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
Basimglurant Fragile X syndrome Hoffmann-La Roche, Inc.;340 Kingsland Street;Nutley, New Jersey, 07110 March 9, 2012
(N-[2,6-bis(1-methylethyl)-pheyl-N’-[[1-4-dimethyl-amino)phenyl]cyclopentyl]methyl]urea, hydrochloride salt Adrenocortical carcinoma Millendo Therapeutics, Inc.;301 North Main Street, Suite 100;Ann Arbor, Michigan, 48104 March 9, 2012
Telotristat etiprate Carcinoid syndrome Lexicon Pharmaceuticals, Inc.;8800 Technology Forest Drive;The Woodlands, Texas, 77381 March 9, 2012
Anti-Lewis Y humanized monoclonal antibody Ovarian cancer Recepta Biopharma S.A.;Rua Tabapua, 1123 cj.36, 04533-014; March 9, 2012
2-Chloro-4-[1-(4-fluoro-phenyl)-2,5-dimethyl-1H-imidazol-4-ylethynyl]-pyridinium sulfate Fragile X syndrome Hoffmann-La Roche, Inc.;340 Kingsland Street;Nutley, NJ, 07110-1199 March 9, 2012
2,2′-{2-[1R)-1-({[(2,5-dichlorobenzoyl)amino]acetyl}amino)-3-methylbutyl]-5-oxo-1,3,2-dioxaborolane-4,4-diyl}diacetic acid (ixazomib citrate) Systemic light chain (AL) amyloidosis Millennium Pharmaceuticals, Inc.;40 Landsdowne Street;Cambridge, Massachusetts, 02139 March 9, 2012
(1-methyl-2-nitro-1H-imidazole-5-yl)methyl N,N’-bis(2-bromoethyl) diamidophosphate Soft tissue sarcoma Threshold Pharmaceuticals, Inc.;170 Harbor Way, Suite 300;South San Francisco, California, 94080 March 9, 2012
2S,4R ketoconazole Cushing’s syndrome Cortendo AB (HQ address);Box 47, SE-433 21 Partille; March 9, 2012
Sialic acid GNE myopathy Ultragenyx Germany GmbH March 5, 2012
Recombinant humanized monoclonal antibody directed against an epidermal growth factor receptor Glioblastoma AbbVie, Inc.;1 N. Waukegan Road;North Chicago, Illinois, 60064 February 17, 2012
Silibinin-C-2′,3-dihydrogensuccinate, disodium salt Prevention of recurrent hepatitis C in liver transplant patients Mylan Specialty LP;781 Chestnut Ridge Road, P. O. Box 4310;Morgantown, West Virginia, 26505 February 17, 2012
2-Chloro-N6–(3-iodobenzyl)adenosine-5′-N-methyluronamide Hepatocellular Carcinoma Can-Fite BioPharma Ltd.;10 Bareket Street, P. O. Box 7537;Petah-Tikva February 17, 2012
Bi-shRNAfurin and granulocyte macrophage colony stimulating factor augmented autologus tumor cell vaccine Metastatic Melanoma Gradalis, Inc.;2545 Golden Bear Drive, Suite 110;Carrollton, Texas, 75006 February 17, 2012
Vestronidase alfa-vjbk Mucopolysaccharidosis VII (MPS VII, Sly Syndrome) Ultragenyx Pharmaceutical, Inc.;60 Leveroni Court;Novato, California, 94949 February 16, 2012
Sodium Thiosulfate Calciphylaxis Luitpold Pharmaceuticals, Inc.;800 Adams Avenue, Suite 100;Norristown, Pennsylvania, 19403 February 16, 2012
Digoxin immune fab (ovine) Severe preeclampsia and eclampsia Velo Bio, LLC;90001 Aerial Center Parkway, Suite 110;Morrisville, North Carolina, 27560 February 3, 2012
Adeno-Associated Viral Vector Expressing Low-Density Lipoprotein Receptor Homozygous Familial Hypercholesterolemia ReGenX Biosciences LLC;750 17th Street, NW, Suite 1100;Washington, District of Columbia, 20006 January 31, 2012
1,2:5,6-Dianhdrogalactitol Malignant gliomas DelMar Pharmaceuticals, Inc.;3475 Edison Way, Suite R;Menlo Park, California, 94025 January 31, 2012
1,2:5,6-Dianhdrogalactitol, NSC-132313 Malignant gliomas DelMar Pharmaceuticals, Inc.;3475 Edison Way, Suite R;Menlo Park, California, 94025 January 31, 2012
N-Hydroxy-4-(3-methyl-2-(S)phenyl-butyrylamino)benzamide Schwannoma Arno Therapeutics, Inc.;200 Route 31 North, Suite 104;Flemington, New Jersey, 08822 January 13, 2012
N-Hydroxy-4-(3-methyl-2-(S)phenyl-butyrylamino)benzamide Meningioma Arno Therapeutics, Inc.;200 Route 31 North, Suite 104;Flemington, New Jersey, 08822 January 13, 2012
1-[(2-Chloro-4-methoxyphenoxy)methyl]-4-[(2,6-dichlorophenoxy) methyl]benzene Poliovirus infection ViroDefense, Inc.;155 Gibbs Street, Suite 529;Rockville, Maryland, 20850 January 9, 2012
Sodium nitrite Chlorine gas poisoning Hope Pharmaceuticals;16416 N. 92nd Street;Scottsdale, Arizona, 85260 January 9, 2012
H-(D)p-Benzoylphenylalanyl-(D)seryl-(D)tryptophanyl-(D)seryl-(D)pentafluorophenylalanyl-(D)cyclohexylalanyl-(D)arginyl-(D)arginyl-(D)arginyl-(D)glutaminyl-(D)arginyl-(D)arginine acetate salt Malignant pleural mesothelioma CanBas Company, Ltd.;2-2-1 Otemachi;Numazu City, Shizuoka December 22, 2011
Beraprost sodium 314d Pulmonary Arterial Hypertension Lung Rx, Inc.;1040 Spring Street;Silver Spring, Maryland, 20910 December 22, 2011