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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 |
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6-{[(1S)-1-phenylethyl]amino}-3-(propan-2-yl)-1,2,3,4 tetrahydropyrimidine-2,4-dione | Symptomatic obstructive hypertrophic cardiomyopathy | MyoKardia, Inc.;333 Allerton Avenue;South San Francisco, California, 94080 | April 27, 2016 |
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Autologous T-cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor | Follicular lymphoma | Kite Pharma, Inc.;2225 Colorado Avenue;Santa Monica, California, 90404 | April 25, 2016 |
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(3S)-3-[({1-(2-Chlorobenzyl)-4-hydroxy-5-methyl-2-oxo-1,2-dihydropyridin-3yl}amino)carbonyl]amino-3-(4-methylphenyl)propanoic acid monosodium salt | For mobilization of hematopoietic stem cells | Aviara Pharmaceuticals, Inc/;3331 Green Tree Park;Houston, Texas, 77027 | April 25, 2016 |
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(E, Z)-4,5,9-trithiadodeca-1, 6, 11-triene 9-oxide (Ajoene) | Cystic fibrosis | Neem Biotech Ltd.;Units G & H, Roseheyworth Business Park;Blaenau Gwent | April 21, 2016 |
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Aphidicolin | Ebola | Biospherics.net LLC;Department of Pharmaceutical Sciences, University of Kentucky;Nicholasville, Kentucky, 40356 | April 20, 2016 |
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Autologous T-cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor | Primary mediastinal B-cell lymphoma | Kite Pharma, Inc.;2225 Colorado Avenue;Santa Monica, California, 90404 | April 20, 2016 |
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Autologous T-cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor | Acute lymphoblastic leukemia. (ALL). | Kite Pharma, Inc.;2225 Colorado Avenue;Santa Monica, California, 90404 | April 20, 2016 |
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Autologous T-cells transduced with retroviral vector encoding an anti-CD19 CD28/CD3-zeta chimeric antigen receptor | Chronic lymphocytic leukemia | Kite Pharma, Inc.;2225 Colorado Avenue;Santa Monica, California, 90404 | April 20, 2016 |
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Allogeneic ex-vivo generated CD56+CD3-natural killer cells from CD34+ umbilical cord blood progenitor cells | Acute myeloid leukemia | IPD-Therapeutics BV;(Trading as Glycostem-Therapeutics), Molenstraat 110 5342 CC Oss; | April 20, 2016 |
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Adenovirus encoding the recombinant CD40 ligand | Stage IIb through IV melanoma | Memgen LLC;12 Greenway Plaza, Suite 1100;Houston, Texas, 77046 | April 19, 2016 |
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1,2:5,6-dianhydrogalactitol | Ovarian cancer | DelMar Pharmaceuticals, Inc. (Clinical Operations);3475 Edison Way, Suite R;Menlo Park, California, 94025 | April 19, 2016 |
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Cannabidiol | Tuberous sclerosis | GW Pharma Ltd.;15 T.W. Alexander Drive, P. O. Box 13547;Research Triangle Park, North Carolina, 27709 | April 19, 2016 |
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HDF-LV-RTS-MMP1 (human dermal fibroblasts genetically modified with LV-RTS-MMP1) and Veledimex | Localized scleroderma | Fibrocell Technologies, Inc.;405 Eagleview Blvd;Exton, Pennsylvania, 19341 | April 19, 2016 |
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(5S,8S, 10aR)-N-benzhydryl-5-((S)-2-(methylamino)propanamido)-3-(3-methylbutanoyl)-6-oxodecahydropyrrolo[1,2-a][1,5] diazocine-8-carboxyamide | Ovarian cancer | DebioPharm International SA;Forum "apres-demain", Chemin Messidor, 5-7;Lausanne | April 14, 2016 |
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Ganaxolone | Status epilepticus | Marinus Pharmaceuticals, Inc.;Three Radnor Corporate Center, 100 Matsonford Road;Radnor, Pennsylvania, 19087 | April 14, 2016 |
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7-ethyl-10-hydroxycamptothecin (EHC) | Small cell lung cancer | Nippon Kayaku Co., Ltd.;1-1, Marunouchi 2-chome;Chiyoda | April 13, 2016 |
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1-(2-Nitro-imidazolyl)-3-[18F]fluoro-2-propanol; 1H-1-(3-[18F]fluoro-2-hydroxypropyl0-2-nitroimidazole (18F-Misonidazole) | Diagnostic for management of glioma | Advanced Imaging Projects, LLC;7947 Brookside Ct.;Lake Worth, Florida, 33467 | April 6, 2016 |
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1-(2,8-Dimethyl-1-thia-3,8-diazaspiro[4.5]dec-3-yl)-3-(1H-indol-3-yl)propan-1-one | Frontotemporal dementia | Anavex Life Sciences Corp.;51 West 52nd Street, 7th Floor;New York, New York, 10019 | April 6, 2016 |
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Pritumumab | Pancreatic cancer | Nascent Biotech, Inc.;8400 Miramar Road, Suite 247;San Diego, California, 92126 | April 6, 2016 |
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Metronidazole | Clostridium difficile infection in pediatric patients (0 through 16 years of age) | Appili Therapeutics, Inc.;1344 Summer Street, Suite 415;Halifax, Nova Scotia | April 5, 2016 |
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Cerebrolysin | Frontotemporal dementia, including all subvariants | EVER Neuro Pharma GmbH;3 Oberburgau, 4866 Unterach; | April 5, 2016 |
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Poly-N,N’-hexamethyleneguanidine-poly-N1,N4-hexamethyleneaminoguanidine | Cystic fibrosis | TGV-Inhalonix, Inc.;303 5th Avenue, Suite 2012;New York, New York, 10016 | April 5, 2016 |
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No-title | Frontotemporal dementia, including all subvariants | EVER Neuro Pharma GmbH;3 Oberburgau, 4866 Unterach; | April 5, 2016 |
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Fexinidazole | African trypanosomiasis | Sanofi US Services, Inc.;55 Corporate Drive, Mail Code 55D-225A;Bridgewater, New Jersey, 08807 | April 4, 2016 |
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Setmelanotide | Pro-opiomelanocortin Deficiency | Rhythm Pharmaceuticals, Inc.;855 Boylston Street, 11th Floor;Boston, Massachusetts, 02116 | April 4, 2016 |