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
Trypsinogen/Chymotrypsinogen Pancreatic Cancer Propanc Pty Ltd.;302/6 Butler Street;Camberwell June 19, 2017
NELATIMOTIDE AND ADEGRAMOTIDE combination Myelodysplastic syndrome Boston Biomedical, Inc.;640 Memorial Drive;Cambridge, Massachusetts, 02139 June 19, 2017
Miltefosine Granulomatous amebic encephalitis (GAE) Profounda, Inc.;5790 Hoffner Avenue, Suite 507;Orlando, Florida, 32822 June 19, 2017
Miltefosine Amebiasis Profounda, Inc.;5790 Hoffner Avenue, Suite 507;Orlando, Florida, 32822 June 19, 2017
Omaveloxolone Friedreich Ataxia Reata Pharmaceuticals, Inc.;2801 Gateway Drive, Suite 150;Irving, Texas, 75063 June 19, 2017
Entospletinib Acute myeloid leukemia Gilead Sciences (formerly Corus Pharma);199 East Blaine St;Seattle, Washington, 98102 June 15, 2017
Pembrolizumab Esophageal carcinoma Merck, Sharp & Dohme, subsidiary of Merck & Co., Inc.;1 Merck Drive;Whitehouse Station, New Jersey, 08889 June 15, 2017
Tezacaftor and Ivacaftor combination therapy Cystic fibrosis Vertex Pharmaceuticals Inc.;50 Northern Avenue;Boston, Massachusetts, 02210 June 15, 2017
Ibutamoren mesylate Growth hormone deficiency Ammonett Pharma, LLC;3606 Salles Ridge Court;Midlothian, Virginia, 23113 June 15, 2017
N-(bromoacetyl)-3,3-dinitroazetidine Neuroendocrine tumors EpicentRx, Inc.;4445 Eastgate Mall, Suite 200;San Diego, California, 92121 June 15, 2017
N-(bromoacetyl)-3,3-dinitroazetidine Small cell lung cancer EpicentRx, Inc.;4445 Eastgate Mall, Suite 200;San Diego, California, 92121 June 15, 2017
Human monoclonal antibody against phosphorylcholine Prevention of arteriovenous fistula or arteriovenous graft failure in patients with end stage renal disease receiving hemodialysis or preparing for hemodialysis Athera Biotechnologies, AB;117 Sankt Eriksgatan, 4th Floor;Norrmalm June 15, 2017
Alirocumab Homozygous Familial Hypercholesterolemia Sanofi-Aventis US, LLC;55 Corporate Drive;Bridgewater, New Jersey, 08807 June 15, 2017
Apatinib mesylate Stomach Cancer LSK BioPartners, Inc.;8 East Broadway, Suite 611;Salt Lake City, Utah, 84111 June 15, 2017
Deferiprone Pantothenate Kinase-associated neurodegeneration ApoPharma, Inc.;200 Barmac Drive, M9L 2Z7; June 15, 2017
Burosumab Oncogenic osteomalacia Ultragenyx Pharmaceutical Inc.;60 Leveroni Court, Suite 200;Novato, California, 94949 June 15, 2017
Cobicistat Human immunodeficiency virus type 1 (HIV-1) infection in pediatric patients Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 June 15, 2017
Tazemetostat Soft tissue sarcoma Epizyme, Inc.;400 Technology Square, 4th Floor;Cambridge, Massachusetts, 02139 June 15, 2017
(3S,4R)-3-ethyl-4-(3H-imidazo[1,2-a]pyrrolo[2,3-e]pyrazin-8-yl)-N-(2,2,2-trifluoroethyl)pyrrolidine-1-carboxamide hydrate pediatric Crohn’s disease AbbVie;1 North Waukegan Road;North Chicago, Illinois, 60044 June 15, 2017
Humanized immunoglobulin G4-variant monoclonal antibody directed against the p19 subunit of interleukin (IL)-23 Ulcerative colitis Eli Lilly & Company;Lilly Corporate Center;Indianapolis, Indiana, 46285 June 15, 2017
Macitentan Chronic thromboembolic pulmonary hypertension (CTEPH) Actelion Clinical Research, Inc.;1820 Chapel Avenue West, Suite 300;Cherry Hill, New Jersey, 08002 June 15, 2017
Cotsiranib Primary sclerosing cholangitis Sirnaomics, Inc.;401 Professional Drive, Suite 280;Gaithersburg, Maryland, 20879 June 15, 2017
Monomethoxypolyethylene glycol recombinant adenosine deaminase Systemic scleroderma Leadiant Biosciences, Inc.;9841 Washingtonian Boulevard, Suite 500;Gaithersburg, Maryland, 20878 June 15, 2017
Humanized IgG1 monoclonal antibody targeting KIR3DL2 Cutaneous T-cell lymphoma Innate Pharma;117 Avenue de Luminy, BP 30191;Marseille June 14, 2017
N-((4-([1,2,4]triazolo[1,5-a]pyridin-6-yl)-5-(6-methylpyridin-2-yl)-1H-imidazol-2-yl)methyl)-2-fluoroaniline Hepatocellular Carcinoma MedPacto, Inc.;145 Gwanggyo-ro, Advanced Institutes of Convergence Technology;Suwon June 14, 2017