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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.
Become a Partner.
| Country | Product | Indication | Sponsor | Date |
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Triamcinolone acetonide | Non-infectious uveitis | S-cubed Ltd | May 21, 2015 |
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Poly-CD-PEG-camptothecin | Ovarian cancer | Cerulean Pharma, Inc.;35 Gatehouse Drive;Waltham, Massachusetts, 02451 | May 21, 2015 |
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Chloroquine | Glioblastoma | DualTpharma B.,V.;Boschstraat 111-D01, 6211 A W Maastricht; | May 20, 2015 |
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Xenon gas | To improve neurological outcome in hospitalized cardiac arrest patients | Neuroprotexeon;Imperial Innovations, London SW7 2PG; | May 18, 2015 |
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Revusiran | Hereditary ATTR amyloidosis | Alnylam Pharmaceuticals, Inc.;300 Third Street;Cambridge, Massachusetts, 02142 | May 18, 2015 |
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(S)-4-(8-amino-3-(1-but-2-ynoylpyrrolidin-2-yl)imidazo[1,5-a]pyrazin-1-yl)-N-(pyridin-2-yl)benzamide | Chronic lymphocytic leukemia | Acerta Pharma BV;Molenstraat 110, 5342 CC Oss; | May 13, 2015 |
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Anti-eTau Humanized IgG4 Monoclonal Antibody | Progressive supranuclear palsy | Biogen, Inc.;225 Binney Street;Cambridge, Massachusetts, 02142 | May 13, 2015 |
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Acalabrutinib | Chronic lymphocytic leukemia | Acerta Pharma BV;Molenstraat 110, 5342 CC Oss; | May 13, 2015 |
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Recombinant human alkaline phosphatase | Hypophosphatasia | AM-Pharma BV;Rumpsterweg 6, 3891 AK Bunnik; | May 13, 2015 |
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2-(7-ethoxy-4-(3-fluorophenyl)-1-oxophthalazin-2(1H)-yl)-N-methyl-N-(2-methylbenzo[d]oxazol-6-yl)acetamide | Cystic fibrosis | Flatley Discovery Lab;529 Main Street, Suite 115;Charlestown, Massachusetts, 02129 | May 13, 2015 |
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Bruton’s Tyrosine Kinase (Btk) Inhibitor | Chronic lymphocytic leukemia | Acerta Pharma BV;Molenstraat 110, 5342 CC Oss; | May 13, 2015 |
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E-selectin antagonist | Acute myeloid leukemia | GlycoMimetics, Inc.;401 Professional Drive, Suite 250;Gaithersburg, Maryland, 20879 | May 13, 2015 |
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Adalimumab | Hidradenitis suppurativa | AbbVie, Inc.;1 North Waukegan Road;North Chicago, Illinois, 60064 | May 13, 2015 |
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Hydrocortisone oral granules | Pediatric adrenal insufficiency (0 through 16 years of age) | Diurnal Limited;Cardiff Medicentre, Cardiff CF14 4UJ; | May 13, 2015 |
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Edaravone | Amyotrophic lateral sclerosis | Mitsubishi Tanabe Pharma Corporation;17-10, Nihonbashi-Koamicho, Chuo-ku, Tokyo 103-8405; | May 12, 2015 |
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N-t-butylhydroxylamine | Infantile neuronal ceroid lipofuscinosis | Andrew Lim;California Institute of Technology, 1200 East California Blvd.;Pasadena, California, 91125 | May 12, 2015 |
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Autologous tumor cells fused with polyethylene glycol to human allogeneic FO-1, DNA plasmid transfected tumor cell line | Glioblastoma | Sebastiano Gattoni-Celli, MD;Med. University of SC- Dept of Radiation Oncology, 114 Doughty St.;Charleston, South Carolina, 29403 | May 6, 2015 |
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Humanized monoclonal antibody that targets the a-subunit of the human IL-3 receptor also known as the CD123 antigen; (JNJ-56022473) | Acute myeloid leukemia | Janssen Research & Development, LLC;920 Route 202, Box 300;Raritan, New Jersey, 08869 | May 5, 2015 |
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Tacrolimus | Pulmonary Arterial Hypertension | Stanford University School of Medicine;300 Pasteur Drive, Room H3143;Stanford, California, 94305 | May 4, 2015 |
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Pantothenate phosphate | Pantothenate Kinase-associated neurodegeneration | Retrophin, Inc.;777 Third Avenue, 22nd Floor;New York, New York, 10017 | May 4, 2015 |
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Pelareorep | Stomach Cancer | Oncolytics Biotech, Inc.;210, 1167 Kensington Crescent NW, Calgary AB T2N1X7; | May 4, 2015 |
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Tadalafil | Duchenne muscular dystrophy | Eli Lilly and Company;Lilly Corporate Center;Indianapolis, Indiana, 46285 | May 4, 2015 |
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4-(pyrimidin-2-ylmethyl)-7-[4-(trifluoromethoxy)phenyl]-3,4-dihydro-1,4-benzoxazepin-5(2H)-one | Congenital long QT syndrome | Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 | May 4, 2015 |
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LOAd703, a modified adenovirus 5/35 containing a CMV promoter-driven transgene cassette with the human transgenes for a membrane-bound CD40 ligand (TMZ-CD40L) and full length 4-1BBL | Pancreatic Cancer | Lokon Pharma AB;Dag Hammarskjoldsvag 40c, 751 83, Uppsala; | May 4, 2015 |
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Antroquinonol | Acute myeloid leukemia | Golden Biotechnology Corporation;10F, No. 9, Ln. 3, Sec. 1, Zhong-Zheng E. Rd, New Taipei City 251; | April 30, 2015 |