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
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
Anti-HER2 bispecific antibody Stomach Cancer Zymeworks Inc.;18 West Mercer Street, Suite 370;Seattle, Washington, 98119 February 6, 2017
Brentuximab vedotin Cutaneous T-cell lymphoma Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 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
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
Laquinimod sodium Huntington disease Teva Branded Pharmaceutical Products R&D;;41 Moores Road, P. O. Box 4011;Frazer, Pennsylvania, 19355 January 31, 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 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
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
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
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
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
D9-ivacaftor Cystic fibrosis Vertex Pharmaceuticals, Inc.;50 Northern Avenue;Boston, Massachusetts, 02210 January 18, 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
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