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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
Diacerein Epidermolysis bullosa Nov 02, 2018
human astrocytes derived from astrocyte progenitor cells originating from clincial-grade human embryonic stem cells Amyotrophic lateral sclerosis Nov 01, 2018
trimetazidine Dilated cardiomyopathy Oct 30, 2018
trimetazidine Idiopathic dilated cardiomyopathy Oct 30, 2018
6'-(R)-methyl-5-O-(5-amino-5,6-dideoxy-alpha-L-talofuranosyl)-paromamine sulfate Cystic fibrosis Oct 26, 2018
(6aR,10aR)-3-(1,1-dimethylheptyl)-delta8-tetrahydro-cannabinol-9-carboxylic acid Dermatomyositis Oct 26, 2018
Recombinant adeno-associated viral vector serotype S3 containing codon-optimised expression cassette encoding human coagulation factor IX variant Haemophilia B Oct 26, 2018
Peptides YMFPNAPYL, SGQAYMFPNAPYLPSCLES, RSDELVRHHNMHQRNMTKL and PGCNKRYFKLSHLQMHSRKHTG Multiple myeloma Oct 26, 2018
Melatonin Acute radiation syndrome Oct 26, 2018
Glycine, L-alanine, L-arginine, L-aspartic acid, L-cysteine, L-cystine, L-glutamic acid, L-histidine, L-lysine monohydrate, L-methionine, L-phenylalanine, L-proline, L-serine, L-threonine, L-tryptophan, L-tyrosine, taurine Maple syrup urine disease Oct 26, 2018
Gefitinib Fanconi anaemia Oct 26, 2018
Autologous CD34+ haematopoietic stem and progenitor cells genetically modified with the lentiviral vector IDUA LV, encoding for the alpha-L-iduronidase cDNA Mucopolysaccharidosis type I Oct 26, 2018
6-(2-hydroxy-2-methylpropoxy)-4-(6-(6-((6-methoxypyridin-3-yl)methyl)-3,6-diazabicyclo[3.1.1]heptan-3-yl)pyridin-3-yl)pyrazolo[1,5-a]pyridine-3-carbonitrile Medullary thyroid carcinoma Oct 26, 2018
2-[3-(2-chloro-4-{[5-cyclopropyl-3-(2,6-dichlorophenyl)-1,2-oxazol-4-yl]methoxy}phenyl)-3-hydroxyazetidin-1-yl]pyridine-4-carboxylic acid�2-amino-2-(hydroxymethyl)propane-1,3-diol (1/1) Primary Sclerosing Cholangitis (PSC) Oct 25, 2018
autologous dendritic cells pulsed with hepatitis B virus antigens Hepatocellular carcinoma Oct 25, 2018
adipose-derived mesenchymal stem cells in a hydrogel sheet Epidermolysis Bullosa Oct 25, 2018
5-Chloro-N-(3-cyclopropyl-5-(((3R,5S)-3,5-dimethylpiperazine-1-yl)methyl)phenyl)-4-(6-methyl-1H-indol-3-yl)pyrimidin-2-amine�monohydrate Acute Myeloid Leukemia Oct 25, 2018
non-replicating, recombinant adeno-associated virus (AAV) serotype 9 (AAV9) vector containing an hCLN2 expression cassette encoding for the soluble lysosomal enzyme tripeptidyl peptidase I (TPP1) Neuronal ceroid lipofuscinosis type 2 (CLN2) disease Oct 25, 2018
humanized monoclonal antibody targeting interleukin-15 Eosinophilic esophagitis Oct 25, 2018
melatonin Acute Radiation Syndrome Oct 22, 2018