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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
filgotinib maleate Pediatric Crohn's disease Jul 18, 2017
One, two, three, or four antisense oligonucleotides of Phosphorodiamidate morpholino oligomer combination that skips exons 4, 5, 6, and 7 of the gamma sarcoglycan (SGCG) gene Limb-girdle muscular dystrophy type 2C that have mutations or deletions in exons 4, 5, 6, or 7of gamma sarcoglycan gene Jul 18, 2017
Bacillus subtilisoxalate decarboxylase Primary hyperoxaluria Jul 17, 2017
Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor Diffuse large B-cell lymphoma Jul 17, 2017
Ranpirnase Ebola virus disease (EVD) Jul 14, 2017
Gilteritinib Acute myeloid leukemia (AML) Jul 13, 2017
(20S)-7-tert-butyldimethysilyl-10-hydroxycamptothecin Glioblastoma multiforme (GBM) Jul 13, 2017
electrokinetically altered saline solution with an elevated dissolved oxygen concentration Amyotrophic lateral sclerosis (ALS) Jul 10, 2017
5-cholesten-3�, 25-diol 3-sulfate sodium salt (25HC3S) Primary sclerosing cholangitis (PSC) Jul 06, 2017
(1'R,6'R)-3-(Benzylamine)-6-hydroxy-3'-methyl-4-pentyl-6'-(prop-1-en-2-yl)-[1,1'-bi(cyclohexane)]-2',3,6-triene-2,5-dione Systemic scleroderma (SSc) Jul 06, 2017
blinatumomab Diffuse large B-cell lymphoma (DLBCL) Jul 06, 2017
humanized immunoglobulin G4-variant monoclonal antibody directed against the p19 subunit of interleukin (IL)-23 Pediatric Crohn's disease Jul 06, 2017
cosyntropin Duchenne muscular dystrophy Jul 06, 2017
bardoxolone methyl Alport Syndrome Jul 03, 2017
Autologous T cells transfected with mRNA encoding hepatitis B virus antigen-specific T cell receptor Hepatocellular carcinoma Jul 03, 2017
autologous T cells transduced with retroviral vector encoding a hepatitis B virus antigen-specific T cell receptor Hepatocellular carcinoma Jul 03, 2017
antagonist of chemokine Ligand-2 Pancreatic cancer Jul 03, 2017
3-(5-amino-2-methyl-4-oxoquinazolin-3(4H)-yl)piperidine-2,6-dione hydrochloride Hepatocellular carcinoma (HCC) Jul 03, 2017
tadekinig alfa Still's disease, including adult-onset Still's disease (AoSD) and systemic juvenile idiopathic arthritis (sJIA) Jul 03, 2017
Small Molecule Bruton's Agammaglobulinemia Tyrosine Kinase (BKT) Inhibitor Pemphigus Vulgaris Jun 29, 2017