoclonal antibody against human KIR3DL2
Treatment of Crigler-Najjar syndrome
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene
Treatment of mastocytosis
Recombinant human diamine oxidase
Treatment of haemophilia B
Recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin
Treatment of acute pancreatitis
Ulinastatin
Treatment of diffuse large B-cell lymphoma
(Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide
Treatment of haemophilia A
Recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin
Treatment of apolipoprotein A-I deficiency
Recombinant human apolipoprotein A-I in a complex with phospholipids
Treatment of ATP-binding cassette transporter A1 deficiency
Recombinant human apolipoprotein A-I in a complex with phospholipids
Treatment of acute myeloid leukaemia
(Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide
Treatment of congenital factor VII deficiency
Recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin
Treatment of Schnitzler syndrome
Gevokizumab
Treatment of Fabry disease
(3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate
Treatment of Duchenne muscular dystrophy
17α,21-dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione
Treatment of autosomal dominant polycystic liver disease
Sodium ascorbate and menadione sodium bisulfite
Prevention of bronchopulmonary dysplasia
Retinol
July 2014
Treatment of Huntington’s disease
Cysteamine bitartrate
Treatment of glioma
Humanised recombinant monoclonal antibody against epidermal growth factor receptor conjugated to maleimidocaproyl monomethylauristatin F
Treatment of myasthenia gravis
Eculizumab
Treatment of ovarian cancer
Cediranib
Treatment of Prader-Willi syndrome
Oxytocin
Treatment of idiopathic pulmonary fibrosis
Humanised anti-alpha ν beta 6 monoclonal antibody
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