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
Hantaan virus and Puumala virus DNA vaccines Prevention of hemorrhagic fever with renal syndrome caused by Hantaan virus and Puumala virus Surgeon General of the U. S. Army;1430 Veterans Drive, ATTN: USAMR and MC;Fort Detrick, Maryland, 21702 November 13, 2012
Crizotinib Neuroblastoma Pfizer, Inc.;10646 Science Center Drive;San Diego, California, 92121 October 31, 2012
5′-GCCATGGTTTTTTCTCAGG-3′ Prophylaxis for patients following documented or suspected exposure to ebolavirus Sarepta Therapeutics, Inc.;215 First Street;Cambridge, Massachusetts, 02142 October 31, 2012
(2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro- 1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid Primary biliary cirrhosis Albireo AB;Arvid Wallgrens Backe 20, S-413 46 Gothenburg; October 31, 2012
Coagulation factor IX (recombinant) Hemophilia B Baxalta US, Inc.;One Baxter Way;Westlake Village, California, 91362 October 31, 2012
(2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro- 1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid Progressive familial intrahepatic cholestatis Albireo AB;Arvid Wallgrens Backe 20, S-413 46 Gothenburg; October 31, 2012
2′-O-methyl phosphorothioate 5′-GCUAGGUUUACGGGACCUCU-3′ Amyotrophic lateral sclerosis LifeSplice Pharma LLC;1 Great Valley Parkway, Suite 20;Malvern, Pennsylvania, 19355 October 31, 2012
5ÿ¿ÿÿÿ¢ÿ¿ÿÿ¬”-GAATATTAACAIACTGACAAGTC-3ÿ¿ÿÿÿ¢ÿ¿ÿÿ¬” Prophylaxis following documented or suspected exposure to marburg virus Sarepta Therapeutics, Inc.;215 First Street;Cambridge, Massachusetts, 02142 October 31, 2012
Crenolanib Acute myeloid leukemia AROG Pharmaceuticals, LLC;12400 Coit Road, Suite 570;Dallas, Texas, 75251 October 31, 2012
5ÿÂâÿì”-GAATATTAACAIACTGACAAGTC-3ÿÂâÿì” Prophylaxis following documented or suspected exposure to marburg virus Sarepta Therapeutics, Inc.;215 First Street, Suite 7;Cambridge, MA, 02142 October 31, 2012
5ÿ¿ÿÿÿ¢ÿ¿ÿÿ¬”-GAATATTAACAIACTGACAAGTC-3ÿ¿ÿÿÿ¢ÿ¿ÿÿ¬” Prophylaxis following documented or suspected exposure to marburg virus Sarepta Therapeutics, Inc.;215 First Street, Suite 415;Cambridge, Massachusetts, 02142 October 31, 2012
5ÿ¿ÿÿÿ¢ÿ¿ÿÿ¬”-GAATATTAACAIACTGACAAGTC-3ÿ¿ÿÿÿ¢ÿ¿ÿÿ¬” Prophylaxis following documented or suspected exposure to marburg virus Sarepta Therapeutics, Inc.;215 First Street, Suite 415;Cambridge, Massachusetts, 02142 October 31, 2012
Crizotinib Anaplastic large cell lymphoma Pfizer, Inc.;10646 Science Center Drive;San Diego, California, 92121 September 28, 2012
ADF-APO-CCN-GUC-K67-MET-MMP-MUC-RGS; HLA class I/II binding tumor associated peptides Renal cancer Immatics Biotechnologies GmbH;Paul-Ehrlich-Strasse 15, 72072 Tubingen; September 28, 2012
Liposomal �}±-galactosylceramide Graft Versus Host Disease REGiMMUNE Corporation;320 Logue Avenue;Mountain View, California, 94043 September 28, 2012
Carbon monoxide Sickle cell anemia Hillhurst Biopharmaceuticals, Inc.;2029 Verdugo Blvd, Suite 125;Montrose, California, 91020 September 28, 2012
Amatuximab Malignant pleural mesothelioma Morphotek, Inc.;210 Welsh Pool Road;Exton, Pennsylvania, 19341 September 28, 2012
Japanese encephalitis vaccine, inactivated, adsorbed Japanese encephalitis Intercell AG;Campus Vienna Biocenter 3, A-1030;Vienna September 25, 2012
Synthetic double-stranded siRNA oligonucleotide against caspase 2 mRNA Anterior ischemic optic neuropathy Quark Pharmaceuticals, Inc.;6501 Dumbarton Circle;Fremont, California, 94555 September 25, 2012
Dantrolene sodium Heat stroke Eagle Pharmaceuticals, Inc.;50 Tice Blvd, Suite 315;Woodcliff Lake, New Jersey, 07677 September 25, 2012
H-Leu-Pro-Pro-Ser-Arg-OH Kaposi sarcoma Immuno Tech, Inc.;6329 256th Street, NE;Arlington, Washington, 98223 September 20, 2012
4-(4-{[2-(4-chlorophenyl)-4,4-dimethylcyclohex-1-en-1-yl]methyl}piperazin-1-yl)-N-({3-nitro-4-[tetrahydro-2H-pyran-4-ylmethyl)amino]phenyl}sulfonyl)-2-(1H-pyrrolo[2,3-b]pyridin-5-yloxy)benzamide Chronic lymphocytic leukemia AbbVie, Inc.;1 N. Waukegan Rd.;North Chicago, IL, 60064 September 20, 2012
Venetoclax Chronic lymphocytic leukemia AbbVie, Inc;1 North Waukegan Road, Dept. PA77, Bldg. AP30-1;North Chicago, Illinois, 60064 September 20, 2012
Alfimeprase ST-elevation myocardial infarcted patients who are undergoing primary percutaneous coronary intervention Niche Therapeutics, LLC;2009 Morton Avenue;Ann Arbor, Michigan, 48104 September 20, 2012
Canakinumab TNF-receptor associated periodic syndrome (TRAPS) Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 September 4, 2012