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
Salmonella Typhi strain Ty21a transfected with a plasmid vector encoding for the human vascular endothelial growth factor receptor 2 Glioma Vaximm GmbH;1 Julius-Hatry-Stra�e;Mannheim August 31, 2017
Venetoclax Mantle cell lymphoma AbbVie Inc.;1 North Waukegan Road;North Chicago, Illinois, 60064 August 31, 2017
Fully human monoclonal antibody specific for lymphocyte activation gene 3 and nivolumab Metastatic Melanoma Bristol-Myers Squibb Co.;5 Research Parkway;Wallingford, Connecticut, 06492 August 18, 2017
2-(1,1-difluoroethyl)-5-methyl-N-[4-(pentafluoro-�{6}-sulfanyl)phenyl]-[1,2,4]triazolo[1,5-a]pyrimidin-7-amine Malaria Paul Maher, MD;10644 Weymouth Street, Suite 104;Bethesda, Maryland, 20814 August 18, 2017
O,O’-Bis({4-[3,5-diiodo-4-(1-methylene-1,2,3-triazol-4-ylmethoxy)phenoxy]-3,5-diiodophenyl}acetic acid)polyethylene glycol Pancreatic Cancer NanoPharmaceuticals, LLC;1 Discovery Place;Rensselaer, New York, 12144 August 18, 2017
Humanized IgG4 Monoclonal Antibody against total complement component 1, subcomponent s (C1s) Bullous pemphigoid True North Therapeutics, Inc.;951 Gateway Boulevard;South San Francisco, California, 94080 August 17, 2017
N-[2,6-bis(1-methylethyl)phenyl]-N’-[[1-[4-(dimethylamino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt Congenital adrenal hyperplasia Millendo Therapeutics, Inc.;301 North Main Street, Suite 100;Ann Arbor, Michigan, 48104 August 17, 2017
Obinutuzumab Splenic marginal zone lymphoma Genentech, Inc.;1 DNA Way, MS 241B;South San Francisco, California, 94080 August 17, 2017
(R)-Azasetron besylate Prevention of platinum-induced ototoxicity in pediatric patients Sensorion SA;375, rue du Professeur Joseph Blayac;Montpellier August 16, 2017
Bitopertin �-thalassemia Hoffmann-La Roche Inc.;c/o Genentech, Inc., 1 DNA Way;South San Francisco, California, 94080 August 16, 2017
Upadacitinib Still’s disease AbbVie, Inc.;1 North Waukegan Road;North Chicago, Illinois, 60064 August 16, 2017
Nivolumab Malignant pleural mesothelioma Bristol-Myers Squibb Company;5 Research Parkway;Wallingford, Connecticut, 06492 August 16, 2017
Nivolumab and ipilimumab Malignant pleural mesothelioma Bristol-Myers Squibb Company;5 Research Parkway;Wallingford, Connecticut, 06492 August 16, 2017
Mobilized perioheral blood cells that have undergone ex-vivo negative functionial selection of GvHD-causing T-cells by short term incubation with Fas-ligand Graft Versus Host Disease Cellect Biotherapeutics;23 Ha'Taas Street, P.O. Box 2442;Kfar Saba August 15, 2017
Dasiglucagon Hypoglycemia in patients with congenital hyperinsulinism (CH) Zealand Pharma A/S;36 Smedeland;Glostrup August 10, 2017
Entospletinib Graft Versus Host Disease Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 August 10, 2017
Recombinant human antibody directed against human misfolded superoxide dismutase 1 Amyotrophic lateral sclerosis AL-S Pharma SA;13 Wagistrasse;Schlieren August 10, 2017
Nimodipine intravenous Treatment of subarachnoid hemorrhage Grace Therapeutics, LLC;675 U.S. 1;North Brunswick Township, New Jersey, 08902 August 10, 2017
Cadazolid Clostridium difficile infection in pediatric patients Actelion Pharmaceuticals Ltd.;16 Gewerbestrasse;Allschwil August 8, 2017
Cobitolimod Ulcerative colitis InDex Pharmaceuticals AB;23a Tomtebodav�gen;Hagalund August 8, 2017
Oxfendazole Cysticercosis Oxfendazole Development Group;736 Cathcart Road;Blue Bell, Pennsylvania, 19422 August 7, 2017
KIT and PDGFR inhibitor Glioblastoma Deciphera Pharmaceuticals, LLC;500 Totten Pond Road;Waltham, Massachusetts, 02451 August 7, 2017
1-[4-Bromo-5-[1-ethyl-7-(methylamino)-2-oxo-1,2-dihydro-1,6-naphthyridin-3-yl]-2-fluorophenyl]-3-phenylurea Glioblastoma Deciphera Pharmaceuticals, LLC;500 Totten Pond Road;Waltham, Massachusetts, 02451 August 7, 2017
Blisibimod Patients with immunoglobulin A nephropathy Anthera Pharmaceuticals, Inc.;25801 Industrial Boulevard, Suite B;Hayward, California, 94545 August 7, 2017
Apolipoprotein E mimetic peptide (AEM-28(R)-14) Homozygous Familial Hypercholesterolemia LipimetiX Development, LLC;5 Commonwealth Road, Suite 2A;Natick, Massachusetts, 01760 August 7, 2017