Treatments
The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.
You can filter the content by clicking on the titles: Product, Indication, Sponsor and Date. Clicking once, will filter the column alphabetically from A – Z and a second click on the same title will filter from Z – A.
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| Country | Product | Indication | Sponsor | Date |
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Sodium (2R,3S,5R)-5-(4-amino-2-oxo-1,3,5-triazin-1(2H)-yl)-2-(hydroxymethyl)tetrahydrofuran-3-yl ((2R,3S,5R)-5-(2-amino-6-oxo-1H-purin-9(6H)-yl)-3-hydroxytetrahydrofuran-2-yl)methyl phosphate | Acute myeloid leukaemia | Otsuka Pharmaceutical Europe Ltd. | December 14, 2015 |
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2-(2-chlorobenzylidene)hydrazinecarboximidamide acetate | Charcot-Marie-Tooth disease | Inflectis Bioscience | December 14, 2015 |
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Variant of recombinant human fibroblast growth factor 19 | Primary sclerosing cholangitis | Diamond BioPharm Limited | December 14, 2015 |
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Recombinant human monoclonal IgG1 antibody against programmed death ligand-1 | Merkel cell cancer | Merck Serono Europe Limited | December 14, 2015 |
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Imetelstat sodium | Myelofibrosis | Janssen-Cilag International NV | December 14, 2015 |
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Live attenuatedListeria monocytogenesbioengineered with a chimeric human epidermal growth factor receptor 2 fused to a truncated form of the Lm protein listeriolysin O | Pediatric osteosarcoma | Coté Orphan Consulting UK Limited | December 14, 2015 |
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Adeno-associated viral vector serotype rh10 containing the human factor IX gene | Hemophilia B | Pharma Gateway AB | December 14, 2015 |
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2-amino-2-[2-[2-chloro-4-[[3-(phenylmethoxy)phenyl]thio]phenyl]ethyl]-1,3-propanediol hydrochloride | Graft Versus Host Disease | Novartis Europharm Limited | December 14, 2015 |
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Sirolimus | Beta thalassaemia intermedia and major | Rare Partners srl Impresa Sociale | December 14, 2015 |
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Recombinant human nerve growth factor | Neurotrophic keratitis | Dompé farmaceutici S.p.A. | December 14, 2015 |
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Combretastatin A1 diphosphate | Acute myeloid leukaemia | Diamond BioPharm Limited | December 14, 2015 |
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(R)-1-[1-(4-acetoxy-3,3-dimethyl-2-oxo-butyl)-2-oxo-5-(pyridin-2-yl)-2,3-dihydro-1H-benzo[e][1,4]diazepin-3-yl]-3-(3-methylamino-phenyl)-urea | Gastro-entero-pancreatic neuroendocrine tumours | Trio Medicines Ltd | December 14, 2015 |
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(3S)-(+)-(5-chloro-2-methoxyphenyl)-1,3-dihydro-3-fluoro-6-(trifluoromethyl)-2H-indol-2-one | Fragile X syndrome | Centre National de la Recherche Scientifique (CNRS);3 Rue Michel-Ange;Paris | December 9, 2015 |
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Propagermanium and irbesartan | Focal segmental glomerulosclerosis | Dimerix Bioscience Ltd.;Level 2, 25 Flinders Lane;Melbourne | December 9, 2015 |
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Cromolyn sodium for inhalation | Mastocytosis | Patara Pharma, LLC;11455 El Camino Real, Suite 460;San Diego, California, 92130 | December 9, 2015 |
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Non-replicating recombinant adeno-associated virus (AAV) vector of serotype 9 expressing human iduronate-2-sulfatase | Hunter Syndrome | REGENXBIO, Inc.;9712 Medical Center Drive, Suite 100;Rockville, Maryland, 20850 | December 8, 2015 |
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Multi-epitope folate receptor alpha peptide vaccine with GM-CSF adjuvant (TPIV200) | Ovarian cancer | Tapimmune, Inc.;50 North Laura Street;Jacksonville, Florida, 32202 | December 7, 2015 |
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Delta-9-tetrahydrocannibinol and cannabidiol | Glioma | GW Research Ltd.;Sovereign House, Vision Park, Chivers Way;Histon | December 3, 2015 |
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Azurin-p28 | Glioma | CDG Therapeutics, Inc.;840 South Wood Street, M/C 820;Chicago, Illinois, 60612 | December 2, 2015 |
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Dodecafluoropentane emulsion (DDFPe) | Glioblastoma | NuvOx Pharma;1635 East 18th Street;Tucson, Arizona, 85719 | December 2, 2015 |
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Tirapazamine | Hepatocellular Carcinoma | Teclison Limited;28 Baltusrol Way;Short Hills, New Jersey, 07078 | November 30, 2015 |
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Temozolomide | Glioblastoma | AmpliPharm Pharmaceuticals, LLC;970 Peachtree Industrial Boulevard, Suite 100;Suwanee, Georgia, 30024 | November 25, 2015 |
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Acetyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-cysteinyl-D-glutaminyl-D-cystenyl-D-arginyl-D-arginyl-D-lysyl-D-asparaginylmaide disulfide | Acute myeloid leukemia | Genus Oncology, LLC;2458 N. Racine Avenue;Chicago, Illinois, 60614 | November 24, 2015 |
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Necitumumab | Non-small cell lung cancer | Eli Lilly and Company;33 ImClone Drive;Branchburg, New Jersey, 08876 | November 20, 2015 |
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Ubenimex | Pulmonary arterial hypertension | Eiger BioPharmaceuticals, Inc.;350 Cambridge Avenue, Suite 350;Palo Alto, California, 94306 | November 19, 2015 |