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
Gallium [Ga-68]-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-cyclic(2-7)disulfide Diagnostic for the Management of Gastro-Entero-Pancreatic Neuroendocrine Tumors Advanced Accelerator Applications;20 Rue Diesel, 01630;Saint Genis Pouilly December 31, 2013
NorLeu3-Angiontensin(1-7)) [NorLeu3-A(1-7)] Leakage from the surgical site following penetrating keratoplasty US Biotest, Inc.;231 Bonetti Dr.,, Suite 240;San Luis Obispo, California, 93401 December 31, 2013
N-acetyl cysteine amide Retinitis pigmentosa Nacuity Pharmaceuticals, Inc.;Fort Worth Club Building, 306 W 17th Street;Fort Worth, Texas, 76106 December 31, 2013
Gallium [Ga-68]-N-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-cyclic(2-7)disulfide Diagnostic for the clinical management of neuroendocrine tumors Advanced Accelerator Applications, USA;350 Fifth Avenue, Suite 6902;New York, New York, 10118 December 31, 2013
Tolcapone Hereditary ATTR amyloidosis Corino Therapeutics, Inc.;57 West 57th Street, 18th Floor;New York, New York, 10019 December 24, 2013
Erdosteine Bronchiectasis Alitair Pharmaceuticals, Inc.;55 Madison Avenue, Suite 400;Morristown, New Jersey, 07960 December 20, 2013
Tacrolimus Prophylaxis of organ rejection in patients receiving allogeneic kidney transplant Veloxis Pharmaceuticals, Inc.;499 Thornall Street, 3rd Floor;Edison, NJ, 08837 December 20, 2013
CD40/CD80/CD86 modified autologous dendritic cell therapy Treatment Type 1 diabetes mellitus patients with residual beta cell function DiaVacs, Inc.;410 Tower Drive;Edgewater, New Jersey, 07020 December 20, 2013
Fenfluramine HCI Dravet Syndrome Zogenix, Inc.;5858 Horton Street, Suite 455, RG9 2LT;Emeryville, California December 20, 2013
Tacrolimus Prophylaxis of organ rejection in patients receiving allogeneic kidney transplant Veloxis Pharmaceuticals, Inc.;499 Thornall Street, 3rd Floor;Edison, New Jersey, 08837 December 20, 2013
Human monoclonal antibody inhibitor of mannan-binding lectin-associated serine protease-2 (MASP-2) Prevention (inhibition) of complement-mediated thrombotic microangiopathy Omeros Corporation;201 Elliott Avenue West;Seattle, Washington, 98119 December 16, 2013
Marizomib Multiple myeloma Triphase Research and Development I Corp.;3210 Merryfield Row;San Diego, CA, 92121 December 13, 2013
O2-(2,4-dinitrophenyl) 1-[(4-ethoxycarbonyl)piperazin-1yl]diazen-1-ium-1,2-diolate Multiple myeloma JSK Therapeutics, Inc.;85 Fort Douglas Boulevard, P.O. Box 58986;Salt Lake City, Utah, 84158 December 13, 2013
Marizomib Multiple myeloma Triphase Research & Development I Corp.;3366 N. Torrey Pines Court, Suite 210;La Jolla, California, 92037 December 13, 2013
Canakinumab Familial Mediterranean fever Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 December 5, 2013
Canakinumab Hyper-IgD syndrome Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 December 5, 2013
Sacituzumab govitecan Small cell lung cancer Immunomedics, Inc.;300 The American Road;Morris Plains, New Jersey, 07950 November 27, 2013
N-(3-fluorobenzyl)-2-(5-(4-morpholinophenyl) pyridin-2-yl) Acetamide Besylate Gliomas Kinex Pharmaceuticals, Inc.;701 Ellicott Street;Buffalo, New York, 14203 November 26, 2013
Bendamustine hydrochloride Follicular lymphoma Cephalon, Inc.;A wholly owned subsidiary of Teva Pharmaceuticals, PO Box 4011;Frazer, Pennsylvania, 19355 November 26, 2013
Siponimod Polymyositis Novartis Pharmaceuticals Corporation;One Health Plaza;East Hanover, New Jersey, 07936 November 26, 2013
Angiotensin (1-7) Limb-girdle muscular dystrophy Tarix Pharmaceuticals Ltd;12 Bow Street;Cambridge, Massachusetts, 02138 November 26, 2013
Adeno-associated viral vector containing the human NADH Dehydrogenase 4 Gene Leber Hereditary Optic Neuropathy Gen Sight Biologics;; November 20, 2013
Filgrastim Subjects at risk of developing myelosuppression after a radiological or nuclear incident Amgen, Inc.;One Amgen Center Drive, M/S 17-1-C;Thousand Oaks, California, 91320 November 20, 2013
Pegfilgrastim Subjects at risk of developing myelosuppression after a radiological or nuclear incident Amgen, Inc.;One Amgen Center Drive, M/S 17-1-C;Thousand Oaks, California, 91320 November 20, 2013
Emricasan Liver transplant recipients with reestablished fibrosis to delay the progression to cirrhosis and end stage liver disease Conatus Pharmaceuticals Inc.;16745 W. Bernardo Drive, Suite 200;San Diego, California, 92127 November 20, 2013