Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

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Country Product Indication Sponsor Date
Combretastatin A1 diphosphate Acute myeloid leukaemia Diamond BioPharm Limited December 14, 2015
(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
Synthetic peptide L-cysteine, L-cysteinylglycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutamyl-L-alanyl-L-lysyl-L-prolyl-L-tryptophyl-L-tyrosyl-, cyclic (1.fwdarw.17)-disulfide Pseudohypoaldosteronism type 1B Apeptico Forschung und Entwicklung GmbH December 14, 2015
[4-aminobutanoic acid-glycyl-L-glutaminyl-L-arginyl-L-.alpha.-glutamyl-L-threonyl-L-prolyl-L-.alpha.-glutamylglycyl-L-alanyl-L-.alpha.-glutamyl-L-alanyl-L-lysyl-L-prolyl-L-tryptophyl-L-tyrosyl-L-aspartyl](cyclo 1-Dgamma17) Pseudohypoaldosteronism type 1B Apeptico Forschung und Entwicklung GmbH December 14, 2015
Live attenuatedListeria monocytogenesdeltaactA/deltainlBstrain expressing human mesothelin Malignant pleural mesothelioma Aduro Biotech Holdings, Europe B.V. December 14, 2015
Bilayer engineered collagen hydrogel-based skin graft composed of autologous keratinocytes and fibroblasts Partial deep dermal and full thickness burns Voisin Consulting S.A.R.L. December 14, 2015
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
Variant of recombinant human fibroblast growth factor 19 Primary sclerosing cholangitis Diamond BioPharm Limited December 14, 2015
2-(2-chlorobenzylidene)hydrazinecarboximidamide acetate Charcot-Marie-Tooth disease Inflectis Bioscience December 14, 2015
Recombinant human monoclonal IgG1 antibody against programmed death ligand-1 Merkel cell cancer Merck Serono Europe Limited December 14, 2015
Imetelstat sodium Myelofibrosis Janssen-Cilag International NV December 14, 2015
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
(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
Propagermanium and irbesartan Focal segmental glomerulosclerosis Dimerix Bioscience Ltd.;Level 2, 25 Flinders Lane;Melbourne December 9, 2015
Cromolyn sodium for inhalation Mastocytosis Patara Pharma, LLC;11455 El Camino Real, Suite 460;San Diego, California, 92130 December 9, 2015
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
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
Delta-9-tetrahydrocannibinol and cannabidiol Glioma GW Research Ltd.;Sovereign House, Vision Park, Chivers Way;Histon December 3, 2015
Dodecafluoropentane emulsion (DDFPe) Glioblastoma NuvOx Pharma;1635 East 18th Street;Tucson, Arizona, 85719 December 2, 2015
Azurin-p28 Glioma CDG Therapeutics, Inc.;840 South Wood Street, M/C 820;Chicago, Illinois, 60612 December 2, 2015
Tirapazamine Hepatocellular Carcinoma Teclison Limited;28 Baltusrol Way;Short Hills, New Jersey, 07078 November 30, 2015
Temozolomide Glioblastoma AmpliPharm Pharmaceuticals, LLC;970 Peachtree Industrial Boulevard, Suite 100;Suwanee, Georgia, 30024 November 25, 2015
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
Necitumumab Non-small cell lung cancer Eli Lilly and Company;33 ImClone Drive;Branchburg, New Jersey, 08876 November 20, 2015
Ubenimex Pulmonary arterial hypertension Eiger BioPharmaceuticals, Inc.;350 Cambridge Avenue, Suite 350;Palo Alto, California, 94306 November 19, 2015