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
O-(3-piperidino-2-hydroxyl-1-propyl)-nicotinic acid amidoxime hydrochloride Duchenne muscular dystrophy Vudbenk Life Sciences;Tancsics Mihaly utca 18, 5900 Oroshaza; January 15, 2013
5-aminolevulinic acid Acute respiratory distress syndrome NX Development Corporation;1827 South Bayshore Lane;Miami, Florida, 33133 January 15, 2013
Ezatiostat hydrochloride Myelodysplastic syndrome Telik, Inc.;700 Hansen Way;Palo Alto, California, 94304 January 9, 2013
P140K MGMT transduced human CD34 cells Glioblastoma Lentigen Corporation;910 Clopper Road, Suite 200 South Building;Gaithersburg, Maryland, 20878 January 9, 2013
Tiprelestat Pulmonary Arterial Hypertension Proteo Biotech AG;Am Kiel-Kanal 44; December 28, 2012
Elafin Pulmonary Arterial Hypertension Proteo Biotech AG;Am Kiel-Kanal 44; December 28, 2012
S-nitrosoglutathione Severe pre-eclampsia Salupont Consulting Ltd;The Old Quarters, Hazells Hall, Everton Road, Sandy;Bedfordshire SG19 2DD December 28, 2012
PGC-C12E-terlipressin Refractory ascites due to all etiologies except for cancer PharmaIN Corporation;19805 N. Creek Pkwy, Suite 200;Bothell, WA, 98011 December 27, 2012
Adeno-associated viral vector serotype 9 containing human N-acetylglucosaminidase alpha gene Mucopolysaccharidosis type IIIB (Sanfilippo B syndrome) Laboratorios del Dr. Esteve, S.A.;Av. Marie de Deu de Montserrat, 221;Barcelona December 27, 2012
Adeno-associated viral vector containing DNA encoding an RNAi targeting rhodopsin in combination with an adeno-associated viral vector containing DNA encoding a rhodopsin gene Retinitis pigmentosa Spark Therapeutics Ireland Ltd.;Smurfit Institute of Genetics, Trinity College;Dublin 2 December 13, 2012
Apolipoprotein E mimetic peptide Homozygous Familial Hypercholesterolemia LipimetiX Development, LLC;5 Commonwealth Road, Suite 2A;Natick, Massachusetts, 01760 December 3, 2012
Yttrium(90Y)-DTPA-radiolabelled chimeric monoclonal antibody against frizzled homologue 10 Soft tissue sarcoma OncoTherapy Science, Inc.;Kanagawa Science Park R & D D-6F, Kawasaki-shi, Kanagawa, 213-0012; December 3, 2012
Afatinib Non-small cell lung cancer Boehringer Ingelheim Pharmaceuticals, Inc.;P. O. Box 368, 900 Ridgebury Road;Ridgefield, Connecticut, 06877 December 3, 2012
Diazoxide Prader-Willi syndrome Sedogen, LLC;1701 East Lake Avenue, Suite260;Glenview, Illinois, 60025 December 3, 2012
Ibrutinib Mantle cell lymphoma Pharmacyclics, LLC;995 E. Arques Avenue;Sunnyvale, California, 94085 December 3, 2012
Recombinant Human Factor VIIa Variant Routine prophylaxis to prevent bleeding episodes in patients with hemophilia A and B patients with inhibitors Pfizer, Inc.;500 Arcola Road;Collegeville, PA, 19426 November 30, 2012
Marzeptacog alfa (activated) Hemophilia A Catalyst Biosciences, Inc.;260 Littlefield Avenue;South San Francisco, California, 94080 November 30, 2012
Chimeric monoclonal antibody to claudin 18 splice variant 2 Stomach Cancer Astellas Pharma Global Development Inc.;1 Astellas Way;Northbrook, Illinois, 60062 November 20, 2012
Adeno-associated virus transgene of follistatin Duchennes and Becker's muscular dystrophy Milo Biotechnology;2322 Delaware Drive;Cleveland Heights, Ohio, 44106 November 19, 2012
Brentuximab vedotin Mycosis fungoides Seattle Genetics, Inc.;21823 30th Drive Southeast;Bothell, Washington, 98021 November 19, 2012
Combretastatin A 1 diphosphate Acute myeloid leukemia Mateon Therapeutics, Inc.;701 Gateway Blvd, Suite 210;South San Francisco, California, 94080 November 19, 2012
Beclomethasone 17,21-dipropionate Prevention of death following a potentially lethal dose of total body irradiation during or after a radiation disaster Soligenix, Inc;29 Emmons Drive, Suite C-10;Princeton, New Jersey, 08540 November 19, 2012
Extract of sorghum bicolor extract Sickle cell anemia Invenux, LLC;1550 Main Street, Suite A3;Windsor, Colorado, 80550 November 19, 2012
1′-{[5-(trifluoromethyl)-2-furyl]methyl}spiro[furo[2,3-f][1,3]benzodioxole-7,3′-indol]-2′(1’H)-one Erythromelalgia Teva Pharmaceuticals;41 Moores Road, P. O. Box 4011;Frazer, Pennsylvania, 19355 November 19, 2012
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