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
upamostat Pancreatic cancer Oct 16, 2017
Synthetic cyclic 8 amino acid analogue of human unacylated ghrelin Prader-Willi syndrome Oct 16, 2017
Seladelpar Primary biliary cholangitis Oct 16, 2017
Recombinant adeno-associated viral vector serotype 5 encodingStaphylococcus aureusCas9 endonuclease and two guide RNAs complementary to two regions of intron 26 of theCEP290gene Leber’s congenital amaurosis Oct 16, 2017
Ofranergene obadenovec Ovarian cancer Oct 16, 2017
Humanised monoclonal antibody targeting B-cell maturation antigen conjugated with maleimidocaproyl monomethyl auristatin F Multiple myeloma Oct 16, 2017
Glucopyranosyl lipid A Follicular lymphoma Oct 16, 2017
Cannabidivarin Rett syndrome Oct 16, 2017
Cannabidiol West syndrome Oct 16, 2017
Autologous ex-vivo-expanded peripheral polyclonal lymphocytes enriched in activated natural killer cells Multiple myeloma Oct 16, 2017
Adenoviral vector of serotype 5 modified to contain a chimeric sequence consisting of a minimal urokinase-type plasminogen activator receptor promoter preceded by three Notch-responsive elements, and coated with oligopeptide end-modified poly (beta-amino) esters Pancreatic cancer Oct 16, 2017
5-amino-1-(2-methyl-1H-benzo[d]imidazol-5-yl)-1H-pyrazol-4-yl 1H-indol-2-yl ketone mono[(S)-2-hydroxysuccinate] Biliary tract cancer Oct 16, 2017
2-[N-(2-hydroxyethyl)]-N-(4-methoxybenzenesulfonyl)]amino-N-(4-chlorocinnamyl)-N-methylbenzylamine Charcot-Marie-Tooth disease Oct 16, 2017
(S)-3-((S)-2-(2-((2,6-difluorophenyl)amino)-2-oxoacetamido)propanamido)-4-oxo-5-(2,3,5,6-tetrafluorophenoxy)pentanoic acid Primary sclerosing cholangitis Oct 16, 2017
adeno-associated virus (AAV2) capsid variant carrying a transgene encoding a codon-optimized human choroideremia (CHM) gene Choroideremia (CHM) Oct 12, 2017
synthetic cyclic 8 amino acid analog of human unacylated ghrelin Prader-Willi syndrome (PWS) Oct 12, 2017
avelumab Gastric cancer (GC) including cancer of the gastroesophageal junction (GEJ) Oct 11, 2017
autologous glioma tumor cells treated with 18-mer oligodeoxynucleotide sequence complementary to insulin-like growth factor type 1 receptor (IGF-1R) codons 2-7 in the IG-1R gene Malignant glioma Oct 11, 2017
filgotinib maleate Pediatric ulcerative colitis (UC) Oct 05, 2017
autologous T lymphocyte enriched population cultured in the rpesence of phosphatidylinositol 3-kinase inhibitor (bb007) that contains cells transduced with an anti-human B cell maturation antigen (BCMA02) chimeric antigen receptor (CAR) lentiviral vector encoding a CAR that targets human BCMA Multiple myeloma Oct 05, 2017