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
FTV1+GDP-fucose To improve homing to bone (treatment of myeloablation) in patients receiving hematopoietic stem cell transplantation America Stem Cell, Inc.;11014 Bar X Trail;Helotes, Texas, 78023 March 18, 2011
Rigosertib Pancreatic cancer Onconova Therapeutics, Inc;375 Pheasant Run;Newtown, Pennsylvania, 18940 March 18, 2011
Acetyl-l-carnitine (ALC) Rett syndrome Leadiant Biosciences, Inc.;9841 Washingtonian Blvd., Suite 500;Gaithersburg, Maryland, 20878 March 18, 2011
Heat Shock Protein 70 Amyotrophic lateral sclerosis ALS Biopharma, LLC;3805 Old Easton Rd;Doylestown, Pennsylvania, 18902 March 18, 2011
(3,5-Bis trifluoromethyl)-N-[4-methyl-3-(4-pyridin-3yl-pyrimidin-2-yl amino) phenyl] benzamide Pancreatic Cancer NATCO Pharma Limited;Natco House;Hyderabad 500033 March 18, 2011
(3,5-Bis trifluoromethyl)-N-[4-methyl-3-(4-pyridin-3yl-pyrimidin-2-yl amino) phenyl] benzamide Chronic myeloid leukemia NATCO Pharma Limited;Natco House;Hyderbad 500033 March 18, 2011
(3,5-Bis trifluoromethyl)-N-[4-methyl-3-(4-pyridin-3yl-pyrimidin-2-yl amino) phenyl] benzamideNRC-AN-019 Glioma NATCO Pharma Limited;Natco House;Hyderbad 500033 March 18, 2011
Brivanib alaninate Hepatocellular Carcinoma Bristol-Myers Squibb;P. O. Box 4000;Princeton, New Jersey, 08543 March 18, 2011
Adeno associated viral vector containing human ARSB gene Mucopolysaccharidosis type VI Fondazione Telethon;Via Varese 16 - 00185 Roma; March 17, 2011
Sar9, Met(O2)11-Substance P Idiopathic pulmonary fibrosis ImmuneRegen BioSciences, Inc.;IR Biosciences Holdings, Inc;Scottsdale, Arizona, 85258 March 16, 2011
Coenzyme Q10 and d-alpha-tocopherol Friedreich Ataxia NBI Pharmaceuticals, Inc.;c/o Neil & Co CPAs, 1184 N. 15th Ave., Suite 1;Bozeman, Montana, 59715 March 14, 2011
Talimogene laherparepvec Metastatic Melanoma BioVex, Inc. (subsidiary of Amgen);Amgen, Mail Stop 17-2-B;Thousand Oaks, California, 91320 March 14, 2011
Eicosapentaenoic acid Familial adenomatous polyposis S.L.A. Pharma Ltd. (UK);Elite House, Leavesden, Watford WD25 7SA; March 8, 2011
Acadesine Chronic lymphocytic leukemia Avanced In Vitro Cell Technologies, S.L.;(Advancell), 08028 Barcelona; March 3, 2011
Adenosine triphosphate type 1 competitive inhibitor of JAK2 V617F tyrosine kinase Myeloproliferative disorders (polycythemia vera, essential throbocythemia, and myelofibrosis) Eli Lilly and Company;Lilly Corporate Center;Indianapolis, Indiana, 46285 March 3, 2011
Revimmune Autoimmune hemolytic anemia Accentia Biopharmaceuticals;324 South Hyde Park Avenue #350;Tampa, Florida, 33606 February 18, 2011
Acetyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-arginyl-D-cysteinyl-D-glutaminyl-D-cysteinyl-D-arginyl-D-arginyl-D-lysyl-Dasparaginylamide disulfide Pancreatic cancer Genus Oncology, LLC;2458 N. Racine Avenue;Chicago, Illinois, 60614 February 18, 2011
S-59 treated FFP (plasma treated with amotosalen hydrochloride & ultraviolet A light Thrombotic thrombocytopenic purpura Cerus Corporation;2550 Stanwell Drive;Concord, California, 94520 February 14, 2011
MLN4924-Inhibitor of Nedd8-activating enzyme (NAE) Acute myeloid leukemia Millennium Pharmaceuticals;40 Landsdowne St.;Cambridge, Massachusetts, 02139 February 4, 2011
N-{[(5S)-3-(3-fluoro-4-thiomorpholin-4-ylphenyl)-2-oxo-1,3-oxazolidin-5-yl]methyl}acetamide Tuberculosis Sequella, Inc.;9610 Medical Center Drive, Suite 200;Rockville, Maryland, 20820 February 4, 2011
D-peptide Acute myeloid leukemia Immune System Key, Ltd;POB 23365;Jerusalem 91233 February 4, 2011
MLN4924-Inhibitor of Nedd8-activating enzyme (NAE) Myelodysplastic syndrome Millennium Pharmaceuticals, Inc.;40 Landsdowne St.;Cambridge, Massachusetts, 02139 February 4, 2011
Eflornithine Familial adenomatous polyposis Cancer Prevention Pharmaceuticals;1760 E. River Road #250;Tucson, Arizona, 85718 February 4, 2011
[AC3-yCD2(V)] and Flucytosine (5-FC XR) Glioblastoma multiforme Tocagen, Inc.;3030 Bunker Hill St.;San Diego, California, 92109 January 12, 2011
N-[(2S)-2,3-dihydroxypropyl]-3-[(2-fluoro-4-iodophenyl)amino] isonicotinamide hydrochloride Acute myeloid leukemia EMD Serono, Inc.;1 Technology Place;Rockland, Massachusetts, 02370 January 12, 2011