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-tert-butyl-3-[5-methyl-2-{[4-(2-pryyolidin-1-ylethoxy)phenyl]amino}pyrimidin-4-yl-amino] benzenesulfonamide dihydrochloride monohydrate Myelofibrosis Impact Biomedicines;12526 High Bluff Drive, Suite 260;San Diego, California, 92130 May 18, 2009
Elosulfase alfa Mucopolysaccharidosis Type IVA BioMarin Pharmaceutical Inc.;105 Digital Drive;Novato, California, 94949 May 15, 2009
Aztreonam Bronchiectasis Gilead Sciences, Inc.;2025 1st Avenue;Seattle, Washington, 98121 May 15, 2009
Dimebon Huntington disease Medivation, Inc.;201 Spear Street;San Francisco, California, 94105 May 12, 2009
S-nitrosoglutathione Cystic fibrosis N30 PHARMAceuticals, LLC;3122 Sterling Circle;Boulder, Colorado, 80301 May 12, 2009
Recombinant adeno-associated virus (serotype 2) (rAAV2) gene transfer agent expressing RPE65 Leber congenital amaurosis AmpliPhi Biosciences Corporation;601 Union St., Suite 4200;Seattle, Washington, 98111 May 11, 2009
Ascorbic acid Charcot-Marie-Tooth disease Murigenetics SAS;Faculte de Medecine Timone, 13005 Marseille; May 11, 2009
Uridine triacetate An antidote in the treatment of 5-fluorouracil or capecitabine poisoning Wellstat Therapeutics Corp.;14200 Shady Grove Road, Suite 600;Rockville, Maryland, 20850 May 1, 2009
Antihemophilic factor (recombinant) formulated with sucrose reconstituted with liposome diluent Hemophilia A Bayer HealthCare LLC;555 Whiteplains Road;Tarrytown, New York, 10591 April 29, 2009
Eculizumab Atypical hemolytic uremic syndrome Alexion Pharmaceuticals, Inc.;352 Knotter Drive;Cheshire, Connecticut, 06410 April 29, 2009
Antisense oligonucleotide directed against connexin43 Persistent corneal epithelial defects CoDa Therapeutics, Inc.;10505 Sorrento Valley Road;San Diego, California, 92121 April 27, 2009
4,6,8-trihydroxy-10-(3,7,11-trimethyldodeca-2,6,10-trienyl)-5,10-dihydrodibenzo b,e][1,4]diazepin-11-one Glioma Thallion Pharmaceuticals, Inc.;7150 Alexander-Fleming; April 27, 2009
D-mannitol and L-proline Cockayne syndrome DNage B.V.;Darwinweg 24; April 20, 2009
Caplacizumab Thrombotic thrombocytopenic purpura Ablynx NV;Technologiepark 21, B-9052;Zwijnaarde April 14, 2009
ALX-0081 nanobody, directed towards the human A1 domain of von Willebrand factor Thrombotic thrombocytopenic purpura Ablynx NV;Technologiepark 21, B-9052;Zwijnaarde April 14, 2009
Chimeric murine/human IgG1 monoclonal antibody to human interleukin-3 receptor alpha chain Acute myeloid leukemia CSl Limited;45 Poplar Road; April 3, 2009
Allogeneic human aortic endothelial cells cultured in a porcine gelatin matrix Prevention of arteriovenous fistula or arteriovenous graft failure in patients with end satge renal disease receiving hemodialysis or preparing for hemodialysis Shire;300 Shire Way;Lexington, Massachusetts, 02421 April 3, 2009
Anti-T cell receptor murine monoclonal antibody Prophylaxis of acute rejection of solid organ transplantation Tolera Therapeutics, Inc.;4717 Campus Drive #800;Kalamazoo, Michigan, 49008 April 3, 2009
Vonapanitase Focal segmental glomerulosclerosis Proteon Therapeutics, Inc.;200 West Street;Waltham, Massachusetts, 02451 April 3, 2009
D-Phe-Cys-Tyr-D-Trp-Lys-Cys-Thr-Nh2 (disulfide bridge: 2-6) acetate Melanoma, stages IIb through IV Thallion Pharmaceuticals, Inc.;7150 Alexander-Fleming; March 19, 2009
2-(2-phenylvinyl)-4-[4-methylpiperazin-l-yl)]-6-(5-methyl-2H-pyrazol-3-ylamino)-pyrimidine L(+) tartrate salt Ovarian cancer EntreMed, Inc.;Research Triangle Park Center, Suite 400;Durham, North Carolina, 27703 March 18, 2009
N-(5-tert-Butylisoxazol-3-yl)-N’-{4-[7-(2-(morpholin-4-yl)ethoxy) imidazo[2,1-b][1,3]benzothiazol-2-yl]phenyl}urea di-hydrochloride salt Acute myeloid leukemia Daiichi Sankyo, Inc.;211 Mount Airy Road;Basking Ridge, New Jersey, 07920 March 18, 2009
Technetium Tc99m sulfur colloid injection, lyophilized For localization of sentinel lymph nodes in patients with melanoma Pharmalucence, Inc.;10 DeAngelo Drive;Bedford, Massachusetts, 01730 March 17, 2009
Beclomethasone Ulcerative colitis Aptalis Pharma US, Inc.;100 Somerset corporate Blvd;Bridgewater, New Jersey, 08807 March 4, 2009
Enzastaurin Diffuse Large B-cell Lymphoma Denovo Biopharma LLC;6331 Nancy Ridge Drive;San Diego, California, 92121 March 4, 2009