| ORPHAN DRUGS | |||
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Recent orphan drug designations
July 2014
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 human MYO7Agene 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 human MYO7Agene 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
December 2013
Treatment of cystic fibrosis
Nitric oxide
Nitric oxide
Treatment of hypoparathyroidism
Recombinant human parathyroid hormone
Recombinant human parathyroid hormone
Treatment of glioma
Allogeneic and autologous haptenised and irradiated cells and cell lysates derived from glioma
Allogeneic and autologous haptenised and irradiated cells and cell lysates derived from glioma
Treatment of follicular lymphoma
Ibrutinib
Ibrutinib
Prevention of necrotizing enterocolitis
Lactobacillus acidophilus and Bifidobacterium bifidum
Lactobacillus acidophilus and Bifidobacterium bifidum
Treatment of Alagille síndrome
(4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
(4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
Treatment of primary biliary cirrosis
(4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
(4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
Treatment of progressive familial intrahepatic cholestasis
(4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
(4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
Treatment of primary sclerosing colangitis
(4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
(4R,5R)-1-[[4-[[4-[3,3-dibutyl-7-(dimethylamino)-2,3,4,5-tetrahydro-4-hydroxy-1,1-dioxido-1-benzothiepin-5-yl]phenoxy]methyl]phenyl]methyl]-4-aza-1-azoniabicyclo[2.2.2]octane chloride
Prevention of arteriovenous access dysfunction in haemodialysis patients
Recombinant human type I pancreatic elastase
Recombinant human type I pancreatic elastase
Treatment of Dravet syndrome
Fenfluramine hydrochloride
Fenfluramine hydrochloride
Treatment of dengue
Poly[2-[(4-{[1-carboxy-2-(hexadecylcarbamoyl)ethyl]sulfanyl}-2,3-bis({2-[((2S)-2-(2-{[(2R)-2-carbamoyl-(2-{[(2S)-1-ethoxy-3-(3-hydroxy-4oxo-1,4-dihydropyridin-1-yl)-1-oxopropan-2-yl]carbamoyl}ethyl]sulfanyl}-3-{[(2S)-1-ethoxy-3-(3-hydroxy-4-oxo-1,4-dihydropyridin-1-yl)-1-oxopropan-2-yl]carbamoyl}propanamido)-3-(3-hydroxy-4-oxo-1,4-dihydropyridin-1-yl)propanoyl Ethyl ester) )-methoxy]acetyl}oxy)butyl)sulfanyl]-3-(hexadecylcarbamoyl)propanoic acid]-poly(ethylene glycol)-ester]
Poly[2-[(4-{[1-carboxy-2-(hexadecylcarbamoyl)ethyl]sulfanyl}-2,3-bis({2-[((2S)-2-(2-{[(2R)-2-carbamoyl-(2-{[(2S)-1-ethoxy-3-(3-hydroxy-4oxo-1,4-dihydropyridin-1-yl)-1-oxopropan-2-yl]carbamoyl}ethyl]sulfanyl}-3-{[(2S)-1-ethoxy-3-(3-hydroxy-4-oxo-1,4-dihydropyridin-1-yl)-1-oxopropan-2-yl]carbamoyl}propanamido)-3-(3-hydroxy-4-oxo-1,4-dihydropyridin-1-yl)propanoyl Ethyl ester) )-methoxy]acetyl}oxy)butyl)sulfanyl]-3-(hexadecylcarbamoyl)propanoic acid]-poly(ethylene glycol)-ester]
Treatment of haemophilia A
Humanised monoclonal modified IgG4 antibody with bispecific structure targeting factors IX, IXa, X and Xa
Humanised monoclonal modified IgG4 antibody with bispecific structure targeting factors IX, IXa, X and Xa
November 2013
Treatment of glioma
Autologous ex-vivo-expanded leucocytes treated with 5-aza-2’-deoxycytidine
Autologous ex-vivo-expanded leucocytes treated with 5-aza-2’-deoxycytidine
Treatment of mucopolysaccharidosis type II (Hunter's syndrome)
Recombinant human insulin receptor monoclonal antibody-fusediduronate 2-sulfatase
Recombinant human insulin receptor monoclonal antibody-fusediduronate 2-sulfatase
Treatment of follicular thyroid cancer
Sorafenib tosylate
Sorafenib tosylate
Treatment of papillary thyroid cancer
Sorafenib tosylate
Sorafenib tosylate
Prevention of graft-versus-host disease
Defibrotide
Defibrotide
Treatment of hepatocellular carcinoma
Tivantinib
Tivantinib
Treatment of diffuse large B-cell lymphoma
Ibrutinib
Ibrutinib
Prevention of arteriovenous access dysfunction in patients undergoing surgical creation of an arteriovenous access for haemodialysis
Sirolimus
Sirolimus
Treatment of eosinophilic oesophagitis
Human monoclonal antibody against human interleukin 13
Human monoclonal antibody against human interleukin 13
Treatment of spinal cord injury
Synthetic 12 amino acids peptide designed after subcommissural organ spondin
Synthetic 12 amino acids peptide designed after subcommissural organ spondin
Treatment of ovarian cancer
Trebananib
Trebananib
Treatment of Stargardt’s disease
Soraprazan
Soraprazan
October 2013
Treatment of congenital factor VII deficiency
Recombinant fusion protein linking coagulation factor VIIa with Albumin
Recombinant fusion protein linking coagulation factor VIIa with Albumin
Treatment of myotonic disorders
Mexiletine hydrochloride
Mexiletine hydrochloride
Treatment of plasma cell myeloma
Recombinant human monoclonal IgM antibody targeting glucose-regulated protein 78
Recombinant human monoclonal IgM antibody targeting glucose-regulated protein 78
Treatment of sarcoidosis
L-Pyr-L-Glu-L-Gln-L-Leu-L-Glu-L-Arg-L-Ala-L-Leu-L-Asn-LSer-L-Ser
L-Pyr-L-Glu-L-Gln-L-Leu-L-Glu-L-Arg-L-Ala-L-Leu-L-Asn-LSer-L-Ser
Treatment of complex regional pain syndrome
Zoledronic acid
Zoledronic acid
Treatment of Allan-Herndon-Dudley syndrome
3,5-diiodothyropropionic acid
3,5-diiodothyropropionic acid
Treatment of Duchenne muscular dystrophy
Naproxcinod
Naproxcinod
Treatment of cystic fibrosis
Antisense oligonucleotide targeting the F508delta mutation of CFTR
Antisense oligonucleotide targeting the F508delta mutation of CFTR
Treatment of Wiskott-Aldrich-syndrome
Autologous CD34+ cells transduced with a lentiviral vector containing the human Wiskott-Aldrich syndrome gene
Autologous CD34+ cells transduced with a lentiviral vector containing the human Wiskott-Aldrich syndrome gene
July & August 2013
Treatment of pulmonary alveolar proteinosis
Granulocyte macrophage colony stimulating factor
Granulocyte macrophage colony stimulating factor
Autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors
Autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors
Autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors
Prevention of ischaemia/reperfusion injury associated with solid organ transplantation
Human hemin
Human hemin
Treatment of B-lymphoblastic leukaemia/lymphoma
Moxetumomab pasudotox
Moxetumomab pasudotox
Treatment of malignant thymomas
Belinostat
Belinostat
Treatment of pancreatic cancer
(1-methyl-2-nitro-1H-imidazole-5-yl) methyl N,N’-bis(2-bromoethyl) diamidophosphate
(1-methyl-2-nitro-1H-imidazole-5-yl) methyl N,N’-bis(2-bromoethyl) diamidophosphate
Treatment of plasma cell myeloma
Daratumumab
Daratumumab
Treatment of ovarian cancer
Fosbretabulin tromethamine
Fosbretabulin tromethamine
Treatment of amyotrophic lateral sclerosis
Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
Prevention of hepatitis B re-infection following liver transplantation
Recombinant human monoclonal antibody against hepatitis B Virus
Recombinant human monoclonal antibody against hepatitis B Virus
Treatment of follicular lymphoma
(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one
(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one
Treatment of ataxia telangiectasia
Dexamethasone sodium phosphate encapsulated in human autologous erythrocytes
Dexamethasone sodium phosphate encapsulated in human autologous erythrocytes
Treatment of carbamoylphosphate synthase-1 deficiency
Heterologous human adult liver-derived progenitor cells
Heterologous human adult liver-derived progenitor cells
Treatment of citrullinaemia type 1
Heterologous human adult liver-derived progenitor cells
Heterologous human adult liver-derived progenitor cells
Treatment of argininosuccinic aciduria
Heterologous human adult liver-derived progenitor cells
Heterologous human adult liver-derived progenitor cells
Treatment of hyperargininaemia
Heterologous human adult liver-derived progenitor cells
Heterologous human adult liver-derived progenitor cells
Treatment of N-acetylglutamate synthetase (NAGS) deficiency
Heterologous human adult liver-derived progenitor cells
Heterologous human adult liver-derived progenitor cells
Treatment of citrullinaemia type 2
Heterologous human adult liver-derived progenitor cells
Heterologous human adult liver-derived progenitor cells
Treatment of ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome)
Heterologous human adult liver-derived progenitor cells
Heterologous human adult liver-derived progenitor cells
Treatment of limbal stem cell deficiency
Ex-vivo expanded autologous human corneal epithelium containing stem cells
Ex-vivo expanded autologous human corneal epithelium containing stem cells
Treatment of osteosarcoma
Lipid-complexed cisplatin
Lipid-complexed cisplatin
Treatment of acromegaly
Octreotide acetate (oral use)
Octreotide acetate (oral use)
Treatment of nodal marginal zone lymphoma
Idelalisib
Idelalisib
Treatment of chronic lymphocytic leukaemia/small lymphocytic lymphoma
Idelalisib
Idelalisib
Treatment of acute myeloid leukaemia
Trans-N1-((1R,2S)-2-phenylcyclopropyl)cyclohexane-1,4-diamine bis-hydrochloride
Trans-N1-((1R,2S)-2-phenylcyclopropyl)cyclohexane-1,4-diamine bis-hydrochloride
Treatment of autosomal dominant polycystic kidney disease
Tolvaptan
Tolvaptan
Treatment of nontraumatic osteonecrosis
Human allogeneic bone marrow derived osteoblastic-like cells
Human allogeneic bone marrow derived osteoblastic-like cells
Treatment of pancreatic cancer
Chimeric monoclonal antibody against claudin-18 splice variant 2
Chimeric monoclonal antibody against claudin-18 splice variant 2
Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Pegylated recombinant anti-Pseudomonas aeruginosa PcrV Fab′antibody
Pegylated recombinant anti-Pseudomonas aeruginosa PcrV Fab′antibody
Treatment of growth hormone deficiency
Recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains
Recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains
Treatment of Behçets' disease
Apremilast
Apremilast
Treatment of eosinophilic oesophagitis
Budesonide
Budesonide
Treatment of mastocytosis
Cladribine
Cladribine
Treatment of congenital sucrase-isomaltase deficiency
Sacrosidase
Sacrosidase
Treatment of sickle cell disease
(1R,3R,4R,5S)-3-O-[2-O-benzoyl-3-O-(sodium(2S)-3-cyclohexyl-propanoate-2-yl)-β-D-galactopyranosyl]-4-O-(α-L-fucopyranosyl)-5-orothylamido-cyclohexane-1-carboxylic acid ethyl-2-amidyl-ethyloxy-2-acetyl-(8-amino-1,3,6-naphthalene-tris sodium sulfonate) amide
(1R,3R,4R,5S)-3-O-[2-O-benzoyl-3-O-(sodium(2S)-3-cyclohexyl-propanoate-2-yl)-β-D-galactopyranosyl]-4-O-(α-L-fucopyranosyl)-5-orothylamido-cyclohexane-1-carboxylic acid ethyl-2-amidyl-ethyloxy-2-acetyl-(8-amino-1,3,6-naphthalene-tris sodium sulfonate) amide
Treatment of neuromyelitis optica
Eculizumab
Eculizumab
Treatment of extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma)
Idelalisib
Idelalisib
Treatment of splenic marginal zone lymphoma
Idelalisib
Idelalisib
June 2013
Treatment of cystic fibrosis
4,6,4’–trimethylangelicin
4,6,4’–trimethylangelicin
Treatment of 5q spinal muscular atrophy
5-[1-(2,6-dichlorobenzyl)piperidin-4-ylmethoxy]quinazoline-2,4-diamine dihydrochloride
5-[1-(2,6-dichlorobenzyl)piperidin-4-ylmethoxy]quinazoline-2,4-diamine dihydrochloride
Treatment of amyotrophic lateral sclerosis
Sodium chlorite
Sodium chlorite
Treatment of retinitis pigmentosa
Expanded human allogeneic neural retinal progenitor cells extracted from neural retina
Expanded human allogeneic neural retinal progenitor cells extracted from neural retina
Treatment of pachyonychia congenital
Synthetic double-stranded siRNA oligonucleotide directed against the keratin 6a N171K mutation
Synthetic double-stranded siRNA oligonucleotide directed against the keratin 6a N171K mutation
Treatment of retinitis pigmentosa
Adenovirus associated viral vector serotype 5 containing the human pde6β gene
Adenovirus associated viral vector serotype 5 containing the human pde6β gene
Treatment of acute liver failure
Immortalised human C3A hepatoblastoma cells
Immortalised human C3A hepatoblastoma cells
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Recombinant human alpha-N-acetylglucosaminidase
Recombinant human alpha-N-acetylglucosaminidase
Treatment of soft tissue sarcoma
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 retinitis pigmentosa
Unoprostone isopropyl
Unoprostone isopropyl
Treatment of non-dystrophic myotonia
Mexiletine hydrochloride
Mexiletine hydrochloride
Treatment of B-cell acute lymphoblastic leukaemia
Inotuzumab ozogamicin
Inotuzumab ozogamicin
Treatment of adrenocortical carcinoma
N-[2,6-bis(1-methylethyl)phenyl]-N’-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt
N-[2,6-bis(1-methylethyl)phenyl]-N’-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt
Treatment 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 transglutaminase-1-deficient autosomal recessive congenital ichthyosis
Recombinant human transglutaminase 1 encapsulated into liposomes
Recombinant human transglutaminase 1 encapsulated into liposomes
Treatment of cystic fibrosis
Recombinant human CXCL8 mutant
Recombinant human CXCL8 mutant
Treatment of malignant mesothelioma
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 cytomegalovirus disease in patients with impaired cell mediated immunity
Maribavir
Maribavir
Treatment of adenosine deaminase-deficient-severe combined immunodeficiency
Autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene
Autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene
Treatment of retinitis pigmentosa
Recombinant human nerve growth factor
Recombinant human nerve growth factor
April 2013
Treatment of follicular thyroid cancer
Lenvatinib
Lenvatinib
Treatment of glioma
4-[2-(6-methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate
4-[2-(6-methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate
Treatment of papillary thyroid cancer
Lenvatinib
Lenvatinib
Treatment of Duchenne muscular dystrophy
R,S-O-(3-piperidino-2-hydroxy-1-propyl)-nicotinic acid amidoxime dihydrochloride
R,S-O-(3-piperidino-2-hydroxy-1-propyl)-nicotinic acid amidoxime dihydrochloride
Treatment of idiopathic pulmonary fibrosis
Nintedanib
Nintedanib
Treatment of Niemann-Pick disease, type C
2-hydroxypropyl-β-cyclodextrin
2-hydroxypropyl-β-cyclodextrin
Treatment of chronic lymphocytic leukaemia/small lymphocytic lymphoma
(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one
(S)-3-(1-(9H-purin-6-ylamino)ethyl)-8-chloro-2-phenylisoquinolin-1(2H)-one
March 2013
Treatment of neuronal ceroid lipofuscinosis type 2
Recombinant human tripeptidyl-peptidase 1
Recombinant human tripeptidyl-peptidase 1
Treatment of Stargardt’s disease
Ramiprilat
Ramiprilat
Treatment of Churg-Strauss Syndrome
Mepolizumab
Mepolizumab
Treatment of mantle cell lymphoma
1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H- pyrazolo [3,4-d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one
1-[(3R)-3-[4-amino-3-(4-phenoxyphenyl)-1H- pyrazolo [3,4-d]pyrimidin-1-yl]-1-piperidinyl]-2-propen-1-one
Treatment of congenital alpha-1 antitrypsin deficiency
Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-Lglutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl] acetate salt
Cyclo[L-alanyl-L-seryl-L-isoleucyl-L-prolyl-L-prolyl-Lglutaminyl-L-lysyl-L-tyrosyl-D-prolyl-L-prolyl-(2S)-2-aminodecanoyl-L-alpha-glutamyl-L-threonyl] acetate salt
Prevention of graft rejection following solid organ transplantation
Murine IgM monoclonal antibody binding to alpha beta T-cell receptor
Murine IgM monoclonal antibody binding to alpha beta T-cell receptor
Treatment of sickle cell disease
Poloxamer 188
Poloxamer 188
Treatment of chronic non-infectious uveitis
Gevokizumab
Gevokizumab
Treatment of Niemann-Pick disease, type C
Recombinant human heat shock protein 70
Recombinant human heat shock protein 70
Treatment of hepatocellular carcinoma
4-[2-(6-methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate
4-[2-(6-methylpyridin-2-yl)-5,6-dihydro-4H-pyrrolo[1,2-b]pyrazol-3-yl]-quinoline-6-carboxamide monohydrate
Treatment of systemic sclerosis
2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid
2-[4-Methoxy-3-(2-m-tolyl-ethoxy)-benzoylamino]-indan-2-carboxylic acid
February 2013
Treatment of achromatopsia caused by mutations in the CNGB3 gene
Recombinant adeno-associated viral vector containing the human CNGB3 gene
Recombinant adeno-associated viral vector containing the human CNGB3 gene
Treatment of amyloid light-chain amyloidosis
Humanised IgG1 kappa antibody against serum amyloid A and AL amyloid
Humanised IgG1 kappa antibody against serum amyloid A and AL amyloid
Treatment of moderate and severe traumatic brain injury
Progesterone
Progesterone
Treatment of high altitude pulmonary oedema
Cyclo-Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys
Cyclo-Cys-Gly-Gln-Arg-Glu-Thr-Pro-Glu-Gly-Ala-Glu-Ala-Lys-Pro-Trp-Tyr-Cys
Treatment of chronic thromboembolic pulmonary hypertension
Treprostinil sodium
Treprostinil sodium
Treatment of systemic sclerosis
Terguride
Terguride
Treatment of Duchenne muscular dystrophy
Humanised monoclonal antibody against myostatin
Humanised monoclonal antibody against myostatin
Treatment of acute myeloid leukaemia
L-asparaginase encapsulated in erythrocytes
L-asparaginase encapsulated in erythrocytes
January 2013
Treatment of follicular lymphoma
Lenalidomide
Lenalidomide
Treatment of familial adenomatous polyposis
Eflornithine in combination with sulindac
Eflornithine in combination with sulindac
Treatment of growth hormone deficiency
Recombinant modified human growth hormone
Recombinant modified human growth hormone
Treatment of 5q spinal muscular atrophy
Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
Treatment of Wilson’s disease
Choline tetrathiomolybdate
Choline tetrathiomolybdate
Treatment of pancreatic cancer
Recombinant human monoclonal antibody of the IgG1 kappa class against prostate stem cell antigen
Recombinant human monoclonal antibody of the IgG1 kappa class against prostate stem cell antigen
Treatment of beta-thalassaemia intermedia and major
Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
Autologous CD34+ haematopoietic stem cells transduced with lentiviral vector encoding the human betaA-T87Q-globin gene
Treatment of ovarian cancer
Chimeric monoclonal antibody against claudin 6
Chimeric monoclonal antibody against claudin 6
Treatment of glioma
1,2:5,6-Dianhydrogalactitol
1,2:5,6-Dianhydrogalactitol
Treatment of achondroplasia
Modified recombinant human C-type natriuretic peptide
Modified recombinant human C-type natriuretic peptide
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene
Adeno-associated viral vector serotype 9 containing the human N-acetylglucosaminidase alpha gene
Treatment of systemic sclerosis
Terguride
Terguride
Treatment of retinitis pigmentosa
Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor
Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor
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