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
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
Crenolanib Soft tissue sarcoma AROG Pharmaceuticals, LLC;12400 Coit Road, Suite 570;Dallas, Texas, 75251 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
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
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
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
Acadesine Chronic lymphocytic leukemia Avanced In Vitro Cell Technologies, S.L.;(Advancell), 08028 Barcelona; March 3, 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
Revimmune Autoimmune hemolytic anemia Accentia Biopharmaceuticals;324 South Hyde Park Avenue #350;Tampa, Florida, 33606 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
Eflornithine Familial adenomatous polyposis Cancer Prevention Pharmaceuticals;1760 E. River Road #250;Tucson, Arizona, 85718 February 4, 2011
MLN4924-Inhibitor of Nedd8-activating enzyme (NAE) Acute myeloid leukemia Millennium Pharmaceuticals;40 Landsdowne St.;Cambridge, Massachusetts, 02139 February 4, 2011
MLN4924-Inhibitor of Nedd8-activating enzyme (NAE) Myelodysplastic syndrome Millennium Pharmaceuticals, Inc.;40 Landsdowne St.;Cambridge, Massachusetts, 02139 February 4, 2011
D-peptide Acute myeloid leukemia Immune System Key, Ltd;POB 23365;Jerusalem 91233 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
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
Vocimagene amiretrorepvec and flucytosine Glioblastoma Tocagen, Inc.;3030 Bunker Hill St.;San Diego, California, 92109 January 12, 2011