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
Abatacept Ataxia Telangiectasia Bristol-Myers Squibb Co. P.O Box 4000 Princeton, New Jersey, 08543 January 9, 2018
2-O, 3-O, desulfated heparin Pseudo-Zellweger syndrome Cantex Pharmaceuticals, Inc. 1792 Bell Tower Lane Weston, Florida, 33326 January 9, 2018
Mosedipimod Complex 5 mitochondrial respiratory chain deficiency Enzychem Lifesciences Corporation 59, Bio valley-ro Jecheon-si January 9, 2018
Fenretinide 1-(2-Nitro-imidazolyl)-3-[18F]fluoro-2-propanol;1H-1-(3-[18F]fluoro-2-hydroxypropyl)-2-nitroimidazole SciTech Development, LLC 281 Kercheval Avenue Grosse Pointe Farms, Michigan, 48236 January 9, 2018
DaxibotulinumtoxinA 1-(2-Nitro-imidazolyl)-3-[18F]fluoro-2-propanol;1H-1-(3-[18F]fluoro-2-hydroxypropyl)-2-nitroimidazole Revance Therapeutics, Inc. 7555 Gateway Boulevard Newark, California, 94560 January 9, 2018
Lentiviral transfected chimeric antigen receptor T-cells modified to target B-cell maturation antigen and CS1 1-(2-Nitro-imidazolyl)-3-[18F]fluoro-2-propanol;1H-1-(3-[18F]fluoro-2-hydroxypropyl)-2-nitroimidazole iCell Gene Therapeutics 25 Health Sciences Drive, Suite 118 Stony Brook, New York, 11793 January 9, 2018
Cholera toxin B subunit 1-(2-Nitro-imidazolyl)-3-[18F]fluoro-2-propanol;1H-1-(3-[18F]fluoro-2-hydroxypropyl)-2-nitroimidazole Aliment Wellness LLC 9462 Brownsboro Road, Suite 340 Louisville, Kentucky, 40241 January 9, 2018
Three-dimensional bioprinted therapeutic liver tissue Organovo Inc. 6275 Nancy Ridge Drive San Diego, California, 92121 January 9, 2018
Eflornithine + sulindac 1-(2-Nitro-imidazolyl)-3-[18F]fluoro-2-propanol;1H-1-(3-[18F]fluoro-2-hydroxypropyl)-2-nitroimidazole Cancer Prevention Pharmaceutical, Inc. 1760 East River Road, Suite 250 Tucson, Arizona, 85718 January 9, 2018
Milrinone Right heart failure (RHF) in left ventricular assist device (LVAD) patients Cardiora Pty Ltd;Level 9, 278 Collins Street;Melbourne January 8, 2018
Atezolizumab + Bevacizumab Hepatocellular Carcinoma Genentech, Inc.;1 DNA Way, Mail Stop # 355e;South San Francisco, California, 94080 January 8, 2018
Cannabidiol (CBD) and Delta-9-tetrahydrocannabinol (THC) Glioblastoma AXIUM Pharmaceuticals, Inc.;265 Eastchester Drive, Suite 133, #201;High Point, North Carolina, 27262 January 8, 2018
Antibody drug conjugate targeting dipeptidase 3 (DPEP3) DPEP3-positive ovarian, fallopian tube, and primary peritoneal cancers AbbVie Stemcentrx, LLC;1 North Waukegan Road;Chicago, Illinois, 60064 January 8, 2018
Eflornithine + sulindac Pancreatic Cancer Cancer Prevention Pharmaceutical, Inc.;1760 East River Road, Suite 250;Tucson, Arizona, 85718 January 8, 2018
Cryopreserved human umbilical cord Spina bifida TissueTech, Inc.;7300 Corporate Center Drive, Suite 700;Miami, Florida, 33126 January 8, 2018
Genetically modified, non-self replicating Adeno-Associated Virus serotype 9 expressing shRNA to knock down mutant PABP1 as well as a codon optimized, shRNA-insensitive, wildtype PABN1 Oculopharyngeal muscular dystrophy Benitec Biopharma;99 Mount Street, Suite 1201;North Sydney January 8, 2018
ICG140:CD19-CD123 cCAR T-cells, a chimeric antigen receptor (CAR) on the surface of T-cells directed against the target proteins CD19 and CD 123 iCell Gene Therapeutics 25 Health Sciences Drive, Suite 118 Stony Brook, New York, 11793 January 4, 2018
Resatorvid Acute-on-chronic liver failure Takeda Development Center Americas, Inc.;One Takeda Parkway;Deerfield, Illinois, 60015 January 2, 2018
Partial myostatin antigen displayed on the surface of Lactobacillus casei Duchenne muscular dystrophy BioLeaders Corporation;7F, U-Tower, 767, Sinsu-ro, Suji-gu;Yongin-si December 28, 2017
(+)-(2S)-2-(4-chloro-2-methoxyphenyl)-2-{[3-methoxy-5-(methylsulfonyl)phenyl]amino}-1-[5-(trifluoromethoxy)-1H-indol-3-yl]ethanone Dengue virus infection Janssen Research & Development, LLC;125 Trenton-Harbourton Road;Titusville, New Jersey, 08560 December 26, 2017
3-[2-(1-methylpyrrolidin-2-yl) ethyl]-6-(2-pyrrolidin-1yl) ethyl-9-benzyloxycarbonyl-12-diethyl-4,7-dioxo-3,6,9,12-tetraazadodecancarboxamide Anterior ischemic optic neuropathy Neurizon Pharma;15 Paul-Ehrlich-Stra�e;T�bingen December 26, 2017
ICG140:CD19-CD123 cCAR T-cells, a chimeric antigen receptor (CAR) on the surface of T-cells directed against the target proteins CD19 and CD 123 Acute lymphoblastic leukemia iCell Gene Therapeutics;25 Health Sciences Drive, Suite 118;Stony Brook, New York, 11793 December 26, 2017
Abatacept Graft Versus Host Disease Bristol-Myers Squibb Co.;P.O Box 4000;Princeton, New Jersey, 08543 December 26, 2017
1-{3-fluoro-4-[7-(5-methyl-lH-imidazol-2-yl)- 1-oxo-2,3-dihydro-lH-isoindo 1-4-yl]-phenyl}-3-(3-trifluoromethyl-phenyl)-urea Acute myeloid leukemia Aptose Biosciences, Inc.;12770 High Bluff Drive, Suite 120;San Diego, California, 92130 December 22, 2017
Fenretinide Follicular lymphoma SciTech Development, LLC;281 Kercheval Avenue;Grosse Pointe Farms, Michigan, 48236 December 22, 2017