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
C20-D3-retinyl acetate Stargardt's disease Alkeus pharmaceuticals, Inc.;2103 Massachusetts Avenue;Cambridge, Massachusetts, 02140 September 16, 2010
Acyclovir Herpetic keratitis GlaxoSmithKline;One Franklin Plaza;Philadelphia, Pennsylvania, 19101 September 16, 2010
Everolimus Waldenstrom macroglobulinemia Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 September 16, 2010
Peptide that inhibits mechanosensitive ion channel (MSC) activity Duchenne muscular dystrophy Rose Pharmaceuticals;8210 Godlen Oak Circle;Williamsville, New York, 14221 September 15, 2010
(1S,3S)-3-amino-4-(difluoromethylene)cyclopentanecarboxylic acid hydrochloride, (1S,3S)-3-amino-4-difluoromethylenyl-1-cyclopentanoic acid hydrochloride West syndrome Catalyst Pharmaceuticals, Inc.;355 Alhambra Circle, Suite 1250;Coral Gables, Florida, 33134 September 15, 2010
Amphotericin B Visceral leishmaniasis iCo Therapeutics Inc.;760-777 Hornby Street;Vancouver, BC V6Z 1S4 September 15, 2010
Adeno-associated viral vector containing modified U11 snRNA Duchenne muscular dystrophy uniQure Biopharma B.V.;Paasheuvelweg 25, 1105 BP;Amsterdam September 15, 2010
Multistem(r) Prophylaxis of graft vs host disease Athersys, Inc.;3201 Carnegie Avenue;Cleveland, Ohio, 44115 September 15, 2010
(1S,3S)-3-amino-4-(difluoromethylene)cyclopentanecarboxylic acid hydrochloride, (1S,3S)-3-amino-4-difluoromethylenyl-1-cyclopentanoic acid hydrochloride Infantile spasms Catalyst Pharmaceutical Partners;355 Alhambra Circle, Suite 1370;Coral Gables, FL, 33134 September 15, 2010
Crizotinib Non-small cell lung cancer Pfizer, Inc.;10646 Science Center Drive;San Diego, California, 92121 September 13, 2010
Darinaparsin Peripheral T-cell lymphoma Solasia Pharma K.K.;3F, Shiodome Building, Toyko 105-0022; September 13, 2010
Autologous dendritic cells pulsed with recombinant human fusion protein (mucin1- glutathione S transferase) coupled to oxidized polymannose Ovarian cancer Prima Biomed Ltd.;1233 High Street, Suite 1;Armadale, Victoria 3143 September 13, 2010
Recombinant human lecithin:cholesterol acyltransferase (rhLCAT) Familial lecithin-cholesterol acyltransferase deficiency (FLD) MedImmune;1 Medimmune Way;Gaithersburg, Maryland, 20878 September 2, 2010
Epitalon Retinitis pigmentosa BioDiem Ltd;Level 10, South Tower, Victoria 3000;Melbourne September 2, 2010
Alpha melanotropin Chronic beryllium disease mondoBIOTECH Laboratories AG;Herrengasse 21, Furstentum Liechtenstein; September 2, 2010
Dichlorphenamide Primary periodic paralyses Strongbridge US Inc.;900 Northbrook Drives, Suite 200;trevose, Pennsylvania, 19053 September 2, 2010
Anakinra Cryopyrin-associated periodic syndromes Swedish Orphan Biovitrum AB (publ);SE-011276;Stockholm August 19, 2010
Maytansinoid DM-1 Conjugated Humanized Monoclonal Antibody N901 Small cell lung cancer ImmunoGen, Inc.;830 Winter Street;Waltham, Massachusetts, 02451 August 17, 2010
14-amino acid peptide derived from wound growth factor Delayed graft function in renal transplant recipients NephRx Corporation;4717 Campus Drive;Kalamazoo, Michigan, 49008 August 16, 2010
Glutathione pegylated liposomal doxorubicin hydrochloride Glioma to-BBB technologies BV;Niels Bohrweg 11, 2333 CA Leiden; August 16, 2010
ActRIIB-IgG1) Duchenne muscular dystrophy Acceleron Pharma, Inc.;128 Sidney St.;Cambridge, Massachusetts, 02139 August 16, 2010
Double-stranded DNA plasmid carrying the gene for the diphtheria toxin A (DT-A) chain Pancreatic Cancer BioCancell Therapeutics USA, Inc.;1 Kendall Square, Building 1400, Suite 14303;Cambridge, Massachusetts, 02139 August 6, 2010
14-amino acid peptide derived from wound growth factor Prevention of delayed graft function in renal transplant recipients NephRx Corporation;4717 Campus Drive;Kalamazoo, Michigan, 49008 August 5, 2010
N-(cyanomethyl)-4-(2-{[4-(morpholin-4-yl(phenyl]amino}pyrimidin-4-yl)benzamide Myelofibrosis Gilead Sciences, Inc.;333 Lakeside Drive;Foster City, California, 94404 August 5, 2010
1-(2-C-cyano-2-deoxy-B-D-arabino-pentafuranosyl)-N4-palmitoylcytosine Acute myeloid leukemia Cyclacel Limited;1 James Linday Place, Dundee DD1 5JJ; June 24, 2010