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
Methotrexate Alkaptonuria aimAKU (Associazione Italiana Malati di Alcaptonuria) August 29, 2016
Venetoclax Multiple myeloma AbbVie Inc.;Regualtory Affairs, 1 North Waukegan Road;North Chicago, Illinois, 60064 August 29, 2016
Nintedanib Systemic scleroderma Boehringer Ingelheim International GmbH August 29, 2016
Recombinant protein derived from the saliva of theOrnithodoros moubatatick Paroxysmal nocturnal hemoglobinuria Akari Therapeutics Plc August 29, 2016
Sodium benzoate Lysinuric protein intolerance Lucane Pharma SA August 29, 2016
S-ethylisothiourea diethylphosphate Symptomatic intradialytic hypotension, including symptomatic hypotension in the immediate post-dialytic period TrioxBio, Inc.;P. O. Box 1000;Wilmington, Delaware, 19801 August 29, 2016
Valproic Acid McArdle’s disease Vall d'Hebron Institute of Research August 29, 2016
Synthetic double-stranded siRNA oligonucleotide directed against delta-aminolevulinic acid synthase 1 mRNA covalently linked to a ligand containing three N-acetylgalactosamine residues Variegate porphyria Alnylam UK Limited August 29, 2016
Synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 messenger ribonucleic acid Amyotrophic lateral sclerosis Biogen Idec Limited August 29, 2016
2-((2-ethyl-6-(4-(2-(3-hydroxyazetidin-1-yl)-2-oxoethyl)-piperazin-1-yl)-8-methylimidazo[1,2-alpha]pyridin-3-yl)-(methyl)amino)-4-(4-fluorophenyl)-thiazole-5-carbonitrile Idiopathic pulmonary fibrosis Galapagos NV August 29, 2016
131-I-8H9 monoclonal antibody Neuroblastoma Memorial Sloan Kettering Cancer Center;1275 York Avenue;New York, New York, 10065 August 29, 2016
Temozolomide Glioma Double Bond Pharmaceutical AB August 29, 2016
Ublituximab Neuromyelitis optica TG Therapeutics, Inc.;3 Columbus Circle, 15th Floor;New York, New York, 10019 August 25, 2016
Recombinant, fully-human, IgG4 immunoglobulin with antigen-binding specificity for human vascular adhesion protein-1 (VAP-1) Primary sclerosing cholangitis Acorda Therapeutics, Inc.;420 Saw Mill River Road;Ardsley, New York, 10502 August 25, 2016
Phosphoinositide-3-kinase delta inhibitor Chronic lymphocytic leukemia TG Therapeutics, Inc.;3 Columbus Circle, 15th Floor;New York, New York, 10019 August 23, 2016
(1,3)-ß-D-glucan synthesis inhibitor Invasive Aspergillus infections SCYNEXIS, Inc.;101 Hudson Street, Suite 3610;Jersey City, New Jersey, 07302 August 22, 2016
Ambrisentan/tadalafil fixed combination Pulmonary Arterial Hypertension Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 August 22, 2016
Live E. coli Nissle bacterium modified to assimilate ammonia Urea cycle disorders Synlogic, Inc.;200 Sidney Street, #320;Cambridge, Massachusetts, 02139 August 22, 2016
Stabilized sulforaphane Subarachnoid hemorrhage Evgen Pharma PLC;146 Brownlow Hill;Liverpool August 22, 2016
Recombinant human interleukin-12 Acute Radiation Syndrome Acute radiation syndrome Neumedicines, Inc.;133 Altadena Drive, Suite 133;Pasadena, California, 91107 August 22, 2016
Rituximab and recombinant human hyaluronidase Chronic lymphocytic leukemia Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 August 22, 2016
Rituximab and recombinant human hyaluronidase Follicular lymphoma Genentech, Inc.;1 DNA Way;South San Francisco, California, 94080 August 22, 2016
Etrolizumab Ulcerative colitis Genentech, Inc.;1 Dna Way;South San Francisco, California, 94080 August 22, 2016
Nivolumab Esophageal cancer Bristol-Myers Squibb Co.;5 Research Parkway;Wallingford, Connecticut, 06492 August 22, 2016
Cultured skin substitute (CSS) Burns requiring skin grafting Regenicin Inc.;10 High Court;Little Falls, New Jersey, 07424 August 22, 2016