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
Copanlisib Extranodal marginal zone lymphoma Bayer HealthCare Pharmaceuticals, Inc.;100 Bayer Boulevard, P. O. Box 915;Hanover, New Jersey, 07981 February 7, 2017
Brentuximab vedotin Cutaneous T-cell lymphoma Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 February 6, 2017
Anti-HER2 bispecific antibody Stomach Cancer Zymeworks Inc.;18 West Mercer Street, Suite 370;Seattle, Washington, 98119 February 6, 2017
Allogeneic human islets of Langerhans Brittle Type 1 Diabetes University of Illinois;Hospital & Health Sciences System, Transplant, 840 South Wood Street;Chicago, Illinois, 60612 February 1, 2017
Laquinimod sodium Huntington disease Teva Branded Pharmaceutical Products R&D;;41 Moores Road, P. O. Box 4011;Frazer, Pennsylvania, 19355 January 31, 2017
Nitric oxide Idiopathic pulmonary fibrosis GeNo, LLC;2941 Oxbow Circle;Cocoa, Florida, 32926 January 31, 2017
6-[(3S,4S-4-methyl-1-(pyrimidin-2-ylmethyl)pyrrolidin-3-yl]-3-tetrahydropyran-4-yl-7H-imadazo[1,5-a]pyrazin-8-one Sickle cell anemia Imara Inc.;700 Technology Square, 3rd Floor;Cambridge, Massachusetts, 02139 January 31, 2017
Adeno-associated virus (AAV) serotype rh.10 expressing beta-galactosidase Gangliosidosis generalized GM1- type 1 Lysogene, Inc.;245 First Street, 18th Floor;Cambridge, Massachusetts, 02142 January 30, 2017
(5R)-5-(4-{[(2-fluorophenyl)methyl]oxy}phenyl)-L-prolinamide, hydrochloride Erythromelalgia Biogen, Inc.;300 Binney Street;Cambridge, Massachusetts, 02142 January 30, 2017
Para-toluenesulfonamide Adenoid cystic carcinoma PTS International Inc.;2211 Newoak Park;San Antonio, Texas, 78230 January 30, 2017
Hydroxypioglitazone X-linked adrenoleukodystrophy Minoryx Therapeutics S.L.;TecnoCampus Mataro-Maresme. TCM3 602, Av. Ernest Lluch, 32.;Matar� January 30, 2017
Adeno-associated viral vector serotype 2.7m8 containing the chrimsonR-tdTomato gene Retinitis pigmentosa GenSight Biologics;74 Rue du Faubourg Saint-Antoine;Paris January 25, 2017
Melatonin Necrotizing enterocolitis Therapicon SrL;21 Via Malachia Marchesi de Taddei;Milano January 25, 2017
Dendritic cell/tumor cell fusion vaccine + recombinant human interleukin 12 Glioma CyTIX Inc.;7F Terada Bldg, 2-3-3 Shibakoen;Minato-ku January 25, 2017
Nitric oxide Pulmonary Arterial Hypertension GeNo, LLC;2941 Oxbow Circle;Cocoa, Florida, 32926 January 25, 2017
(R)-2-(5-cyano-2-(6-(methoxycarbonyl)-7-methyl-3-oxo-8-(3-(trifluoromethyl)phenyl)-2,3,5,8-tetrahydro-[1,2,4]triazolo[4,3-a]pyrimidin-5-yl)phenyl)-N,N,N-trimethylethanaminium methanesulfonate dehydrate Cystic fibrosis Chiesi Farmaceutici S.p.A.;26A Via Palermo;Parma January 25, 2017
Nicotinamide riboside Mitochondrial encephalomyopathy lactic acidosis and stroke-like episodes Rejuvenation Therapeutics;774 Mays Boulevard, 10-509 PMB;Incline Village, Nevada, 89451 January 25, 2017
3-[4-(lH-imidazol-l-ylmethyl)phenyl]-5-(2-methylpropyl) thiophene-2-[(N-butyloxylcarbamate)-sulphonamide] sodium salt Idiopathic pulmonary fibrosis Vicore Pharma;c/o AstraZeneca, Pepparedsleden 1;M�lndal January 25, 2017
Human monoclonal antibody directed against human Activin A Fibrodysplasia ossificans progressiva Regeneron Pharmaceuticals, Inc.;777 Old Saw Mill River Road;Tarrytown, New York, 10591 January 19, 2017
D-tagatose Prader-Willi syndrome Biospherics.net LLC;Department of Pharmaceutical Sciences, University of Kentucky;Nicholasville, Kentucky, 40356 January 19, 2017
Ublituximab in combination with phosphoinositide-3-kinase (PI3K) delta inhibitor Diffuse Large B-cell Lymphoma TG Therapeutics, Inc.;2 Gansevoort Street, 9th Floor;New York, New York, 10014 January 19, 2017
Humanized IgG4 monoclonal antibody binding to human C1q Guillain-Barré syndrome Annexon Biosciences;280 Utah Avenue, Suite 110;South San Francisco, California, 94080 January 19, 2017
Hyaluronic acid Emphysema due to alpha1-antitrypsin deficiency Gerard M. Turino, MD;Division of Pulmonary, Critical Care & Sleep Medicine, Mt. Sinai Roosevelt Hospital Center;New York, New York, 10019 January 18, 2017
D9-ivacaftor Cystic fibrosis Vertex Pharmaceuticals, Inc.;50 Northern Avenue;Boston, Massachusetts, 02210 January 18, 2017
2-Amino-4-[11C]methylsulfanyl-butanoic acid, l-[S methyl[11C]methionine Medulloblastoma Advanced Imaging Projects, LLC;7947 Brookside Ct.;Lake Worth, Florida, 33467 January 17, 2017