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
3-(1�-butyloxy)ethyl-3-deacetyl-bacteriopurpurin-18-N-butylimide methyl ester Glioma Dec 06, 2017
D-amino acid oxidase inhibitor Friedreich's ataxia (FRDA) Dec 06, 2017
cholesterol 24S-hydroxylase inhibitor Lennox-Gastaut syndrome Dec 06, 2017
idasanutlin Polycythemia vera Nov 29, 2017
cotsiranib Cholangiocarcinoma (CCA) Nov 29, 2017
cholesterol 24S-hydroxylase inhibitor Dravet syndrome Nov 29, 2017
Eubacterial Spores, Purified Suspension, Encapsulated Pediatric ulcerative colitis Nov 27, 2017
CD4-mediated activated regulatory T cells Graft-versus-host disease Nov 27, 2017
eflornithine Neuroblastoma Nov 22, 2017
Synthetic PreImplantation Factor (H-Met-Val-Arg-Ile-Lys-Pro-Gly-Ser-Ala-Asn-Lys-Pro-Ser-Asp-Asp-OH Autoimmune hepatitis (AIH) Nov 22, 2017
(1R,2R)-1,2-cyclohexane-diaminepyrophosphato-platinum(II) Multiple myeloma Nov 22, 2017
2�-O-(2-methoxyethyl) modified antisense oligonucleotide targeting exon 13 in the USH2A gene Retinitis pigmentosa Nov 20, 2017
human IgG2 anti-monoclonal antibody directed against mucosal addressin cell adhesion molecule (MAdCAM) Pediatric patients with Ulcerative Colitis (UC) Nov 16, 2017
daxibotulinumtoxinA Cervical dystonia Nov 16, 2017
cholera toxin B subunit Ulcerative colitis in pediatric patients Nov 16, 2017
tazemetostat Follicular lymphoma (FL) Nov 14, 2017
adeno-associated virus type 2 encoding human aromatic L-amino acid decarboxylase Aromatic L-amino acid decarboxylase deficiency Nov 14, 2017
Tiratricol Allan-Herndon-Dudley syndrome Nov 08, 2017
Tamoxifen citrate Duchenne muscular dystrophy Nov 08, 2017
Recombinant adeno-associated viral vector serotype 9 containing human iduronate-2-sulfatase gene Mucopolysaccharidosis type II (Hunter’s syndrome) Nov 08, 2017