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
Ex-vivo-expanded autologous keratinocytes transduced with retroviral vector containing theCOL7A1gene Dystrophic epidermolysis bullosa Abeona Therapeutics Europe SL February 27, 2017
Asciminib Chronic myeloid leukemia Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 February 27, 2017
Humanised IgG4 monoclonal antibody to the human toll-like receptor type 2 Myelodysplastic syndromes Opsona Therapeutics Limited February 27, 2017
Recombinant human histidyl-tRNA synthetase Limb-girdle muscular dystrophy aTyr Pharma;3545 John Hopkins Court, Suite 250;San Diego, California, 92121 February 23, 2017
6′-((6-aminopyrimidin-4-yl)amino)-8′-methyl-2’H-spiro[cyclohexane-1,3′-imidazo[1,5a]pyridine]-1′,5′-dione hydrochloride Diffuse Large B-cell Lymphoma eFFECTOR Therapeutics;11180 Roselle Street, Suite A;San Diego, California, 92121 February 23, 2017
Ustekinumab Ulcerative colitis Janssen Biotech, Inc.;1400 McKean Road;Spring House, Pennsylvania, 19477 February 22, 2017
Abatacept Giant cell arteritis Bristol-Myers Squibb Research & Development;Marketed Products Development - Regulatory Strategy, P.O. Box 5326;Princeton, New Jersey, 08543 February 22, 2017
Abatacept Idiopathic inflammatory myopathy (IIM) Bristol-Myers Squibb Research & Development;Marketed Products Development - Regulatory Strategy, P.O. Box 5326;Princeton, New Jersey, 08543 February 22, 2017
Sofosbuvir/velpatasvir Pediatric chronic hepatitis C virus (HCV) infection Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 February 22, 2017
5-aza-2′-dexocytidine combined with 1-[(2R,3R,4S,5R)-3,4-dihydroxy-5-(hydroxymethyl)oxolan-2-yl]-4-hydroxy-1,3-diazinan-2-one Thalassemia EpiDestiny Inc.;7536 Royal Portrush Drive;Solon, Ohio, 44139 February 16, 2017
EnGeneIC delivery vehicles loaded with doxorubicin and targeted to tumor with sequences from Vectibix and mouse monoclonal antibody Glioblastoma EnGeneIC Ltd;25 Sirius Road, Building 2;Lane Cove West February 14, 2017
Nelfinavir Multiple myeloma Swiss Group for Clinical Cancer Research (SAKK);Effingerstrasse 33;Bern February 14, 2017
Glucopyranosyl lipid A stable emulsion Follicular lymphoma Immune Design Corp.;1616 Eastlake Ave. E, Suite 310;Seattle, Washington, 98102 February 14, 2017
Dextromethorphan hydrobromide/quinidine sulfate Amyotrophic lateral sclerosis Avanir Pharmaceuticals;30 Enterprise, Suite 400;Aliso Viejo, California, 92656 February 14, 2017
Melatonin Short bowel syndrome Therapicon SrL;21 Via Malachia Marchesi de Taddei;Milano February 14, 2017
6-{[(1R,2S)-2-aminocyclohexyl]amino}-7-fluoro-4-(1-methyl-1H-pyrazol-4-yl)-1,2-dihydro-3H-pyrrolo[3,4-c]pyridine-3-one monocitrate Diffuse Large B-cell Lymphoma Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 February 14, 2017
Atezolizumab Metastatic Melanoma Genentech, Inc.;1 DNA Way, MS 35-5E;South San Francisco, California, 94080 February 13, 2017
Atezolizumab Metastatic Melanoma Genentech, Inc.;1 DNA Way, MS 35-5E;South San Francisco, California, 94080 February 13, 2017
Sirolimus Tuberous sclerosis Aucta Pharmaceuticals, LLC;675 US Highway One;North Brunswick, New Jersey, 08902 February 13, 2017
(3’R,4’S,5’R)-N-[(3R,6S)-6-Carbamoyltetrahydro-2H-pyran-3-yl]-6”-chloro-4′-(2-chloro-3-fluoropyridin-4-yl)-4,4-dimethyl-2”-oxo-1”,2”-dihydrodispiro[cyclohexane-1,2′-pyrrolidine-3′,3”-indole]-5′-carboxamide mono(4-methylbenzenesulfonate) monohydrate Liposarcoma Daiichi Sankyo, Inc.;399 Thornall Street;Edison, New Jersey, 08837 February 13, 2017
Copanlisib Nodal marginal zone lymphoma Bayer HealthCare Pharmaceuticals, Inc.;100 Bayer Boulevard, P. O. Box 915;Hanover, New Jersey, 07981 February 7, 2017
Copanlisib Splenic marginal zone lymphoma Bayer HealthCare Pharmaceuticals, Inc.;100 Bayer Boulevard, P. O. Box 915;Hanover, New Jersey, 07981 February 7, 2017
7-benzyl-4-(2-methylbenzyl)-1,2,6,7,8,9-hexahydroimidazo[1,2-a]pyrido[3,4-e]pyrimidin-5(4H)-one-2HCL Mantle cell lymphoma Oncoceutics, Inc.;3624 Market Street, Suite 5E;Philadelphia, Pennsylvania, 19104 February 7, 2017
Copanlisib Extranodal marginal zone lymphoma Bayer HealthCare Pharmaceuticals, Inc.;100 Bayer Boulevard, P. O. Box 915;Hanover, New Jersey, 07981 February 7, 2017
N-[(2S)-5-{[(2S)-2-(4-fluorophenyl)cyclopropyl]amino}-1-(4-methylpiperazin-1-yl)-1-oxopentan-2-yl]-4-(1H-1,2,3-triazol-1-yl)benzamide bis-tosylate salt Acute myeloid leukemia Imago BioSciences, Inc.;2729 Debbie Court;San Carlos, California, 94070 February 7, 2017