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
Everolimus Stomach Cancer Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 May 24, 2011
Camostat Chronic Pancreatitis NIXS Corporation;Nichi-Iko Pharmaceutical Co., Ltd., 7th Floor - NOF Nihonbashi Honcho Bldg;Tokyo May 18, 2011
Adalimumab Ulcerative colitis AbbVie, Inc.;1 N. Waukegan Road;North Chicago, Illinois, 60064 May 11, 2011
Acadesine Multiple myeloma Advancell-Advanced In Vitro Cell Technologies S.A.;Via Augusta 59, 3rd floor, door 313;Barcelona May 4, 2011
Humanized monoclonal antibody to TumorEndothelial Marker-1 Soft tissue sarcoma Morphotek;210 Welsh Pool Rd;Exton, Pennsylvania, 19341 April 29, 2011
Ferumoxytol For use in MR imaging for the mangement of brain tumors Edward A. Neuwelt, MD;Oregon Health & Science University, 3181 SW Sam Jackson Park Road - Mail Code L603;Portland, Oregon, 97239 April 29, 2011
Clarithromycin, rifabutin, clofazimine Pediatric Crohn's disease RedHill Biopharma Ltd.;42 Givati St.; April 26, 2011
Methyl O-4-O-[2-[2-[2-[2-[[N-[(1R)-1-[[4-(aminoiminomethyl)phenyl]methyl]-2-oxo-2-(1-piperidinyl)ethyl]-N2-[(4-methoxy-2,3,6-trimethylphenyl)sulfonyl]-L-Ã?ÂÃÃ?Ã?Â&Ã?ýÃ?ÂÃÃ?ÂÃ?ñ-aspararginyl-4-aminobutanoyl-N6-[5-[(3aS,4S,6aR)-hexahydro-2-oxo-1H-thieno[ Prevention of ischemia reperfusion injury associated wth solid organ transplantation Endotis Pharma;102, avenue Gaston Roussel; April 18, 2011
Tissue Plasminogen activator Acute ischemic stroke (AIS) in children age 16 years and younger Catherine Amlie-Lefond, MD;University of Washington, 4800 Sand Point Way NE - M/S B5552;Seattle, Washington, 98105 April 18, 2011
4,5,6,7-tetrachloro-2′,4′,5′,7′-tetraiodofluorescein disodium salt Hepatocellular Carcinoma Provectus Pharmaceuticals, Inc.;7327 Oak Ridge Highway;Knoxville, Tennessee, 37931 April 18, 2011
Recombinant humanized anti-LOXL2 monoclonal antibody Idiopathic pulmonary fibrosis Gilead Sciences, Inc.;199 East Blaine St.;Seattle, Washington, 98102 April 18, 2011
DNA plasmid encoding the acetylcholine receptor Myasthenia gravis Myasthenia gravis Bayhill Therapeutics;1804 Embarcadero Rd., Suite 201;Palo Alto, California, 94303 April 18, 2011
Etirinotecan pegol Ovarian cancer Nektar Therapeutics;455 Mission Bay Blvd, South;San Francisco, California, 94158 April 18, 2011
DOXO-EMCH Adenocarcinoma of the pancreas NantCell, Inc.;9920 Jefferson Boulevard;Culver City, California, 90232 April 18, 2011
Methyl O-4-O-[2-[2-[2-[2-[[N-[(1R)-1-[[4-(aminoiminomethyl)phenyl]methyl]-2-oxo-2-(1-piperidinyl)ethyl]-N2-[(4-methoxy-2,3,6-trimethylphenyl)sulfonyl]-L-�?���?�?�&�?�½�?���?��?�±-aspararginyl-4-aminobutanoyl-N6-[5-[(3aS,4S,6aR)-hexahydro-2-oxo-1H-thieno[ Prevention of ischemia reperfusion injury associated wth solid organ transplantation Endotis Pharma;102, avenue Gaston Roussel; April 18, 2011
Heptadecasodium salt of an 18-base residue phosphorothioate oligonucleotide Spinal muscular atrophy Isis Pharmaceuticals, Inc.;1896 Rutherford Road;Carlsbad, CA, 92008 April 18, 2011
Abeotaxane inhibitor of microtubules Pediatric neuroblastoma Cortice Biosciences;1345 Avenue of the Americas, 42nd Floor;New York, New York, 10105 April 18, 2011
Nusinersen Spinal muscular atrophy Biogen, Inc;225 Binney Street;Cambridge, Massachusetts, 02142 April 18, 2011
5-(3-ethyl-1,2,4-oxadiazol-5-yl)-1,4,5,6-tetrahydropyrimidine hydrochloride Progressive supranuclear palsy Mithridion, Inc.;505 Science Drive;Madison, Wisconsin, 53711 April 18, 2011
Bi-shRNAfurin and granulocyte macrophage colony stimulating factor augmented autologus tumor cell vaccine Ovarian cancer Gradalis, Inc.;2545 Golden Bear Drive;Carrollton, Texas, 75006 April 18, 2011
Firtecan pegol Neuroblastoma Enzon Pharmaceuticals, Inc.;20 Kingsbridge Road;Piscataway, New Jersey, 08854 April 18, 2011
(R)-1-phenylethyl-5-(4-biphenyl-4-cyclopropanecarboxylic acid)-3-methylisoxazole-4-yl carbamate sodium salt Idiopathic pulmonary fibrosis Bristol-Myers Squibb Company;P. O. Box 4000;Princeton, New Jersey, 08543 April 15, 2011
Synthetic double-stranded siRNA oligonucleotide directed against transthyretin mRNA Amyloidosis Alnylam UK Limited April 15, 2011
Selective deacylglycerol acyltransferase 1 inhibitor Hypertriglyceridemia in te setting of Type I hyperlipoproteinemia, also known as Familial Chylomicronemia Syndrome Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 March 28, 2011
Acetyl-l-carnitine (ALC) Rett syndrome Leadiant Biosciences, Inc.;9841 Washingtonian Blvd., Suite 500;Gaithersburg, Maryland, 20878 March 18, 2011