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
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
Afatinib Fanconi anaemia Dec 14, 2018
Adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase Phenylalanine hydroxylase deficiency Dec 14, 2018
Adeno-associated viral vector expressing human 21-hydroxylase Congenital adrenal hyperplasia Dec 14, 2018
Acetylcysteine Pseudomyxoma peritonei Dec 14, 2018
6-fluoro-9-methyl-9H-pyrido[3,4-b]-indole Sudden sensorineural hearing loss Dec 14, 2018
6,8-bis(benzylthio)octanoic acid Pancreatic cancer Dec 14, 2018
(4-{(2S,4S)-4-ethoxy-1-[(5-methoxy-7-methyl-1H-indol-4-yl)methyl]piperidin-2-yl}benzoic acid-hydrogen chloride(1/1)) C3 glomerulopathy Dec 14, 2018
(2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid Biliary atresia Dec 14, 2018