Latest marketing authorisations and orphan drug designations: Recent orphan drug designations

Latest marketing authorisations and orphan drug designations

List of the latest marketing authorisations and medicinal products designations

Detailed information on European orphan medicinal products designation applications is available on theEMA website. A full list of designated and authorised orphan medicinal products in Europe available at:ec.europa.eu.

Learn more about the Orphan designation process in Europe

Recent orphan drug designations

March 2015

Treatment of Wilson’s disease
Trientine tetrahydrochloride

Treatment of biliary tract cancer
5,10,15,20-tetrakis(2,6-difluoro-3-Nmethylsulfamoylphenyl)bacteriochlorin

Treatment of ovarian cancer
Human reovirus type 3 Dearing strain

Treatment of Gaucher disease
Adeno-associated viral vector serotype 9 containing the human glucocerebrosidase gene

Treatment of epidermolysis bullosa
Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with aCOL17A1-encoding retroviral vector

Treatment of epidermolysis bullosa
Ex-vivo-expanded autologous human keratinocytes containing epidermal stem cells transduced with aCOL7A1-encoding retroviral vector

Treatment of epidermolysis bullosa
Ex-vivo-expanded autologous human keratinocytes containing
epidermal stem cells transduced with a LAMB3-encoding retroviral vector
Treatment of systemic sclerosis
Autologous adipose tissue-derived stromal vascular fraction cells

Treatment of plasma cell myeloma
Melphalan flufenamide

Treatment of retinitis pigmentosa
Sodium 3-[(4aR,6R,7R,7aS)-7-hydroxy-2-oxido-2-sulfanylidene-4a,6,7,7a-tetrahydro-4H-furo[3,2-d][1,3,2]dioxaphosphinin-6-yl]-2-bromo-6-phenyl-5Himidazo[1,2-a]purin-9-one

Treatment of hepatocellular carcinoma
Lenvatinib

Treatment of amyotrophic lateral sclerosis
Enoxacin

Treatment of ovarian cancer
Humanised anti-folate receptor 1 monoclonal antibody conjugated to maytansinoid DM4

Treatment of tenosynovial giant cell tumour, localised and diffuse type
[5-(5-chloro-1H-pyrrolo[2,3-b]pyridin-3-ylmethyl)-pyridin-2-yl]-(6-trifluoromethyl-pyridin-3-ylmethyl)-amine hydrochloride

Prevention of bronchopulmonary dysplasia
Recombinant human club cell 10 KDa protein

Treatment of graft-versus-host disease
Human plasma-derived alpha-1 proteinase inhibitor

Treatment of Netherton syndrome
6-ethoxy-7-methoxy-2-(2-methylsulfanylphenyl)-3,1-benzoxazin-4-one

Treatment of Huntington’s disease
Chimeric 2'-O-(2-methoxyethyl) modified oligonucleotide targeted to huntingtin RNA

Treatment of fragile X syndrome
Tideglusib

Treatment of Alport syndrome
5'-ASCSASTSCSASGSTSCSTSGSASUSASASGSCSTSA-3'

Diagnosis of gastro-entero-pancreatic neuroendocrine tumours
Gallium (68Ga)-edotreotide

February 2015

Treatment of facioscapulohumeral muscular dystrophy
505 amino acid protein, corresponding to amino acids 2-506 of the wild type human histidyl-tRNA synthetase

Treatment of soft tissue sarcoma
Olaratumab

Treatment of progressive supranuclear palsy
N-(3-(4-(3-(diisobutylamino)propyl)piperazin-1-yl)propyl)-1Hbenzo[d]imidazol-2-amine disulphate salt

Treatment of acute myeloid leukaemia
Ulocuplumab

Treatment of narcolepsy
Mazindol

Treatment of glioma
Recombinant human glutamate oxaloacetate transaminase 1

Treatment of Ebola virus disease
Fibrinogen-coated albumin spheres

Treatment of sickle cell disease
5-hydroxymethyl-2-furfural

Treatment of cytomegalovirus infection following haematopoietic stem cell transplantation
Allogeneic CD4+ and CD8+ T lymphocytes ex vivo incubated with synthetic peptides of the viral antigens of cytomegalovirus, adenovirus and Epstein-Barr virus

Treatment of acute myeloid leukaemia
Alvocidib

Prevention of necrotising enterocolitis
“Lactobacillus reuteri”

Treatment of systemic sclerosis
Nitroglycerin

Treatment of ovarian cancer
Chimeric group B adenovirus (11p/3) with deletions in the E3 and E4 Regions

Treatment of sickle cell disease
Sevuparin sodium

Treatment of hereditary angioedema
3-[2-(4-carbamimidoyl-phenylcarbamoyl)-5-methoxy-4-vinyl-phenyl]-6-(cyclopropylmethyl-carbamoyl)-pyridine-2-carboxylic acid

January 2015

Treatment of congenital factor VII deficiency
Adeno-associated viral vector serotype 8 containing the human factor VII gene

Treatment of ovarian cancer
N-methyl-4-({4-[({3-methyl(methylsulfonyl)aminopyrazin-2-yl}methyl)amino]-5-(trifluoromethyl)pyrimidin-2-yl}amino)benzamide hydrochloride

Treatment of Wolfram syndrome
Sodium valproate

Treatment of hypophosphatasia
Recombinant human alkaline phosphatase

Prevention of graft-versus-host disease
Allogeneic peripheral blood mononuclear cells induced to an early apoptotic state

Treatment of spinocerebellar ataxia
Ceftriaxone

Treatment of diffuse large B-cell lymphoma
Humanised Fc engineered monoclonal antibody against CD19

Treatment of plasma cell myeloma
Synthetic signal peptide of human mucin-1 (amino acids 1-21)

Treatment of mucopolysaccharidosis type IIIB (Sanfilippo B syndrome)
Chimeric fusion protein of recombinant human alpha-Nacetylglucosaminidase and human insulin-like growth factor 2

Treatment of gastrointestinal stromal tumours
Ponatinib hydrochloride

Treatment of Aicardi-Goutières syndrome
Emtricitabine

Treatment of Aicardi-Goutières syndrome
Tenofovir disoproxil fumarate

Treatment of adult T-cell leukaemia/lymphoma
A lentiviral vector pseudotyped by the New-Jersey serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and p30II HTLV-1 proteins

Treatment of adult T-cell leukaemia/lymphoma
A lentiviral vector pseudotyped by the Indiana serotype of the vesicular stomatitis virus G protein encoding an antigen derived from the Tax, HBZ, p12I and p30II HTLV-1 proteins

Treatment of neuroblastoma
Chimeric monoclonal antibody to O-acetyl-GD2 antigen

Treatment of placental insufficiency
Adenoviral vector serotype 5 containing the vascular endothelial growth factor D isoform (preprocessed short form) from a CMV promoter

Treatment for calciphylaxis
Sodium thiosulfate

Treatment of acute myeloid leukaemia
Allogeneic, umbilical cord blood-derived, ex vivo-expanded, haematopoietic CD133+ cells / allogeneic, umbilical cord blood-derived, non-expanded, haematopoietic CD133- cells

Treatment of pancreatic cancer
Herpes simplex type 1 virus containing cellular B-myb gene as tumour specific promoter

Treatment of interstitial cystitis
Pentosan polysulfate sodium

Treatment of aspartylglucosaminuria
Recombinant human aspartylglucosaminidase

Treatment of malignant mesothelioma
Pegylated recombinant arginine deiminase

December 2014

Treatment of glioma
5,5'-(4-(trifluromethyl)benzylazanediyl)bis(methylene)diquinolin-8-ol

Treatment of epidermolysis bullosa
Allogeneic adipose-derived adult mesenchymal stem cells contained in a fibrin-based bioengineered dermis

Treatment of inborn errors of primary bile acid synthesis
Chenodeoxycholic acid

Treatment of non-infectious uveitis
Autologous collagen type II-specific regulatory T cells

Treatment of multiple system atrophy
1-(2-isopropoxyethyl)-2-thioxo-1,2,3,5-tetrahydro-pyrrolo[3,2-d]pyrimidin-4-one

Treatment of WHIM syndrome
Plerixafor

Treatment of beta-thalassaemia intermedia and major
Benserazide hydrochloride

Treatment of traumatic spinal cord injury
Riluzole

Treatment of neurotrophic keratitis
(1S,4R,5R,7S)-3,4-dibenzyl-2-oxo-6,8-dioxa-3-azabyciclo[3.2.1]octane-7-carboxylic acid-L-lysine

Treatment of amyotrophic lateral sclerosis
Edaravone

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

Treatment of Pseudomonas aeruginosa lung infection in cystic fibrosis
Amikacin sulfate

Treatment of glioma
Adenovirus serotype 5 containing partial E1A deletion and an integrin-binding domain

Treatment of acute myeloid leukaemia
Allogeneic ex vivo-generated natural killer cells from CD34+ umbilical cord blood progenitor cells

Treatment of pancreatic cancer
Pegylated recombinant human hyaluronidase PH20

Treatment of diffuse large B cell lymphoma
Autologous T cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3 zeta chimeric antigen receptor

Treatment of Huntington’s disease
5-bromo-N-(prop-2-yn-1-yl)-2-(1H-1,2,4-triazol-1-yl)pyrimidine-4,6-diamine

Treatment of malignant mesothelioma
5-[8-methyl-9-(1-methylethyl)-2-(4-morpholinyl)-9H-purin-6-yl]-2-pyrimidinamine

Treatment of hereditary haemorrhagic telangiectasia
Bevacizumab

Treatment of mucopolysaccharidosis type IIIA (Sanfilippo A syndrome)
Adeno-associated viral vector serotype 10 carrying the human Nsulfoglucosamine sulfohydrolase cDNA

Prevention of graft-versus-host disease
Allogeneic bone marrow derived mesenchymal cells expanded ex vivo in synthetic media

Treatment of familial cerebral cavernous malformations
Exisulind

Treatment of ovarian cancer
2-hydroxymethyl-2-methoxymethyl-1-azabicyclo[2,2,2]octan-3-one

Treatment of pancreatic cancer
Heat-killed Mycobacterium obuense (whole cell)

Treatment of WHIM syndrome
((E)-1-(4'-chlorophenyl)-3-(4-hydroxy-3-metoxyphenyl)prop-2-en-1-one)

Treatment of pleural empyema
Single-chain urokinase plasminogen activator

Treatment of acute lymphoblastic leukaemia
Allogeneic CD34+ cells expanded ex vivo with an aryl hydrocarbon receptor antagonist

November 2014

Treatment of Duchenne muscular dystrophy
Adeno-associated viral vector serotype 8 containing the human MD1 gene

Treatment of mucopolysaccharidosis type I
Ataluren

Treatment of malignant hyperthermia
Dantrolene sodium

Treatment of hepatocellular carcinoma
Diaspirin cross-linked haemoglobin

Treatment of Niemann-Pick disease, type C
Arimoclomol citrate

Treatment of xeroderma pigmentosum
Pro-Pro-Thr-Val-Pro-Thr-Arg

Treatment of Gaucher disease
(3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate

Treatment of granulomatosis with polyangiitis
(2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide

Treatment of microscopic polyangiitis
(2R,3S)-2-(4-cyclopentylaminophenyl)-1-(2-fluoro-6-methylbenzoyl)piperidine-3-carboxylic acid(4-methyl-3-trifluoromethylphenyl)amide

Treatment of systemic sclerosis
Humanised IgG1 monoclonal antibody against human eotaxin-2

Treatment of polymyositis
Siponimod

Treatment of dermatomyositis
Siponimod

Treatment of fibrodysplasia ossificans progressiva
Palovarotene

Treatment of hereditary haemorrhagic telangiectasia
Bazedoxifene acetate

Treatment of primary myelofibrosis
Recombinant human pentraxin-2

Treatment of post-polycythaemia vera myelofibrosis
Recombinant human pentraxin-2

Treatment of glioma
Olaptesed pegol

Treatment of idiopathic pulmonary fibrosis
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid

Treatment of systemic sclerosis
1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid

Treatment of haemophilia A
A combination of H-Lys-Lys-Gly-Pro-Arg-Cys(SH)-Leu-Thr-Arg-Tyr-Tyr-Ser-Ser-Phe-Val-Asn-Met-Glu-Gly-Lys-Lys-OH and H-Lys-Lys-Gly-Asp-Asn-Ile-Met-Val-Thr-Phe-Arg-Asn-Gln-Ala-Ser-Arg-Pro-Tyr-Gly-Lys-Lys-OH

Treatment of mucopolysaccharidosis type I
Pentosan polysulfate sodium

Treatment of post-essential thrombocythaemia myelofibrosis
Recombinant human pentraxin-2

Treatment of acute respiratory distress syndrome
Imatinib

Treatment of acute myeloid leukaemia
Donor T lymphocytes depleted ex vivo of host alloreactive T cells using photodynamic treatment

Treatment of plasma cell myeloma
Selinexor

Treatment of chronic lymphocytic leukaemia / small lymphocytic lymphoma
Selinexor

Treatment of myotonic disorders
Mexiletine hydrochloride

October 2014

Treatment of inhalation anthrax disease
Raxibacumab

Treatment of X-linked hypophosphataemia
Recombinant human monoclonal IgG1 antibody for fibroblast growth factor 23

Treatment of tenosynovial giant cell tumour, localised and diffused type
Recombinant human monoclonal antibody of the IgG1 kappa class against human macrophage colony-stimulating factor

Treatment of mucopolysaccharidosis type I
Recombinant human insulin receptor monoclonal antibody-fused-α-Liduronidase

Treatment of glioma
Recombinant human bone morphogenetic protein 4

Treatment of pyridoxamine 5'-phosphate oxidase deficiency
Pyridoxal 5'-phosphate

Treatment of short bowel syndrome
Oxalobacter formigenes strain HC-1

Treatment of Cushing's syndrome
Osilodrostat

Treatment of cystic fibrosis
Nitric oxide

Treatment of pancreatic cancer
Immunoglobulin G1, anti-(human tumour-associated calcium signaltransducer 2)(human-Mus musculus monoclonal hRS7 heavy chain), disulfide with human-Musmusculus monoclonal hRS7 k-chain, dimer, hexakis(thioether) with (4S)-4-[[[[4-[[(2S)-2-(4-aminobutyl)-2-[[2-[2-[[26-[4-[[[[4-[(3-mercapto-2,5-dioxo-1-pyrrolidinyl)methyl]cyclohexyl]carbonyl]amino]methyl]-1H-1,2,3-triazol-1-yl]-3,6,9,12,15,18,21,24-octaoxahexacos-1-yl]amino]-2-oxoethoxy]acetyl]amino]-1-oxoethyl]amino]phenyl]methoxy]carbonyl]oxy]-4,11-diethyl-9-hydroxy-1Hpyrano[3',4':6,7]indolizino[1,2-b]quinoline-3,14(4H,12H)-dione

Treatment of congenital hyperinsulinism
Glucagon

Treatment of cystinosis
Cysteamine hydrochloride

Treatment of limbal stem cell deficiency
Cultured allogeneic corneal limbal stem cells

Treatment of Dravet syndrome
Cannabidiol

Treatment of Crigler-Najjar syndrome
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene

Treatment of fragile X syndrome
Acamprosate calcium

Treatment of Leigh syndrome
(S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride

Treatment of pemphigus
(S)-2-(1-((6-amino-5-cyanopyrimidin-4-yl)amino)ethyl)-4-oxo-3-phenyl-3,4-dihydropyrrolo[2,1-f][1,2,4]triazine-5-carbonitrile

August 2014

Treatment of cystic fibrosis
Lumacaftor / ivacaftor

Prevention of ischaemia reperfusion injury associated with solid organ transplantation
Macromolecular conjugate of heparin sodium on a polymer backbone

Treatment of myelodysplastic syndromes
Recombinant fusion protein consisting of a modified form of the extracellular domain of human activin receptor IIB linked to the human IgG1 Fc domain

Treatment of primary biliary cirrhosis
Vector lentiviral que conteniene el gen piruvato quinasa humano de hígado y eritroide (PKLR)

Treatment of acute myeloid leukaemia
4-{[(2R,3S,4R,5S)-4-(4-chloro-2-fluoro-phenyl)-3-(3-chloro-2-fluorophenyl)-4-cyano-5-(2,2-dimethyl-propyl)-pyrrolidine-2-carbonyl]-amino}-3-methoxy-benzoic acid

Treatment of paroxysmal nocturnal haemoglobinuria
S3,S13-cyclo(D-tyrolsyl-L-isoleucyl-L-cysteinyl-L-valyl-1-methyl-Ltryptophyl-L-glutaminyl-L-aspartyl-L-tryptophyl-N-methyl-L-glycyl-L-alanyl-L-histidyl-Larginyl-L-cysteinyl-N-methyl-L-isoleucinamide)

Treatment of mitochondrial neurogastrointestinal encephalomyopathy
Vector based on an adeno-associated virus serotype 2 backbone, pseudoserotyped with a type 8 capsid, which carries the coding sequence of the human TYMP gene under the control of the human thyroxine binding globulin promoter

Treatment of diffuse large B-cell lymphoma
Obinutuzumab

Treatment of small cell lung cancer
2-(2-methyl-5-nitro-1H-imidazol-1-yl)ethylsulfamide

Treatment of pancreatic cancer
[5-amino-1-(4-fluoro-phenyl)-1H-pyrazol-4-yl]-[3-(2,3-dihydroxypropoxy)-phenyl]-methanone

Treatment of cutaneous T-cell lymphoma
Humanised IgG1 monoclonal antibody against human KIR3DL2

Treatment of Crigler-Najjar syndrome
Adeno-associated viral vector serotype 8 containing the human UGT1A1 gene

Treatment of mastocytosis
Recombinant human diamine oxidase

Treatment of haemophilia B
Recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin

Treatment of acute pancreatitis
Ulinastatin

Treatment of diffuse large B-cell lymphoma
(Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide

Treatment of haemophilia A
Recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin

Treatment of apolipoprotein A-I deficiency
Recombinant human apolipoprotein A-I in a complex with phospholipids

Treatment of ATP-binding cassette transporter A1 deficiency
Recombinant human apolipoprotein A-I in a complex with phospholipids

Treatment of acute myeloid leukaemia
(Z)-3-(3-(3,5-bis(trifluoromethyl)phenyl)-1H-1,2,4-triazol-1-yl)-N'-(pyrazin-2-yl)acrylohydrazide

Treatment of congenital factor VII deficiency
Recombinant factor VIIa modified with three terminal repeats derived from the β chain of human chorionic gonadotropin

Treatment of Schnitzler syndrome
Gevokizumab

Treatment of Fabry disease
(3S)-1-azabicyclo[2.2.2]oct-3-yl{2-[2-(4-fluorophenyl)-1,3-thiazol-4-yl]propan-2-yl}carbamate

Treatment of Duchenne muscular dystrophy
17α,21-dihydroxy-16α-methyl-pregna-1,4,9(11)-triene-3,20-dione

Treatment of autosomal dominant polycystic liver disease
Sodium ascorbate and menadione sodium bisulfite

Prevention of bronchopulmonary dysplasia
Retinol

July 2014

Treatment of Huntington’s disease
Cysteamine bitartrate

Treatment of glioma
Humanised recombinant monoclonal antibody against epidermal growth factor receptor conjugated to maleimidocaproyl monomethylauristatin F

Treatment of myasthenia gravis
Eculizumab

Treatment of ovarian cancer
Cediranib

Treatment of Prader-Willi syndrome
Oxytocin

Treatment of idiopathic pulmonary fibrosis
Humanised anti-alpha ν beta 6 monoclonal antibody

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