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
Itraconazole Nevoid basal cell carcinoma syndrome HedgePath Pharmaceuticals, Inc;324 South Hyde Park Avenue, Suite 350;Tampa, Florida, 33606 May 19, 2016
Ropidoxuridine (5-iodo-2-pyrimidinone-2′-deoxyribose) Glioblastoma EMEK, Inc.;196 Channel View;Warwick, Rhode Island, 02889 May 19, 2016
Tetrandrine Chronic myeloid leukemia Escend Pharmaceuticals, Inc.;3475 Edison Way, Suite R;Menlo Park, California, 94025 May 19, 2016
(2,2-diphenyl-tetrahydro-furan-3-yl-methyl)-dimethylamine hydrochloride Rett like syndrome Anavex Life Sciences Corporation;51 West 52nd Street, 7th Floor;New York, New York, 10019 May 18, 2016
Anti-HER2 bispecific antibody Ovarian cancer Zymeworks Inc.;18 West Mercer Street, Suite 370;Seattle, Washington, 98119 May 18, 2016
Human monoclonal antibody directed against tissue factor pathway inhibitor Hemophilia A Pfizer, Inc.;500 Arcola Road;Collegeville, Pennsylvania, 19426 May 18, 2016
Ustekinumab pediatric Crohn’s disease Janssen Research & Development, LLC;P. O. Box 776, 1400 McKean Road;Spring House, Pennsylvania, 19477 May 18, 2016
Combination of disulfiram and copper gluconate Glioblastoma Cantex Pharmaceuticals, Inc.;1792 Bell Tower Lane;Weston, Florida, 33326 May 18, 2016
Liposomal glutathione Dengue Virus infection Your Energy Systems, LLC;555 Bryant Street #305;Palo Alto, California, 94301 May 18, 2016
2-amino-N-({methyl-[(6-trifluoromethoxy-benzothiazol-2-ylcarbamoyl)-methyl]-carbamoyl}-methyl)-acetamide monohydrochloride Spinocerebellar ataxia Biohaven Pharmaceuticals, Inc.;234 Church Street, Suite 304;New Haven, Connecticut, 06510 May 18, 2016
Copper 64-DOTA-TATE Diagnostic for the management of neuroendocrine tumors RadioMedix, Inc.;9701 Richmond Avenue, Suite 222;Houston, Texas, 77042 May 18, 2016
Taurolidine Pancreatic cancer Geistlich Pharma NA, Inc.;202 Carnegie Center Drive;Princeton, New Jersey, 08540 May 16, 2016
(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 Gastric neuroendocrine tumors (gastric NETs) Trio Medicines Ltd.;Pavillion View, Brighton BN1 1EY; May 12, 2016
Naratuximab emtansine Diffuse Large B-cell Lymphoma Debiopharm International S.A.;Forum "apres-demain", Chemin Messidor 5-7, CP 5911; May 12, 2016
Maytansinoid-conjugated humanized monoclonal antibody against CD37 Diffuse Large B-cell Lymphoma Debiopharm International S.A.;Forum "apres-demain", Chemin Messidor 5-7, CP 5911; May 12, 2016
Florilglutamic acid Glioma Piramal Imaging S.A.;13 Route de l'Ecole;Matran May 11, 2016
Autologous T-lymphocytes transduced with anti-BCMA02 CAR lentiviral vector encoding a chimeric antigen receptor (CAR) specific for the human B cell maturation antigen Multiple myeloma Celgene Corporation;86 Morris Avenue;Summit, New Jersey, 07901 May 11, 2016
6-methoxy-3-(3′,4′,5′-trimethoxybenzoyl) indole Bile duct cancer SynCore Biotechnology Co. Ltd.;4F, 69 Dongxing Road, XinYi Dist; May 11, 2016
Autologous ex vivo expanded polyclonal CD4+CD25+CD1271o/-FOXP3+ Regulatory T cells Type 1 diabetes mellitus with residual beta cell function Caladrius Biosciences;110 Allen Road, 2nd Floor;Basking Ridge, New Jersey, 07920 May 10, 2016
Selumetinib Adjuvant treatment of patients with stage III or stage IV differentiated thyroid cancer AstraZeneca Pharmaceuticals LP;1800 Concord Pike, P. O. Box 8355;Wilmington, Delaware, 19803 May 10, 2016
Enfumafungin derivative B-(1,3)-D-glucan synthestis inhibitor Invasive Candida infections, including candidemia SCYNEXIS, Inc.;101 Hudson Street, Suite 3610;Jersey City, New Jersey, 07302 May 10, 2016
Recombinant protein inhibitor of complement factor 5 Guillain-Barré syndrome Akari Therapeutics Plc;24 West 40th Street, 8th Floor;New York, New York, 10018 May 10, 2016
2′-O-(2-methoxyethyl)phosphorothioate antisense oligonucleotide Acromegaly Antisense Therapeutics Limited;6 Wallace Avenue, TOORAK VIC 3142;Trevose May 10, 2016
Clotrimazole otic solution Fungal otitis externa (otomycosis) Laboratorios SALVAT S.A.;C/ Gall, 30-36, Esplugues de Llobregat;Barcelona May 10, 2016
Human recombinant bone morphogenetic protein 4 Glioblastoma HyperStem Therapeutics, Inc.;13223-1 Black Mountain Road;San Diego, California, 92129 May 4, 2016