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
Lonafarnib Hepatitis D Eiger Biopharmaceuticals, Inc.;PO Box 430;San Carlos, California, 94070 November 19, 2013
Encorafenib Stage IIB-IV melanoma positive for BRAF mutation Array BioPharma, Inc.;125 Cambridge Park Drive, Suite 301;Cambridge, Massachusetts, 02140 November 19, 2013
Encorafenib + binimetinib Treatment in Stage IIB-IV melanoma positive for the BRAF mutation Array BioPharma, Inc.;125 Cambridge Park Drive, Suite 301;Cambridge, Massachusetts, 02140 November 19, 2013
Autologous CD4+CD25hiFoxP3+regulatory T cells Prevention of graft rejection following solid organ transplantation iREG Medical AB;Tallmov 2A,;Uppsala November 19, 2013
Binimetinib Treatment Stage IIB-IV melanoma Array BioPharma, Inc.;125 Cambridge Park Drive, Suite 301;Cambridge, Massachusetts, 02140 November 19, 2013
2-[(1R,6R)-3-Methyl-6-(1-methylethenyl)-2-cyclohexen-1-yl]-5-pentyl-1,3-benzenediol Dravet Syndrome GW Pharma Ltd.;Porton Down Science Park, Salisbury, Wiltshire SP4 0JQ; November 14, 2013
Cannabidiol Dravet Syndrome GW Pharma Ltd.;Porton Down Science Park, Salisbury, Wiltshire SP4 0JQ; November 14, 2013
Eltrombopag Aplastic anemia aplastic anemia Novartis Pharmaceuticals Corp.;One Health Plaza;East Hanover, New Jersey, 07936 November 8, 2013
Flubendazole Onchocerciasis Janssen Research & Development, LLC;920 Route 202 South;Raritan, New Jersey, 08869 October 25, 2013
TXA127 Acute Radiation Syndrome Acute radiation syndrome US Biotest, Inc.;231 Bonetti Dr.,, Suite 240;San Luis Obispo, California, 93401 October 25, 2013
3-chloro-4-fluorophenyl-[4-fluoro-4-[[(5-methylpyrimidin-2-ylmethyl)amino]methyl]piperidin-1yl]methanone Rett syndrome Neurolixis, Inc.;3210 Merryfield Row;San Diego, California, 92121 October 25, 2013
Gallium-68 (DOTA0-Phel-Tyr3)octreotide The management of neuroendocrine tumors University of Iowa Hospital & Clinics;Department of Radiology/PET Imaging Center, 200 Hawkins Drive;Iowa City, Iowa, 52242 October 25, 2013
PEGylated recombinant anti-Pseudomonas aeruginosa PcrV Fab’ antibody Cystic fibrosis KaloBios Pharmaceuticals, Inc.;442 Littlefield Avenue;South San Francisco, California, 94080 October 25, 2013
Somavaratan Growth hormone deficiency Versartis, Inc.;275 Shoreline Drive, Suite 450;Redwood City, 94065 October 16, 2013
6-amino-5-chloro-N-[1R)-1-[5-[[[5-hloro-4-(trifluoromethyl)-2pyridinyl]amino]carbonyl]-2-thiazoyl]ethyl]-4-pyrimdinecarboxamide Stage IIb-IV melanoma Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 October 15, 2013
Ceritinib Non-small cell lung cancer Novartis Pharmaceuticals Corp.;One Health Plaza;East Hanover, New Jersey, 07936 September 27, 2013
1-(4-benzhydrylpiperazin-1-yl)-3,3-diphenylpropan-1-one Management of postherpetic neuralgia Zalicus Pharmaceuticals Ltd.;245 First Street;Cambridge, Massachusetts, 02142 September 26, 2013
Bacterium Bacteroides thetaiotaomicron pediatric Crohn’s disease 4D Pharma Research Ltd;Life Sciences Innovation Bldg., Cornhill Rd.; September 26, 2013
Ethiodized oil injection Hepatocellular Carcinoma Guerbet LLC;120 West 7th St., Suite 108;Bloomington, Indiana, 47404 September 26, 2013
Chimeric monoclonal antibody against claudin-18 splice variant 2 Pancreatic Cancer Astellas Pharma Global Development Inc.;1 Astellas Way;Northbrook, Illinois, 60062 September 26, 2013
6,8-bis-benzylsulfanyl-octanoic acid Myelodysplastic syndrome Cornerstone Pharmaceuticals, Inc.;25 Health Science Drive;Stony Brook, New Mexico, 11790 September 26, 2013
Dovitinib Adenoid cystic carcinoma Novartis Pharmaceuticals Corporation;One Health Plaza, Bldg 315;East Hanover, New Jersey, 07936 September 26, 2013
Manganese (II) chloride tetrahydrate (with L-alanine and vitamin D3 as promoters of absorption) Use as a targeted contrast agent for diagnostic MRI for the detection and localization of focal liver lesions in patients where use of gadolinium based contrast agents may be medically inadvisable or where gadolinium based contrast agents cannot be administered Ascelia Pharma AB;Scion DTU, DK-2800 Lyngby; September 26, 2013
Anti-sense oligonucleotide consisting of 2ÿƿÿ⿿ÿ⿿ÿ¢ÿƿÿ¢ÿ¢â⿿¬ÿ¡ÿ⿿ÿ¬ÿƿÿ¢ÿ¢â⿿¬ÿ¾ÿ⿿ÿ¢0-Me RNA with a phosphorothioate backbone Cystic fibrosis ProQR Therapeutics III B.V.;Zernikedreef 9, 2333 CK Leiden, The Netherlands; September 23, 2013
Anti-sense oligonucleotide consisting of 2’0-Me RNA with a phosphorothioate backbone Cystic fibrosis ProQR Therapeutics B.V.;Darwinweg 24, 2333 CR Leiden, The Netherlands; September 23, 2013