Treatments

The following database contains products that have been granted orphan status, or have been market approval in the EU or the USA.

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Country Product Indication Sponsor Date
4-[4-[[[[3-(1,1-dimethylethyl)-1-(6-quinolinyl)-1H-pyrazol-5-yl]amino]carbonyl]-amino]-3-fluorophenoxyl]-N-methyl-2-pyridinecarboxamide, p-toluenesulfonate salt Chronic myeloid leukemia Deciphera Pharmaceuticals, LLC;643 Massachusetts, Suite 200;Lawrence, Kansas, 66044 September 3, 2009
Taliglucerase alfa Gaucher disease Pfizer, Inc.;235 East 42nd Street;New York, New York, 10017 September 3, 2009
Rigosertib Myelodysplastic syndromes Onconova Therapeutics, Inc.;375 Pheasant Run;Newtown, Pennsylvania, 18940 September 3, 2009
8-[4-(1-aminocyclobutyl)phenyl]-9-phenyl-1,2,4-triazolo[3,4-f][1,6]naph-thyrin-3(2H)-one mono-hydrochloride Ovarian cancer Merck Sharp & Dohme Corp.;P. O. Box 1000, UG-2C50;North Wales, Pennsylvania, 19454 September 3, 2009
Autologus CD34+ cells transfected with retroviral vector containing adenosine deaminase gene Severe combined immunodeficiency Glaxo Group Limited;I-3 Iron Bridge Road, Stockley Park West; August 26, 2009
5-[1-(2,6-Dichlorobenzyl)-piperidin-4-yl]methoxyquinazoline-2,4-diamine Spinal muscular atrophy CureSMA;925 Busse Avenue;Elk Grove Village, Illinois, 60007 August 25, 2009
Doxorubicin Hepatocellular Carcinoma Delcath Systems, Inc.;1633 Broadway, Suite 22C;New York, New York, 10019 August 25, 2009
Exon 51 specific phosphorothioate oligonucleotide Duchenne muscular dystrophy BioMarin Pharmaceutical, Inc.;105 Digital Drive;Novato, California, 94949 August 25, 2009
Expanded allogeneic human dermal fibroblasts in hypothermosol(r)-FRS Dystrophic epidermolysis bullosa Intercytex Ltd.;St. John's Innovation Centre, Cambridge CB4 0WS; August 20, 2009
Recombinant antibody construct against human CD30 and CD16A Hodgkin’s disease Affimed GmbH;Technologie Park, Heidelberg,; August 20, 2009
DNA plasmid expressing diphtheria toxin triggered by the presence of oncofetal gen H19 Ovarian cancer BioCancell Therapeutics US, Inc.;1 Kendall Square, Building 1400, Suite 14303;Cambridge, Massachusetts, 02139 August 20, 2009
Anti-EphA1 monoclonal antibody conjugated to mcMMAF Ovarian cancer MedImmune, LLC;One MedImmune Way;Gaithersburg, Maryland, 20878 July 27, 2009
(S)-3′-(OH)-desazadesferrithiocin-polyether, magnesium salt Chronic iron overload requiring chelation therapy Shire Pharmaceutical Development Limited July 24, 2009
Beclomethasone 17,21-dipropionate Gastrointestinal symptoms with chronic graft versed host disease in patients undergoing allogenic hematopoietic cell transplantation Soligenix, Inc.;29 Emmons Drive;Princeton, New Jersey, 08540 July 24, 2009
Ecopipam hydrochloride Symptomatic treatment of self injurious behaviors in patients with Lesch-Nyhan disease Psyadon Pharmaceuticals, Inc.;20451 Seneca Meadows Parkway;Germantown, Maryland, 20876 July 21, 2009
Gastrin 17C Diphtheria Toxoid Immunogen Pancreatic Cancer Astrimmune Ltd.;Mansfield i-cente, Hamilton Way;Mansfield July 7, 2009
Midostaurin Acute myeloid leukemia Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 July 7, 2009
Anti-nogo-A monoclonal antibody Amyotrophic lateral sclerosis GlaxoSmithKline;Glaxo Wellcome House, Greenford, Middlesex, UK UB6 0NN; July 7, 2009
Midostaurin Acute myeloid leukemia Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 July 7, 2009
Catumaxomab Stomach Cancer Neoviibiotech North America, Inc.;920 Winter Street;Waltham, Massachusetts, 02451 July 1, 2009
Everolimus Lymphangioleiomyomatosis Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 June 8, 2009
Astuprotimut-R Non-small cell lung cancer GlaxoSmithKline Biologicals, S.A.;Rue de I'Institut 89, B-1330 Rixensart; May 29, 2009
Sterile Bicarbonate Infusate, Hemofiltration, Hemodiafiltration, and Hemopdialysis Solution For use as a replacement solution in adults and children during Continous Renal Replacement Therapy Nikkiso America, Inc.;5910 Pacific Center Blvd, Suite 110;San Diego, California, 92121 May 29, 2009
Astuprotimut-R MAGE-A3 positive stages IIb to IV malignant melanoma GlaxoSmithKline Biologicals, S.A.;Rue de l'Institut 89; May 29, 2009
Capsaicin Management of neuropathic pain in patients with postherpetic neuralgia Acorda Therapeutics, Inc.;420 Saw Mill River Road;Ardsley, New York, 10502 May 22, 2009