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Treatment Database

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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Product Indication Date Designated
ceramide Acute Myeloid Leukemia Dec 18, 2018
venetoclax + idasanutlin Acute myeloid leukemia Dec 17, 2018
mosunetuzumab Follicular lymphoma Dec 17, 2018
autologous T cells genetically modified to express CD123 specific hinge-optimized CD28-costimulatory chimeric receptor and a truncated human epidermal growth factor receptor Blastic Plasmacytoid Dendritic Cell Neoplasm Dec 17, 2018
autologous CD34+ cell- enriched population transduced with lentiviral vector encoding the codon-optimized human alpha-galactosidase A complementary deoxyribonucleic acid sequence. Fabry Disease Dec 17, 2018
15(S)-hydroxy-(5Z,8Z,11Z,13E,17Z)-eicosapentaenoic acid ethyl ester Idiopathic pulmonary fibrosis Dec 17, 2018
(1S,3S)-3-((2-methyl-6-(1-methyl-5-(((methyl(propyl)carbamoyl)oxy)methyl)-1H-1,2,3-triazol-4-yl)pyridin-3-yl)oxy)cyclohexane-1-carboxylic acid Idiopathic Pulmonary Fibrosis Dec 17, 2018
poly (lactide-co-glycolide)-polyethylene glycol nanoparticles encapsulating quercetin Glioblastoma Dec 17, 2018
CDC-like kinase inhibitor of the Wnt Pathway Pancreatic cancer Dec 17, 2018
Venglustat Autosomal dominant polycystic kidney disease Dec 14, 2018
Sodium 2-hydroxylinoleate Biliary tract cancer Dec 14, 2018
Pevonedistat Myelodysplastic syndromes Dec 14, 2018
Marizomib Glioma Dec 14, 2018
Lonafarnib Hutchinson-Gilford progeria syndrome Dec 14, 2018
Ivacaftor, potassium(benzenesulfonyl)({[6-(3-{2-[1-(trifluoromethyl) cyclopropyl]ethoxy}-1H-pyrazol-1-yl)-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridin-3-yl]carbonyl})azanide, tezacaftor Cystic fibrosis Dec 14, 2018
Ivacaftor, N-(1,3-dimethyl-1H-pyrazole-4-sulfonyl)-6-[3-(3,3,3-trifluoro-2,2-dimethylpropoxy)-1H-pyrazol-1-yl]-2-[(4S)-2,2,4-trimethylpyrrolidin-1-yl]pyridine-3-carboxamide, tezacaftor Cystic fibrosis Dec 14, 2018
Human anti-promyostatin monoclonal antibody Spinal muscular atrophy Dec 14, 2018
Bromelain Pseudomyxoma peritonei Dec 14, 2018
Bifunctional fusion protein composed of two extracellular domains of transforming growth factor beta receptor II fused with a human immunoglobulin G1 monoclonal antibody against programmed death ligand 1 Biliary tract cancer Dec 14, 2018
Allogeneic ABCB5-positive limbal stem cells Limbal stem cell deficiency Dec 14, 2018