jueves, 18 de diciembre de 2014

Latest marketing authorisations and orphan drug designations [1/2]

Latest marketing authorisations and orphan drug designations

Eurordis, Rare Diseases Europe

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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.

Orphan drug regulatory processLearn more about the Orphan designation process in Europe




Recent orphan drug designations

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

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
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
Treatment of systemic sclerosis

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
Treatment of haemophilia A

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
Treatment of plasma cell myeloma

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
Treatment of AL amyloidosis

Recombinant monoclonal antibody to human serum amyloid P component
Treatment of AL amyloidosis

Carboxy pyrrolidine hexanoyl pyrrolidine carboxylate
Treatment of gastric cancer

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
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
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
Treatment of invasive aspergillosis

Isavuconazonium sulfate
Treatment of familial benign chronic pemphigus (Hailey-Hailey disease)

Afamelanotide
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma

Humanised Fc engineered monoclonal antibody against CD19
Treatment of Prader-Willi syndrome

Beloranib
Treatment of primary sclerosing cholangitis

Norursodeoxycholic acid
Treatment of pre-eclampsia

Recombinant human alpha-1-microglobulin
Treatment of choroideraemia

Adeno-associated viral vector serotype 2 containing the human REP1 gene
June 2014
Treatment of gastro-entero-pancreatic neuroendocrine tumours

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
Treatment of follicular lymphoma

177Lu-tetraxetan-tetulomab
Treatment of epidermolysis bullosa

Recombinant human alpha 1 chain homotrimer of type VII collagen
Treatment of glioma

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
Treatment of central retinal vein occlusion

Aganirsen
Treatment of mucormycosis

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
Treatment of choroideraemia

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
Treatment of mucopolysaccharidosis IIIA (Sanfilippo A syndrome)

Autologous CD34+ cells transduced with a lentiviral vector containing the human SGSH gene
April 2014
Treatment of lymphoplasmacytic lymphoma

Ibrutinib
Treatment of ovarian cancer

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
Treatment of ATTR amyloidosis

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
Prevention of bronchopulmonary dysplasia

Recombinant human surfactant protein D
March 2014
Treatment of ATTR amyloidosis

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
Treatment of pancreatic cancer

Cysteamine bitartrate
Prevention of graft rejection following solid organ transplantation

Ex-vivo-cultured human mesenchymal stromal cells
Prevention of graft rejection following solid organ transplantation

Eculizumab
Treatment of acute myeloid leukaemia

Volasertib
Treatment of Leber’s congenital amaurosis

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
Treatment of soft tissue sarcoma

Doxorubicin(6-maleimidocaproyl)hydrazone
Treatment of nontuberculous mycobacterial lung disease

Amikacin sulfate
Treatment of Charcot-Marie-Tooth disease type 1A

Fixed-dose combination of (R-S) baclofen, naltrexone hydrochloride and D-sorbitol

February 2014
Treatment of Farber disease

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
Treatment of epidermolysis bullosa

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
Prevention of delayed graft function after solid organ transplantation

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
Treatment of cystic fibrosis

Cysteamine
Treatment of Duchenne muscular dystrophy

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
Treatment of polycythaemia vera

Ruxolitinib
Treatment of adrenoleukodystrophy

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
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)
Treatment of optic neuritis

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
January 2014
Treatment of malignant mesothelioma

Amatuximab
Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma

Inecalcitol
Treatment of aneurysmal subarachnoid haemorrhage

Sodium nitrite
Treatment of hepatitis delta virus infection

Lonafarnib
Treatment of dystrophic myotonia

(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
Treatment of primary sclerosing cholangitis

Obeticholic acid
Treatment of malignant mesothelioma

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
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
Treatment of epidermolysis bullosa

Allantoin
Prevention of graft-versus-host disease

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

December 2013
Treatment of cystic fibrosis

Nitric oxide
Treatment of hypoparathyroidism

Recombinant human parathyroid hormone
Treatment of glioma

Allogeneic and autologous haptenised and irradiated cells and cell lysates derived from glioma
Treatment of follicular lymphoma

Ibrutinib
Prevention of necrotizing enterocolitis

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
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
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
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
Prevention of arteriovenous access dysfunction in haemodialysis patients

Recombinant human type I pancreatic elastase
Treatment of Dravet syndrome

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]
Treatment of haemophilia A

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
Treatment of mucopolysaccharidosis type II (Hunter's syndrome)

Recombinant human insulin receptor monoclonal antibody-fusediduronate 2-sulfatase
Treatment of follicular thyroid cancer

Sorafenib tosylate
Treatment of papillary thyroid cancer

Sorafenib tosylate
Prevention of graft-versus-host disease

Defibrotide
Treatment of hepatocellular carcinoma

Tivantinib
Treatment of diffuse large B-cell lymphoma

Ibrutinib
Prevention of arteriovenous access dysfunction in patients undergoing surgical creation of an arteriovenous access for haemodialysis

Sirolimus
Treatment of eosinophilic oesophagitis

Human monoclonal antibody against human interleukin 13
Treatment of spinal cord injury

Synthetic 12 amino acids peptide designed after subcommissural organ spondin
Treatment of ovarian cancer

Trebananib
Treatment of Stargardt’s disease

Soraprazan
October 2013
Treatment of congenital factor VII deficiency

Recombinant fusion protein linking coagulation factor VIIa with Albumin
Treatment of myotonic disorders

Mexiletine hydrochloride
Treatment of plasma cell myeloma

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
Treatment of complex regional pain syndrome

Zoledronic acid
Treatment of Allan-Herndon-Dudley syndrome

3,5-diiodothyropropionic acid
Treatment of Duchenne muscular dystrophy

Naproxcinod
Treatment of cystic fibrosis

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
July & August 2013
Treatment of pulmonary alveolar proteinosis

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
Prevention of ischaemia/reperfusion injury associated with solid organ transplantation

Human hemin
Treatment of B-lymphoblastic leukaemia/lymphoma

Moxetumomab pasudotox
Treatment of malignant thymomas

Belinostat
Treatment of pancreatic cancer

(1-methyl-2-nitro-1H-imidazole-5-yl) methyl N,N’-bis(2-bromoethyl) diamidophosphate
Treatment of plasma cell myeloma

Daratumumab
Treatment of ovarian cancer

Fosbretabulin tromethamine
Treatment of amyotrophic lateral sclerosis

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
Treatment of follicular lymphoma

(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
Treatment of carbamoylphosphate synthase-1 deficiency

Heterologous human adult liver-derived progenitor cells
Treatment of citrullinaemia type 1

Heterologous human adult liver-derived progenitor cells
Treatment of argininosuccinic aciduria

Heterologous human adult liver-derived progenitor cells
Treatment of hyperargininaemia

Heterologous human adult liver-derived progenitor cells
Treatment of N-acetylglutamate synthetase (NAGS) deficiency

Heterologous human adult liver-derived progenitor cells
Treatment of citrullinaemia type 2

Heterologous human adult liver-derived progenitor cells
Treatment of ornithine translocase deficiency (hyperornithinaemia-hyperammonaemia homocitrullinuria (HHH) syndrome)

Heterologous human adult liver-derived progenitor cells
Treatment of limbal stem cell deficiency

Ex-vivo expanded autologous human corneal epithelium containing stem cells
Treatment of osteosarcoma

Lipid-complexed cisplatin
Treatment of acromegaly

Octreotide acetate (oral use)
Treatment of nodal marginal zone lymphoma

Idelalisib
Treatment of chronic lymphocytic leukaemia/small lymphocytic lymphoma

Idelalisib
Treatment of acute myeloid leukaemia

Trans-N1-((1R,2S)-2-phenylcyclopropyl)cyclohexane-1,4-diamine bis-hydrochloride
Treatment of autosomal dominant polycystic kidney disease

Tolvaptan
Treatment of nontraumatic osteonecrosis

Human allogeneic bone marrow derived osteoblastic-like cells
Treatment of pancreatic cancer

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
Treatment of growth hormone deficiency

Recombinant human growth hormone modified by fusion with two hydrophilic polypeptide chains
Treatment of Behçets' disease

Apremilast
Treatment of eosinophilic oesophagitis

Budesonide
Treatment of mastocytosis

Cladribine
Treatment of congenital sucrase-isomaltase deficiency

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
Treatment of neuromyelitis optica

Eculizumab
Treatment of extranodal marginal zone lymphoma of mucosa-associated lymphoid tissue (MALT lymphoma)

Idelalisib
Treatment of splenic marginal zone lymphoma

Idelalisib

June 2013
Treatment of cystic fibrosis

4,6,4’–trimethylangelicin
Treatment of 5q spinal muscular atrophy

5-[1-(2,6-dichlorobenzyl)piperidin-4-ylmethoxy]quinazoline-2,4-diamine dihydrochloride
Treatment of amyotrophic lateral sclerosis

Sodium chlorite
Treatment of retinitis pigmentosa

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
Treatment of retinitis pigmentosa

Adenovirus associated viral vector serotype 5 containing the human pde6β gene
Treatment of acute liver failure

Immortalised human C3A hepatoblastoma cells
Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)

Recombinant human alpha-N-acetylglucosaminidase
Treatment of soft tissue sarcoma

Genetically modified serotype 5/3 adenovirus coding for granulocyte-macrophage colony-stimulating factor
Treatment of retinitis pigmentosa

Unoprostone isopropyl
Treatment of non-dystrophic myotonia

Mexiletine hydrochloride
Treatment of B-cell acute lymphoblastic leukaemia

Inotuzumab ozogamicin
Treatment of adrenocortical carcinoma

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
Treatment of transglutaminase-1-deficient autosomal recessive congenital ichthyosis

Recombinant human transglutaminase 1 encapsulated into liposomes
Treatment of cystic fibrosis

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
Treatment of cytomegalovirus disease in patients with impaired cell mediated immunity

Maribavir
Treatment of adenosine deaminase-deficient-severe combined immunodeficiency

Autologous CD34+ cells transduced with a lentiviral vector containing the human ADA gene
Treatment of retinitis pigmentosa

Recombinant human nerve growth factor

April 2013
Treatment of follicular thyroid cancer

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
Treatment of papillary thyroid cancer

Lenvatinib
Treatment of Duchenne muscular dystrophy

R,S-O-(3-piperidino-2-hydroxy-1-propyl)-nicotinic acid amidoxime dihydrochloride
Treatment of idiopathic pulmonary fibrosis

Nintedanib
Treatment of Niemann-Pick disease, type C

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
March 2013
Treatment of neuronal ceroid lipofuscinosis type 2

Recombinant human tripeptidyl-peptidase 1
Treatment of Stargardt’s disease

Ramiprilat
Treatment of Churg-Strauss Syndrome

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
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
Prevention of graft rejection following solid organ transplantation

Murine IgM monoclonal antibody binding to alpha beta T-cell receptor
Treatment of sickle cell disease

Poloxamer 188
Treatment of chronic non-infectious uveitis

Gevokizumab
Treatment of Niemann-Pick disease, type C

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
Treatment of systemic sclerosis

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
Treatment of amyloid light-chain amyloidosis

Humanised IgG1 kappa antibody against serum amyloid A and AL amyloid
Treatment of moderate and severe traumatic brain injury

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
Treatment of chronic thromboembolic pulmonary hypertension

Treprostinil sodium
Treatment of systemic sclerosis

Terguride
Treatment of Duchenne muscular dystrophy

Humanised monoclonal antibody against myostatin
Treatment of acute myeloid leukaemia

L-asparaginase encapsulated in erythrocytes
January 2013
Treatment of follicular lymphoma

Lenalidomide
Treatment of familial adenomatous polyposis

Eflornithine in combination with sulindac
Treatment of growth hormone deficiency

Recombinant modified human growth hormone
Treatment of 5q spinal muscular atrophy

Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
Treatment of Wilson’s disease

Choline tetrathiomolybdate
Treatment of pancreatic cancer

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
Treatment of ovarian cancer

Chimeric monoclonal antibody against claudin 6
Treatment of glioma

1,2:5,6-Dianhydrogalactitol
Treatment of achondroplasia

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
Treatment of systemic sclerosis

Terguride
Treatment of retinitis pigmentosa

Encapsulated human retinal pigment epithelial cell line transfected with plasmid vector expressing human ciliary neurotrophic factor

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