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
Thioureidobutyronitrile Pancreatic cancer Innovation Pharmaceuticals, Inc.;100 Cummings Center, Suite 151-B;Beverly, Massachusetts, 01915 January 20, 2016
90Y-DOTA-tyr3-Octreotide Neuroendocrine tumors M. Sue O'Dorisio, MD, PhD;2519 JPP, 200 Hawkins Drive;Iowa City, Iowa, 52242 January 20, 2016
Glibenclamide Neonatal diabetes AMMTeK January 15, 2016
Mifepristone Ovarian cancer Corcept Therapeutics, Inc.;149 Commonwealth Drive;Menlo Park, California, 94025 January 14, 2016
Pembrolizumab Primary mediastinal B cell lymphoma Merck, Sharp & Dohme Corp.;One Merck Drive;Whitehouse Station, New Jersey, 08889 January 14, 2016
Recombinant human serum albumin human growth hormone Growth hormone deficiency TEVA Branded Pharmaceutical Products R&D;, Inc.;41 Moores Road, P. O. Box 4011;Frazer, Pennsylvania, 19355 January 13, 2016
His-His- Ile-Tyr-Leu-Gly-Ala-Val-Asn-Tyr-Ile-Tyr-NH2 Retinal detachment ONL Therapeutics, Inc;1600 Huron Parkway, Second Floor;Ann Arbor, Michigan, 48109 January 13, 2016
S-nitrosoglutathione reductase inhibitor Cystic fibrosis Alpine Immune Sciences;201 Elliott Ave. West, Suite 230;Seattle, Washington, 98119 January 13, 2016
Synthetic double-stranded oligomer specific to theSERPINA1gene and containing a cholesterol-conjugated, acyclic nucleobase analogue Congenital alpha-1 antitrypsin deficiency Pharma Gateway AB January 11, 2016
(S)-N-(5-((R)-2-(2,5-difluorophenyl)pyrrolidin-1-yl)pyrazolo[1,5-a]pyrimidin-3-yl)-3-hydroxypyrrolidine-1-carboxamide hydrogen sulfate Soft tissue sarcoma Loxo Oncology Limited January 11, 2016
Recombinant humanized IgG4 monoclonal antibody that binds human colony stimulating factor 1 receptor Pigmented villonodular synovitis and tenosynovial giant cell tumor Five Prime Therapeutics, Inc.;2 Corporate Drive;South San Francisco, California, 94080 January 11, 2016
Sodium benzoate Hyperargininaemia Syri Limited January 11, 2016
Live attenuatedListeria monocytogenesdeltaactA/deltainlBstrain expressing human mesothelin Pancreatic Cancer Aduro Biotech Holdings, Europe B.V. January 11, 2016
Two allogenic irradiated pancreatic tumour cell lines Pancreatic Cancer Aduro Biotech Holdings, Europe B.V. January 11, 2016
Entolimod Acute Radiation Syndrome Acute radiation syndrome TMC Pharma Services Ltd January 11, 2016
Sodium benzoate Argininosuccinic Aciduria Syri Limited January 11, 2016
Live attenuatedListeria monocytogenestransfected with plasmids encoding the HPV-16E7 protein fused to a truncated fragment of the Lm protein listeriolysin O Anal cancer Dr Ulrich Granzer January 11, 2016
Cabiralizumab Pigmented villonodular synovitis and tenosynovial giant cell tumor Five Prime Therapeutics, Inc.;2 Corporate Drive;South San Francisco, California, 94080 January 11, 2016
Gemcitabine Bile duct cancer InnoPharmax, Inc.;9F, No. 22, Lane 478, Rueiguang Road;Taipei January 11, 2016
Dendritic-cell targeting, lentiviral vector encoding the NY-ESO-1 gene Soft tissue sarcoma Immune Design Corp.;1616 Eastlake Ave. E, Suite 310;Seattle, Washington, 98102 January 6, 2016
Recombinant NY-ESO-1 protein mixed with glucopyranosyl lipid A Soft tissue sarcoma Immune Design Corp.;1616 Eastlake Ave. E, Suite 310;Seattle, Washington, 98102 January 6, 2016
Small Molecule Inhibitor of Exon 17 mutant KIT Gastrointestinal Stromal Tumors Blueprint Medicines Corporation;38 Sidney Street, Suite 200;Cambridge, Massachusetts, 02139 January 6, 2016
1-(2-Nitro-imidazolyl)-3-[18F]fluoro-2-propanol;1H-1-(3-[18F]fluoro-2-hydroxypropyl)-2-nitroimidazole As a diagnostic for clinical management of soft tissue sarcoma, including rhabdomyosarcoma Advanced Imaging Projects, LLC;7947 Brookside Ct.;Lake Worth, Florida, 33467 January 6, 2016
5-imino-13-deoxydoxorubicin HCl Soft tissue sarcoma Monopar Therapeutics, Inc.;5 Revere Drive, Suite 200;Northbrook, Illinois, 60062 December 31, 2015
Anlotinib hydrochloride Ovarian cancer Advenchen Laboratories, LLC;887 Patriot Drive, Suite A;Moorpark, California, 93021 December 31, 2015