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
The Voice of Rare Disease
Patients in Europe
Patients in Europe
Recent orphan drug designations
August 2015
Treatment of acute lymphoblastic leukaemia
Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2
Allogeneic umbilical cord blood cells treated ex vivo with 16,16-dimethyl prostaglandin E2
Treatment of acute myeloid leukaemia
CD33-directed antibody-drug conjugate consisting of an antibody conjugated to a DNA cross-linking pyrrolobenzodiazepine dimer drug
CD33-directed antibody-drug conjugate consisting of an antibody conjugated to a DNA cross-linking pyrrolobenzodiazepine dimer drug
Treatment of hepatocellular carcinoma
2-(2-phenylvinyl)-4-[4-methylpiperazin-1-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt
2-(2-phenylvinyl)-4-[4-methylpiperazin-1-yl)]-6-(5-methyl-2H-pyrazol-3-yl-amino)-pyrimidine L(+) tartrate salt
Treatment of mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride
Treatment of diffuse large B-cell lymphoma
2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxythymidylyl-(3',5'-phosphoryl)- 2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxycytidilyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxythymidylyl-(3',5'-phosphoryl)-2'-deoxyadenosyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxyguanosylyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxyadenosyl-(3',5'-phosphoryl)-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxyguanosylyl-2'-deoxycytidylyl-(3',5'-phosphoryl)-2'-deoxyadenosyl-(3',5'-phosphoryl)-2'-deoxycytidine, sodium salt
Treatment of marginal zone lymphoma
Ibrutinib
Ibrutinib
Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene
Adeno-associated viral vector serotype 9 containing the human iduronate-2-sulfatase gene
Treatment of X-linked myotubular myopathy
Adeno-associated viral vector serotype 8 containing the human MTM1gene
Adeno-associated viral vector serotype 8 containing the human MTM1gene
Treatment of cystic fibrosis
Fixed-dose combination of fosfomycin disodium and tobramycin
Fixed-dose combination of fosfomycin disodium and tobramycin
Treatment of congenital adrenal hyperplasia
Verucerfont
Verucerfont
Treatment of cystic fibrosis
Recombinant human acid ceramidase
Recombinant human acid ceramidase
Treatment of acute radiation syndrome
Fibrinogen-coated albumin spheres
Fibrinogen-coated albumin spheres
Treatment of Rett syndrome
Glycyl-L-2-methylprolyl-L-glutamic acid
Glycyl-L-2-methylprolyl-L-glutamic acid
Treatment of osteogenesis imperfecta
Human allogeneic bone-marrow-derived osteoblastic cells
Human allogeneic bone-marrow-derived osteoblastic cells
Treatment of short bowel syndrome
Insulin human
Insulin human
July 2015
Treatment of Rett syndrome
Sarizotan hydrochloride
Sarizotan hydrochloride
Treatment of craniopharyngioma
Beloranib
Beloranib
Treatment of fragile X syndrome
Glycyl-L-2-methylprolyl-L-glutamic acid
Glycyl-L-2-methylprolyl-L-glutamic acid
Treatment of primary hyperoxaluria type 1
Synthetic double-stranded RNA oligonucleotide specific to hydroxyacid oxidase 1 gene
Synthetic double-stranded RNA oligonucleotide specific to hydroxyacid oxidase 1 gene
Treatment of amyotrophic lateral sclerosis
Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg]acetate
Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg]acetate
Treatment of carnitine palmitoyltransferase II deficiency
Triheptanoin
Triheptanoin
Treatment of mitochondrial trifunctional protein deficiency
Triheptanoin
Triheptanoin
Treatment of long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency
Triheptanoin
Triheptanoin
Treatment of acute myeloid leukaemia
Inecalcitol
Inecalcitol
Treatment of progressive supranuclear palsy
Humanised IgG4 monoclonal antibody against extracellular tau
Humanised IgG4 monoclonal antibody against extracellular tau
Treatment of malaria
Artesunate
Artesunate
Treatment of perinatal asphyxia
Cannabidiol
Cannabidiol
Treatment of Duchenne muscular dystrophy
Allogeneic human adult stem cells, isolated from skeletal muscle and expanded ex vivo
Allogeneic human adult stem cells, isolated from skeletal muscle and expanded ex vivo
Treatment of retinitis pigmentosa
Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene
Adenovirus-associated viral vector serotype 2 containing the human RPE65 gene
Treatment of acute myeloid leukaemia
2-((3-((4-((3-aminopropyl)amino)butyl)amino)propyl)amino)-N-((5S,5aS,8aR,9R)-9-(4-hydroxy-3,5-dimethoxyphenyl)-8-oxo-5,5a,6,8,8a,9-hexahydrofuro[3',4':6,7]naphtho[2,3-d][1,3]dioxol-5-yl)acetamide, tetrahydrochloride
2-((3-((4-((3-aminopropyl)amino)butyl)amino)propyl)amino)-N-((5S,5aS,8aR,9R)-9-(4-hydroxy-3,5-dimethoxyphenyl)-8-oxo-5,5a,6,8,8a,9-hexahydrofuro[3',4':6,7]naphtho[2,3-d][1,3]dioxol-5-yl)acetamide, tetrahydrochloride
Treatment of pancreatic cancer
Modified adenovirus serotype 5/35 containing a CMV promoter-driven transgene cassette with the human transgenes for a membrane-bound CD40 ligand and full length 4-1BBL
Modified adenovirus serotype 5/35 containing a CMV promoter-driven transgene cassette with the human transgenes for a membrane-bound CD40 ligand and full length 4-1BBL
Treatment of cutaneous T-cell lymphoma
Synthetic hypericin
Synthetic hypericin
Treatment of autosomal dominant polycystic kidney disease
Lanreotide acetate
Lanreotide acetate
Treatment of hepatoblastoma
Doxorubicin
Doxorubicin
Treatment of avian influenza
Anti-H5N1 equine immunoglobulin F(ab')2 fragments
Anti-H5N1 equine immunoglobulin F(ab')2 fragments
Treatment of plasminogen deficiency
Human plasminogen
Human plasminogen
June 2015
Treatment of amyotrophic lateral sclerosis
Edaravone
Edaravone
Treatment of spinal muscular atrophy
Adeno-associated viral vector serotype 9 containing the human SMN Gene
Adeno-associated viral vector serotype 9 containing the human SMN Gene
Treatment of very long-chain acyl-CoA dehydrogenase deficiency
Triheptanoin
Triheptanoin
Treatment of non-infectious uveitis
3-{[2,3,5,6-tetrafluoro-3'-(trifluoromethoxy)biphenyl-4-yl]carbamoyl}thiophene-2-carboxylic acid
3-{[2,3,5,6-tetrafluoro-3'-(trifluoromethoxy)biphenyl-4-yl]carbamoyl}thiophene-2-carboxylic acid
Prevention of scarring post glaucoma filtration surgery
Antisense oligonucleotide directed against TGF-β2 mRNA
Antisense oligonucleotide directed against TGF-β2 mRNA
Treatment of marginal zone lymphoma
Obinutuzumab
Obinutuzumab
Treatment of follicular lymphoma
Obinutuzumab
Obinutuzumab
Prevention of bronchopulmonary dysplasia
Allogeneic ex-vivo-expanded human umbilical cord blood-derived mesenchymal stem cells
Allogeneic ex-vivo-expanded human umbilical cord blood-derived mesenchymal stem cells
Treatment of spinocerebellar ataxia
Trehalose
Trehalose
Treatment of haemophilia B
Adeno-associated viral vector containing the human factor IX gene
Adeno-associated viral vector containing the human factor IX gene
Treatment of hepatitis delta virus infection
Synthetic 47-amino-acid N-myristoylated lipopeptide, derived from the preS region of hepatitis B virus
Synthetic 47-amino-acid N-myristoylated lipopeptide, derived from the preS region of hepatitis B virus
May 2015
Treatment of plasma cell myeloma
Reduced oxydised N-acetyl heparin
Reduced oxydised N-acetyl heparin
Treatment of ovarian cancer
{2-amino-8-[4-(pyrrolidinylcarbonyl)phenyl]-(3H-benzo[f]azepin-4-yl)}-N,N-dipropylcarboxamide
{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
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
Triamcinolone acetonide
Treatment of mucopolysaccharidosis IIIC (Sanfilippo C syndrome)
Adeno-associated viral vector serotype 9 containing the human HGSNAT gene
Adeno-associated viral vector serotype 9 containing the human HGSNAT gene
Treatment of Huntington’s disease
AASSGVSTPGSAGHDIITEQPRS
AASSGVSTPGSAGHDIITEQPRS
Treatment of perinatal asphyxia
Allopurinol sodium
Allopurinol sodium
Treatment of diffuse large B-cell lymphoma
Humanised anti-CD37 monoclonal antibody conjugated to maytansinoid DM1
Humanised anti-CD37 monoclonal antibody conjugated to maytansinoid DM1
Treatment of glucose transporter type-1 deficiency syndrome
Triheptanoin
Triheptanoin
Treatment of oculopharyngeal muscular dystrophy
Trehalose
Trehalose
Treatment of Huntington’s disease
5,7-dichloro-2-dimethylaminomethyl-8-hydroxyquinoline hydrochloride
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
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
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
Recombinant human mesencephalic astrocyte-derived neurotrophic factor
Treatment of neuroblastoma
Sodium 2-hydroxylinoleate
Sodium 2-hydroxylinoleate
Treatment of cystic fibrosis
Nitric oxide
Nitric oxide
Treatment of perinatal asphyxia
Xenon
Xenon
Treatment of choroideremia
Adeno-associated viral vector serotype 5 containing the human CHM gene
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
Phenol, 4-[2-(aminomethyl)-4-thiazolyl]-2,6-bis(1,1-dimethylethyl)monohydrochloride
Treatment of Ebola virus disease
Rintatolimod
Rintatolimod
Prevention of graft rejection following solid organ transplantation
Recombinant monoclonal IgG1 antibody against T-cell immune response cDNA 7
Recombinant monoclonal IgG1 antibody against T-cell immune response cDNA 7
Treatment of Duchenne muscular dystrophy
Rimeporide
Rimeporide
Treatment of pancreatic cancer
Human reovirus type 3 Dearing strain
Human reovirus type 3 Dearing strain
Treatment of tularaemia
1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride
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
1-(4-(N-glycylamido)phenyl)-3-trifluoromethyl-5-(phenanthren-2-yl)-pyrazole-hydrochloride
Treatment of Stargardt's disease
Ecothiophate iodide
Ecothiophate iodide
Treatment of marginal zone lymphoma
Lenalidomide
Lenalidomide
Diagnosis of glioma
Fluciclovine (18F)
Fluciclovine (18F)
March 2015
Treatment of Wilson’s disease
Trientine tetrahydrochloride
Trientine tetrahydrochloride
Treatment of biliary tract cancer
5,10,15,20-tetrakis(2,6-difluoro-3-Nmethylsulfamoylphenyl)bacteriochlorin
5,10,15,20-tetrakis(2,6-difluoro-3-Nmethylsulfamoylphenyl)bacteriochlorin
Treatment of ovarian cancer
Human reovirus type 3 Dearing strain
Human reovirus type 3 Dearing strain
Treatment of Gaucher disease
Adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene
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
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
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 aLAMB3-encoding retroviral vector
Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with aLAMB3-encoding retroviral vector
Treatment of systemic sclerosis
Autologous adipose tissue-derived stromal vascular fraction cells
Autologous adipose tissue-derived stromal vascular fraction cells
Treatment of plasma cell myeloma
Melphalan flufenamide
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
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
Lenvatinib
Treatment of amyotrophic lateral sclerosis
Enoxacin
Enoxacin
Treatment of ovarian cancer
Humanised anti-folate receptor 1 monoclonal antibody conjugated to maytansinoid DM4
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
[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
Recombinant human club cell 10 KDa protein
Treatment of graft-versus-host disease
Human plasma-derived alpha-1 proteinase inhibitor
Human plasma-derived alpha-1 proteinase inhibitor
Treatment of Netherton syndrome
6-ethoxy-7-methoxy-2-(2-methylsulfanylphenyl)-3,1-benzoxazin-4-one
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
Chimeric 2'-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA
Treatment of fragile X syndrome
Tideglusib
Tideglusib
Treatment of Alport syndrome
5'-ASCSASTSCSASGSTSCSTSGSASUSASASGSCSTSA-3'
5'-ASCSASTSCSASGSTSCSTSGSASUSASASGSCSTSA-3'
Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
Gallium (68Ga)-edotreotide
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
505 amino acid protein, corresponding to amino acids 2-506 of the wild type human histidyl-tRNA synthetase
Treatment of soft tissue sarcoma
Olaratumab
Olaratumab
Treatment of progressive supranuclear palsy
N-(3-(4-(3-(diisobutylamino)propyl)piperazin-1-yl)propyl)-1Hbenzo[d]imidazol-2-amine disulphate salt
N-(3-(4-(3-(diisobutylamino)propyl)piperazin-1-yl)propyl)-1Hbenzo[d]imidazol-2-amine disulphate salt
Treatment of acute myeloid leukaemia
Ulocuplumab
Ulocuplumab
Treatment of narcolepsy
Mazindol
Mazindol
Treatment of glioma
Recombinant human glutamate oxaloacetate transaminase 1
Recombinant human glutamate oxaloacetate transaminase 1
Treatment of Ebola virus disease
Fibrinogen-coated albumin spheres
Fibrinogen-coated albumin spheres
Treatment of sickle cell disease
5-hydroxymethyl-2-furfural
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
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
Alvocidib
Prevention of necrotising enterocolitis
“Lactobacillus reuteri”
“Lactobacillus reuteri”
Treatment of systemic sclerosis
Nitroglycerin
Nitroglycerin
Treatment of ovarian cancer
Chimeric group B adenovirus (11p/3) with deletions in the E3 and E4 Regions
Chimeric group B adenovirus (11p/3) with deletions in the E3 and E4 Regions
Treatment of sickle cell disease
Sevuparin sodium
Sevuparin sodium
Treatment of hereditary angioedema
3-[2-(4-carbamimidoyl-phenylcarbamoyl)-5-methoxy-4-vinyl-phenyl]-6-(cyclopropylmethyl-carbamoyl)-pyridine-2-carboxylic acid
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
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
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
Sodium valproate
Treatment of hypophosphatasia
Recombinant human alkaline phosphatase
Recombinant human alkaline phosphatase
Prevention of graft-versus-host disease
Allogeneic peripheral blood mononuclear cells induced to an early apoptotic state
Allogeneic peripheral blood mononuclear cells induced to an early apoptotic state
Treatment of spinocerebellar ataxia
Ceftriaxone
Ceftriaxone
Treatment of diffuse large B-cell lymphoma
Humanised Fc engineered monoclonal antibody against CD19
Humanised Fc engineered monoclonal antibody against CD19
Treatment of plasma cell myeloma
Synthetic signal peptide of human mucin-1 (amino acids 1-21)
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
Chimeric fusion protein of recombinant human alpha-Nacetylglucosaminidase and human insulin-like growth factor 2
Treatment of gastrointestinal stromal tumours
Ponatinib hydrochloride
Ponatinib hydrochloride
Treatment of Aicardi-Goutières syndrome
Emtricitabine
Emtricitabine
Treatment of Aicardi-Goutières syndrome
Tenofovir disoproxil fumarate
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
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
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
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
Adenoviral vector serotype 5 containing the vascular endothelial growth factor D isoform (preprocessed short form) from a CMV promoter
Treatment for calciphylaxis
Sodium thiosulfate
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
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
Herpes simplex type 1 virus containing cellular B-myb gene as tumour specific promoter
Treatment of interstitial cystitis
Pentosan polysulfate sodium
Pentosan polysulfate sodium
Treatment of aspartylglucosaminuria
Recombinant human aspartylglucosaminidase
Recombinant human aspartylglucosaminidase
Treatment of malignant mesothelioma
Pegylated recombinant arginine deiminase
Pegylated recombinant arginine deiminase
December 2014
Treatment of glioma
5,5'-(4-(trifluromethyl)benzylazanediyl)bis(methylene)diquinolin-8-ol
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
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
Chenodeoxycholic acid
Treatment of non-infectious uveitis
Autologous collagen type II-specific regulatory T cells
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
1-(2-isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d]pyrimidin-4-one
Treatment of WHIM syndrome
Plerixafor
Plerixafor
Treatment of beta-thalassaemia intermedia and major
Benserazide hydrochloride
Benserazide hydrochloride
Treatment of traumatic spinal cord injury
Riluzole
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
(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
Edaravone
Treatment of malignant mesothelioma
Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor
Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor
Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Amikacin sulfate
Amikacin sulfate
Treatment of glioma
Adenovirus serotype 5 containing partial E1A deletion and an integrin-binding domain
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
Allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells
Treatment of pancreatic cancer
Pegylated recombinant human hyaluronidase PH20
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
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
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
5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine
Treatment of hereditary haemorrhagic telangiectasia
Bevacizumab
Bevacizumab
Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)
Adeno-associated viral vector serotype 10 carrying the human Nsulfoglucosamine sulfohydrolase cDNA
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
Allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media
Treatment of familial cerebral cavernous malformations
Exisulind
Exisulind
Treatment of ovarian cancer
2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one
2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one
Treatment of pancreatic cancer
Heat-killed Mycobacterium obuense (whole cell)
Heat-killed Mycobacterium obuense (whole cell)
Treatment of WHIM syndrome
((E)-1-(4'-chlorophenyl)-3-(4-hydroxy-3-metoxyphenyl)prop-2-en-1-one)
((E)-1-(4'-chlorophenyl)-3-(4-hydroxy-3-metoxyphenyl)prop-2-en-1-one)
Treatment of pleural empyema
Single-chain urokinase plasminogen activator
Single-chain urokinase plasminogen activator
Treatment of acute lymphoblastic leukaemia
Allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonist
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
Adeno-associated viral vector serotype 8 containing the human MD1 gene
Treatment of mucopolysaccharidosis type I
Ataluren
Ataluren
Treatment of malignant hyperthermia
Dantrolene sodium
Dantrolene sodium
Treatment of hepatocellular carcinoma
Diaspirin cross-linked haemoglobin
Diaspirin cross-linked haemoglobin
Treatment of Niemann-Pick disease, type C
Arimoclomol citrate
Arimoclomol citrate
Treatment of xeroderma pigmentosum
Pro-Pro-Thr-Val-Pro-Thr-Arg
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
(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
(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
(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
Humanised IgG1 monoclonal antibody against human eotaxin-2
Treatment of polymyositis
Siponimod
Siponimod
Treatment of dermatomyositis
Siponimod
Siponimod
Treatment of fibrodysplasia ossificans progressiva
Palovarotene
Palovarotene
Treatment of hereditary haemorrhagic telangiectasia
Bazedoxifene acetate
Bazedoxifene acetate
Treatment of primary myelofibrosis
Recombinant human pentraxin-2
Recombinant human pentraxin-2
Treatment of post-polycythaemia vera myelofibrosis
Recombinant human pentraxin-2
Recombinant human pentraxin-2
Treatment of glioma
Olaptesed pegol
Olaptesed pegol
Treatment of idiopathic pulmonary fibrosis
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid
Treatment of systemic sclerosis
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid
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
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
Pentosan polysulfate sodium
Treatment of post-essential thrombocythaemia myelofibrosis
Recombinant human pentraxin-2
Recombinant human pentraxin-2
Treatment of acute respiratory distress syndrome
Imatinib
Imatinib
Treatment of acute myeloid leukaemia
Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment
Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment
Treatment of plasma cell myeloma
Selinexor
Selinexor
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Selinexor
Selinexor
Treatment of myotonic disorders
Mexiletine hydrochloride
Mexiletine hydrochloride
October 2014
Treatment of inhalation anthrax disease
Raxibacumab
Raxibacumab
Treatment of X-linked hypophosphataemia
Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23
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
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
Recombinant human insulin receptor monoclonal antibody-fused-α-Liduronidase
Treatment of glioma
Recombinant human bone morphogenetic protein 4
Recombinant human bone morphogenetic protein 4
Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Pyridoxal 5'-phosphate
Pyridoxal 5'-phosphate
Treatment of short bowel syndrome
Oxalobacter formigenes strain HC-1
Oxalobacter formigenes strain HC-1
Treatment of Cushing's syndrome
Osilodrostat
Osilodrostat
Treatment of cystic fibrosis
Nitric oxide
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
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
Glucagon
Treatment of cystinosis
Cysteamine hydrochloride
Cysteamine hydrochloride
Treatment of limbal stem cell deficiency
Cultured allogeneic corneal limbal stem cells
Cultured allogeneic corneal limbal stem cells
Treatment of Dravet syndrome
Cannabidiol
Cannabidiol
Treatment of Crigler-Najjar syndrome
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene
Treatment of fragile X syndrome
Acamprosate calcium
Acamprosate calcium
Treatment of Leigh syndrome
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride
(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
(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
Lumacaftor / ivacaftor
Prevention of ischaemia reperfusion injury associated with solid organ transplantation
Macromolecular conjugate of heparin sodium on a polymer backbone
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
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)
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
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)
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
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
Obinutuzumab
Treatment of small cell lung cancer
2-(2-methyl-5-nitro-1H-imidazol-1-yl)ethylsulfamide
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
[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
Humanised IgG1 monoclonal antibody against human KIR3DL2
Treatment of Crigler-Najjar syndrome
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene
Treatment of mastocytosis
Recombinant human diamine oxidase
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
Recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin
Treatment of acute pancreatitis
Ulinastatin
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
(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
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
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
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
(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
Recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin
Treatment of Schnitzler syndrome
Gevokizumab
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
(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
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
Sodium ascorbate and menadione sodium bisulfite
Prevention of bronchopulmonary dysplasia
Retinol
Retinol
July 2014
Treatment of Huntington’s disease
Cysteamine bitartrate
Cysteamine bitartrate
Treatment of glioma
Humanised recombinant monoclonal antibody against epidermal growth factor receptor conjugated to maleimidocaproyl monomethylauristatin F
Humanised recombinant monoclonal antibody against epidermal growth factor receptor conjugated to maleimidocaproyl monomethylauristatin F
Treatment of myasthenia gravis
Eculizumab
Eculizumab
Treatment of ovarian cancer
Cediranib
Cediranib
Treatment of Prader-Willi syndrome
Oxytocin
Oxytocin
Treatment of idiopathic pulmonary fibrosis
Humanised anti-alpha ν beta 6 monoclonal antibody
Humanised anti-alpha ν beta 6 monoclonal antibody
Treatment of beta-thalassaemia intermedia and major
Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain
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 systemic sclerosis
Riociguat
Riociguat
Treatment of haemophilia B
Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Treatment of haemophilia A
Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Synthetic double-stranded siRNA oligonucleotide directed against antithrombin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Treatment of catecholaminergic polymorphic ventricular tachycardia
Adeno-associated viral vector serotype 9 containing the human cardiac calsequestrin gene
Adeno-associated viral vector serotype 9 containing the human cardiac calsequestrin gene
Treatment of plasma cell myeloma
Marizomib
Marizomib
Treatment for necrotising soft tissue infections
Sodium acetate salt of the synthetic peptide H-D-Ala-Ser-Pro-Met-Leu-Val-Ala-Tyr-Asp-D-Ala-OH
Sodium acetate salt of the synthetic peptide H-D-Ala-Ser-Pro-Met-Leu-Val-Ala-Tyr-Asp-D-Ala-OH
Treatment of AL amyloidosis
Recombinant monoclonal antibody to human serum amyloid P component
Recombinant monoclonal antibody to human serum amyloid P component
Treatment of AL amyloidosis
Carboxy pyrrolidine hexanoyl pyrrolidine carboxylate
Carboxy pyrrolidine hexanoyl pyrrolidine carboxylate
Treatment of gastric cancer
Rilotumumab
Rilotumumab
Treatment of cystic fibrosis
1-(2,2-difluoro-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropanecarboxamide
1-(2,2-difluoro-1,3-benzodioxol-5-yl)-N-{1-[(2R)-2,3-dihydroxypropyl]-6-fluoro-2-(1-hydroxy-2-methylpropan-2-yl)-1H-indol-5-yl}cyclopropanecarboxamide
Treatment of Usher syndrome
Mixture of two adeno-associated viral vectors of serotype 8 containing the 5'-half sequence of humanMYO7A gene and the 3'-half sequence of human MYO7A gene
Mixture of two adeno-associated viral vectors of serotype 8 containing the 5'-half sequence of humanMYO7A gene and the 3'-half sequence of human MYO7A gene
Treatment of Stargardt's disease
Mixture of two adeno-associated viral vectors of serotype 8 containing the 5'-half sequence of human ABCA4 gene and the 3'-half sequence of human ABCA4 gene
Mixture of two adeno-associated viral vectors of serotype 8 containing the 5'-half sequence of human ABCA4 gene and the 3'-half sequence of human ABCA4 gene
Treatment of invasive aspergillosis
Isavuconazonium sulfate
Isavuconazonium sulfate
Treatment of familial benign chronic pemphigus (Hailey-Hailey disease)
Afamelanotide
Afamelanotide
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Humanised Fc engineered monoclonal antibody against CD19
Humanised Fc engineered monoclonal antibody against CD19
Treatment of Prader-Willi syndrome
Beloranib
Beloranib
Treatment of primary sclerosing cholangitis
Norursodeoxycholic acid
Norursodeoxycholic acid
Treatment of pre-eclampsia
Recombinant human alpha-1-microglobulin
Recombinant human alpha-1-microglobulin
Treatment of choroideraemia
Adeno-associated viral vector serotype 2 containing the human REP1 gene
Adeno-associated viral vector serotype 2 containing the human REP1 gene
June 2014
Treatment of gastro-entero-pancreatic neuroendocrine tumours
Lutetium (177Lu) edotreotide
Lutetium (177Lu) edotreotide
Treatment of biliary tract cancer
(5R,5aR,8aR,9S)-9-[[4,6-O-[(R)-Ethylidene]-(-D-glucopyranosyl]-oxy]-5-(4-({[(2,2-dimethyl-1,3-dioxolan-4-yl)methoxy]carbonyl}oxy)-3,5-dimethoxyphenyl)-5,8,8a,9 tetrahydroisobenzofuro[5,6-f][1,3]benzodioxol-6(5aH)-one
(5R,5aR,8aR,9S)-9-[[4,6-O-[(R)-Ethylidene]-(-D-glucopyranosyl]-oxy]-5-(4-({[(2,2-dimethyl-1,3-dioxolan-4-yl)methoxy]carbonyl}oxy)-3,5-dimethoxyphenyl)-5,8,8a,9 tetrahydroisobenzofuro[5,6-f][1,3]benzodioxol-6(5aH)-one
Treatment of follicular lymphoma
177Lu-tetraxetan-tetulomab
177Lu-tetraxetan-tetulomab
Treatment of epidermolysis bullosa
Recombinant human alpha 1 chain homotrimer of type VII collagen
Recombinant human alpha 1 chain homotrimer of type VII collagen
Treatment of glioma
Autologous dendritic cells pulsed with RNA from glioma stem cells
Autologous dendritic cells pulsed with RNA from glioma stem cells
Treatment of glioma
Paclitaxel-succinate-Arg-Arg-Leu-Ser-Tyr-Ser-Arg-Arg-Arg-Phe
Paclitaxel-succinate-Arg-Arg-Leu-Ser-Tyr-Ser-Arg-Arg-Arg-Phe
Treatment of central retinal vein occlusion
Aganirsen
Aganirsen
Treatment of mucormycosis
Isavuconazonium sulfate
Isavuconazonium sulfate
Treatment of cystic fibrosis
Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a non-viral, cationic lipid based gene transfer agent
Plasmid DNA encoding the human cystic fibrosis transmembrane conductance regulator gene complexed with a non-viral, cationic lipid based gene transfer agent
Treatment of choroideraemia
Adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1
Adeno-associated viral vector serotype 2 containing the human CHM gene encoding human Rab escort protein 1
Prevention of scarring post glaucoma filtration surgery
4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid
4-(4-Methoxy-phenylamino)-6-methylcarbamyl-quinoline-3-carboxylic acid
Treatment of mucopolysaccharidosis IIIA (Sanfilippo A syndrome)
Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
April 2014
Treatment of lymphoplasmacytic lymphoma
Ibrutinib
Ibrutinib
Treatment of ovarian cancer
Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor
Genetically modified serotype 5/3 adenovirus coding for granulocyte macrophage colony-stimulating factor
Treatment of B-lymphoblastic leukaemia/lymphoma
Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19
Autologous T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19
Treatment of ATTR amyloidosis
Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA and covalently linked to a ligand containing three N-acetylgalactosamine residues
Treatment of plasma cell myeloma
Humanised monoclonal antibody against CD38
Prevention of bronchopulmonary dysplasia
Caffeine citrate
Caffeine citrate
Prevention of bronchopulmonary dysplasia
Recombinant human surfactant protein D
Recombinant human surfactant protein D
March 2014
Treatment of ATTR amyloidosis
Phosphorothioate oligonucleotide targeted to transthyretin
Phosphorothioate oligonucleotide targeted to transthyretin
Treatment of glycogen storage disease type II (Pompe's disease)
Recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan
Recombinant human alpha-glucosidase conjugated with multiple copies of synthetic bismannose-6-phosphate-tetra-mannose glycan
Treatment of pancreatic cancer
Cysteamine bitartrate
Cysteamine bitartrate
Prevention of graft rejection following solid organ transplantation
Ex-vivo-cultured human mesenchymal stromal cells
Ex-vivo-cultured human mesenchymal stromal cells
Prevention of graft rejection following solid organ transplantation
Eculizumab
Eculizumab
Treatment of acute myeloid leukaemia
Volasertib
Volasertib
Treatment of Leber’s congenital amaurosis
Adeno-associated viral vector serotype 8 containing the human GUCY2D gene
Adeno-associated viral vector serotype 8 containing the human GUCY2D gene
Treatment of recombination-activating gene 1 deficient severe combined immunodeficiency
Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene
Autologous CD34+ cells transduced with a lentiviral vector containing the human RAG1 gene
Treatment of soft tissue sarcoma
Doxorubicin(6-maleimidocaproyl)hydrazone
Doxorubicin(6-maleimidocaproyl)hydrazone
Treatment of nontuberculous mycobacterial lung disease
Amikacin sulfate
Amikacin sulfate
Treatment of Charcot-Marie-Tooth disease type 1A
Fixed-dose combination of (R-S) baclofen, naltrexone hydrochloride and D-sorbitol
Fixed-dose combination of (R-S) baclofen, naltrexone hydrochloride and D-sorbitol
February 2014
Treatment of Farber disease
Recombinant human acid ceramidase
Recombinant human acid ceramidase
Prevention of congenital cytomegalovirus infection following primary cytomegalovirus infection
Mixture of recombinant human IgG1 monoclonal antibodies against human cytomegalovirus envelope glycoproteins
Mixture of recombinant human IgG1 monoclonal antibodies against human cytomegalovirus envelope glycoproteins
Treatment of epidermolysis bullosa
Diacerein
Diacerein
Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
Gallium [Ga-68]-N-[(4,7,10-tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-Lcysteinyl-L-threonine-cyclic(27)disulfide
Gallium [Ga-68]-N-[(4,7,10-tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-Lcysteinyl-L-threonine-cyclic(27)disulfide
Prevention of delayed graft function after solid organ transplantation
Eculizumab
Eculizumab
Treatment of cystic fibrosis
11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-azacyclopentadecane-13,15-dione
11-(4-Dimethylamino-3-hydroxy-6-methyl-tetrahydro-pyran-2-yloxy)-2-ethyl-3,4,10-trihydroxy-3,5,6,8,10,12,14-heptamethyl-1-oxa-6-azacyclopentadecane-13,15-dione
Treatment of cystic fibrosis
Cysteamine
Cysteamine
Treatment of Duchenne muscular dystrophy
Asp-Arg-Val-Tyr-Ile-His-Pro
Asp-Arg-Val-Tyr-Ile-His-Pro
Treatment of Rett síndrome
3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl)amino]methyl}piperidin-1-yl]methanone
3-Chloro-4-fluorophenyl-[4-fluoro-4-{[(5-methylpyrimidin-2-ylmethyl)amino]methyl}piperidin-1-yl]methanone
Treatment of polycythaemia vera
Ruxolitinib
Ruxolitinib
Treatment of adrenoleukodystrophy
Pioglitazone
Pioglitazone
Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
68Ga-2,2'-(7-(4-((S)-1-((4S,7S,10S,13R,16S,19R)-4-((R)-1-amino-3-(4-hydroxyphenyl)-1-oxopropan-2-ylcarbamoyl)-10-(4-aminobutyl)-16-(4-((S)-2,6-dioxohexahydropyrimidine-4-carboxamido)benzyl)-7-((R)-1-hydroxyethyl)-6,9,12,15,18-pentaoxo-13-(4-ureidobenzyl)-1,2-dithia-5,8,11,14,17-pentaazacycloicosan-19-ylamino)-3-(4-chlorophenyl)-1-oxopropan-2-ylamino)-1-carboxy-4-oxobutyl)-1,4,7-triazonane-1,4-diyl)diacetic acid
68Ga-2,2'-(7-(4-((S)-1-((4S,7S,10S,13R,16S,19R)-4-((R)-1-amino-3-(4-hydroxyphenyl)-1-oxopropan-2-ylcarbamoyl)-10-(4-aminobutyl)-16-(4-((S)-2,6-dioxohexahydropyrimidine-4-carboxamido)benzyl)-7-((R)-1-hydroxyethyl)-6,9,12,15,18-pentaoxo-13-(4-ureidobenzyl)-1,2-dithia-5,8,11,14,17-pentaazacycloicosan-19-ylamino)-3-(4-chlorophenyl)-1-oxopropan-2-ylamino)-1-carboxy-4-oxobutyl)-1,4,7-triazonane-1,4-diyl)diacetic acid
Treatment of glioma
Autologous dendritic cells pulsed with tumour antigen-derived synthetic peptides (MAGE-1, HER-2, AIM-2, TRP-2, gp-100, and interleukin-13 receptor alpha)
Autologous dendritic cells pulsed with tumour antigen-derived synthetic peptides (MAGE-1, HER-2, AIM-2, TRP-2, gp-100, and interleukin-13 receptor alpha)
Treatment of optic neuritis
N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)-propyl]-carbamoyl}-methyl)-2-[2-(2-fluoro-phenyl)-ethylamino]-N-isobutyl-acetamide
N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)-propyl]-carbamoyl}-methyl)-2-[2-(2-fluoro-phenyl)-ethylamino]-N-isobutyl-acetamide
Treatment of familial chylomicronemia syndrome
Phosphorothioate oligonucleotide targeted to apolipoprotein CIII
Phosphorothioate oligonucleotide targeted to apolipoprotein CIII
January 2014
Treatment of malignant mesothelioma
Amatuximab
Amatuximab
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Inecalcitol
Inecalcitol
Treatment of aneurysmal subarachnoid haemorrhage
Sodium nitrite
Sodium nitrite
Treatment of hepatitis delta virus infection
Lonafarnib
Lonafarnib
Treatment of dystrophic myotonia
(6aS)-1,10-dimethoxy-6-methyl-5,6,6a,7-tetrahydro-4Hdibenzo[de,g]quinoline-2,9-diol
(6aS)-1,10-dimethoxy-6-methyl-5,6,6a,7-tetrahydro-4Hdibenzo[de,g]quinoline-2,9-diol
Treatment of adenovirus infection in allogeneic haematopoietic stem-cell transplant recipients
Adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo
Adenovirus-specific T-cells derived from allogeneic donor leukocytes, expanded ex vivo
Treatment of primary sclerosing cholangitis
Obeticholic acid
Obeticholic acid
Treatment of malignant mesothelioma
Autologous dendritic cells pulsed with allogeneic tumour cell lysate
Autologous dendritic cells pulsed with allogeneic tumour cell lysate
Treatment of acute myeloid leukaemia
(2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-((((1r,3S)-3-(2-(5-(tert-butyl)-1H benzo[d]imidazol-2-yl)ethyl)cyclobutyl)(isopropyl) amino) methyl)tetrahydrofuran-3,4-diol
(2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-((((1r,3S)-3-(2-(5-(tert-butyl)-1H benzo[d]imidazol-2-yl)ethyl)cyclobutyl)(isopropyl) amino) methyl)tetrahydrofuran-3,4-diol
Treatment of acute lymphoblastic leukaemia
(2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-((((1r,3S)-3-(2-(5-(tert-butyl)-1Hbenzo[d]imidazol-2-yl)ethyl)cyclobutyl)(isopropyl)amino)methyl)tetrahydrofuran-3,4-diol
(2R,3R,4S,5R)-2-(6-amino-9H-purin-9-yl)-5-((((1r,3S)-3-(2-(5-(tert-butyl)-1Hbenzo[d]imidazol-2-yl)ethyl)cyclobutyl)(isopropyl)amino)methyl)tetrahydrofuran-3,4-diol
Treatment of epidermolysis bullosa
Allantoin
Allantoin
Prevention of graft-versus-host disease
Allogeneic bone-marrow derived ex-vivo expanded multipotent adult progenitor cells
Allogeneic bone-marrow derived ex-vivo expanded multipotent adult progenitor cells
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
N-(3-(5-fluoro-2-(4-(2-methoxyethoxy)phenylamino)pyrimidin-4-ylamino) phenyl)acrylamide benzenesulfonic acid salt
N-(3-(5-fluoro-2-(4-(2-methoxyethoxy)phenylamino)pyrimidin-4-ylamino) phenyl)acrylamide benzenesulfonic acid salt
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