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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
Anti-transforming growth factor beta (TGF-�) trap fusion protein Systemic sclerosis Mar 25, 2019
methotrexate Ectopic pregnancy Mar 25, 2019
human monoclonal IgG2 antibody against tissue factor pathway inhibitor (TFPI) Hemophilia A Mar 25, 2019
nanatinostat and valganciclovir Angioimmunoblastic T-cell lymphoma Mar 15, 2019
meplazumab Malaria Mar 15, 2019
encapsulated rapamycin Familial adenomatous polyposis Mar 15, 2019
deferoxamine Sickle cell leg ulcers Mar 15, 2019
corticotropin releasing factor 1 receptor antagonist Congenital adrenal hyperplasia Mar 15, 2019
capmatinib Non-small cell lung cancer with MET genomic tumor aberrations Mar 15, 2019
tinostamustine hydrochloride T-cell prolymphocytic leukemia Mar 15, 2019
saracatinib Idiopathic pulmonary fibrosis Mar 11, 2019
[11C]Acetic Acid Diagnostic for the management of hepatoblastoma Feb 28, 2019
sorafenib Desmoid tumors/aggressive fibromatosis Feb 28, 2019
small molecule inhibitor of the pro-survival protein Myeloid Cell Leukemia sequence 1 (MCL 1) Multiple Myeloma Feb 28, 2019
efmitermant alfa Charcot-Marie-Tooth disease Feb 28, 2019
Autologous genetically modified CD34+ hematopoietic cells transduced with a lentiviral vector (LV-EFS-TCIRG1-WPRE) that encodes for the human TCIRG1 transgene Infantile malignant osteopetrosis Feb 28, 2019
Ac-225-4-PHenylbutyryl-His-D-Phe-Arg-Trp-Gly-Lys(eAhx-DOTA)-NH2 Uveal Melanoma Feb 28, 2019
4-(((3-chloro-1,4-dioxo-1,4 dihydronaphthalen-2-yl)amino)methyl)-N-(pyridine-3-yl)benzamide Multiple Myeloma Feb 28, 2019
(S)-1-(4-((3-amino-5-(4-amino-2-oxa-8-azaspiro[4.5]decan-8-yl)pyrazin-2-yl)thio)-3,3-difluoroindolin-1-yl)ethan-1-one Esophageal cancer Feb 28, 2019
src homology region 2-containing protein tyrosine phosphatase 2 (SHP2) inhibitor JAB-3068 Esophageal cancer Feb 28, 2019