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
Cannabidiol Pediatric schizophrenia (pediatrics is defined as 0 through 16 years of age) Insys Development Company, Inc.;1333 S. Spectrum Blvd, Suite 100;Chandler, Arizona, 85286 November 17, 2014
(S)-3-((3-(1-((6-((3,4-dimethoxyphenyl)pryazin-2-yl)amino)ethyl)phenyl)carbamoyl)-5-methylpridin-1-ium Pulmonary Arterial Hypertension Pulmokine, Inc.;7 University Place, B127B;Rensselaer, New York, 12144 November 17, 2014
N-(2-amino-2-oxoethyl)-2-(2-((4-fluorophenethyl)amino)-N-isobutylacetamido)-N-(3-(2-oxopyrrolidin-1-yl)propyl)acetamide N-({Carbamoylmethyl-[3-(2-oxo-pyrrolidin-1-yl)propyl}-carbamoyl}-methyl)-2-[2-(2-fluorophenyl)-ethylamino]-N-isobutyl-acetamide Optic neuritis Bionure Farma SL;Dalmases 27, 08017;Barcelona November 17, 2014
N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide Duchenne muscular dystrophy Catabasis Pharmaceuticals, Inc.;One Kendall Square, Suite B14202, Suite B14202;Cambridge, Massachusetts, 02139 November 17, 2014
Autologous CD3+ T cells transduced with retroviral vector containing a chimeric antigen receptor directed against CD19 (Autologous CD3+ T cells containing CD19 chimeric antigen receptor) Acute lymphoblastic leukemia Juno Therapeutics, Inc.;307 Westlake Ave. N., St. 300;Seattle, Washington, 98109 November 13, 2014
(S)-perillyl alcohol temozolomide Glioma NeOnc Technologies, Inc.;8335 Sunset Blvd., #240;Los Angeles, California, 90069 November 12, 2014
Autologous CD4+/CD8+ T cells transduced with lentiviral vector containing a chimeric antigen receptor directed against CD19 Acute lymphoblastic leukemia Juno Therapeutics, Inc.;307 Westlake Avenue, North, Suite 300;Seattle, Washington, 98109 November 6, 2014
D6-tetrabenazine, deutetrabenazine Huntington disease Teva Branded Pharmaceutical Products R&D;, Inc.;3333 N. Torrey Pines Court, Suite 400;La Jolla, California, 92037 November 5, 2014
Adeno-associated viral vector serotype 2 containing the human Rab escort protein 1 gene Choroideremia NightstaRx Ltd.;215 Euston Road, NW1 2BE;London November 5, 2014
(S)-1-(6,7-Dihydro-5H-benzo[6,7]cyclohepta[1,2-c]pyridazin-3-yl)-N3-(7-(pyrrolidin-1-yl)-6,7,8,9-tetrahydro-5H-benzo[7]annulen-2-yl)-1H-1,2,4-triazole-3,5-diamine Acute myeloid leukemia BerGenBio AS;Jonas Lies vei 91, N-5021 Bergen; November 5, 2014
Clenbuterol Pompe disease Duke University Medical Center;Box 103856;Durham, North Carolina, 27710 October 27, 2014
Alpha-1 proteinase inhibitor (human) Graft Versus Host Disease Kamada Ltd.;2 Holtsman Street;Rehovot October 23, 2014
Bi-shRNA furin and GMCSF Autologous Tumor Cell Vaccine Ewing's sarcoma Gradalis, Inc.;2545 Golden Bear Drive, Suite 110;Carrollton, Texas, 75006 October 22, 2014
Autologous adipose derived mesenchymal stromal cells Amyotrophic lateral sclerosis Mayo Clinic;200 First Street SW;Rochester, Minnesota, 55905 October 22, 2014
Istiratumab Pancreatic Cancer Merrimack Pharmaceuticals, Inc.;1 Kendall Square, Suite B7201;Cambridge, Massachusetts, 02139 October 22, 2014
N-(4-{3-(2-aminopyrimidin-4-yl)pyridin-2-yl]oxy}phenyl)-4-(4-methylthiophen-2-yl)phthalazin-1-amine bis-mesylate dihydrate Ovarian cancer Amgen, Inc.;One Amgen Center Drive, Mail Stop 17-2-B;Thousand Oaks, California, 91320 October 21, 2014
Autologous bone marrow CD34+ cells transduced ex vivo with a self activating HIV-1 -based lentiviral vector, EFS-ADA Severe combined immunodeficiency Donald B. Kohn, MD - Professor, UC-UCLA;Microbiology, Immunology & Molecular Genetics, 610 Charles E. Yound Drive, East;Los Angeles, California, 90095 October 21, 2014
Alteplase Plastic bronchitis Kathleen A Stringer, PharmD, FCCP - Professor;University of MI, 428 Church St.;Ann Arbor, Michigan, 48109 October 20, 2014
[5-Amino-1-(4-fluoro-phenyl)-1H-pyrazol-4-yl]-[3-(2,3-dihydroxy-propoxy)-phenyl]-methanone Pancreatic Cancer Synovo GmbH;Paul-Ehrlich-Str. 15, D-72076 Tubingen; October 20, 2014
Dabrafenib Non-small cell lung cancer Novartis Pharmaceuticals Corporation;1 Health Plaza;East Hanover, New Jersey, 07936 October 20, 2014
Cardiotrophin-1 Prevention of ischemia-reperfusion injury in kidney transplant recipients Digna Biotech, S.L.;Avenida Pio XII 22, Pamplona (Navarra); October 16, 2014
Immunoglobulin G1, anti-(human tumour-associated calcium signal transducer 2)(human-Mus musculus monoclonal hRS7 heavy chain), disulfide with human-Mus musculus monoclonal hRS7 k-chain, dimer, hexakis(thioether) with (4S)-4-[[[[4-[[(2S)-2-(4-aminobutyl)-2-[[2-[2-[[26-[4-[[[[4-[(3-mercapto-2,5-dioxo-1-pyrrolidinyl)methyl]cyclohexyl]carbonyl]amino]methyl]-1H-1,2,3-triazol-1-yl]-3,6,9,12,15,18,21,24-octaoxahexacos-1-yl]amino]-2-oxoethoxy]acetyl]amino]-1-oxoethyl]amino]phenyl]methoxy]carbonyl]oxy]-4,11-diethyl-9-hydroxy-1H-pyrano[3′,4′:6,7]indolizino[1,2-b]quinoline-3,14(4H,12H)-dione Pancreatic Cancer Immunomedics GmbH October 15, 2014
N-Methanocarbathymidine Neonatal herpes N & N Pharmaceuticals, Inc.;2400 Research Blvd, Suite 310;Rockville, Maryland, 20850 October 15, 2014
Diacerein Dystrophic epidermolysis bullosa Castle Creek Pharmaceuticals;1033 Skokie Blvd, Suite 600;Northbrook, Illinois, 60062 October 15, 2014
N-(3,4-dihydroxyphenyl)-3,4-dihydroxybenzamide AL Amyloidosis ProtaMed, Inc.;14241 Woodinville Duvall Road, Suite # 370;Woodinville, Washington, 98072 October 15, 2014