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
lenabasum Dermatomyositis Jul 18, 2018
recombinant adeno-associated viral vector containing a single-stranded transgene encoding a codon-optimized human SMN1 complementary deoxyribonucleic acid under the control of the chicken beta actin promoter Spinal Muscular Atrophy Jul 16, 2018
Recombinant human insulin receptor monoclonal antibody-N-heparan sulfamidase fusion protein Mucopolysaccharidosis Type IIIA, Sanfilippo A Syndrome Jul 12, 2018
lisocabtagene maraleucel Primary mediastinal large B-cell lymphoma Jul 12, 2018
competitive and reversible small molecule inhibitor of the O-linked-B-N-acetylglucosaminidase enzyme Progressive supranuclear palsy Jul 10, 2018
soidum (5-(3-cyanopyrazolo[1,5-a]pyridin-5-yl)-2-methoxypyridin-3-yl)((2,4-difluorophenyl)sulfonyl)amide Idiopathic pulmonary fibrosis (IPF) Jul 09, 2018
3-[5-[(1R)-1-[bis(aziridin-1-yl(phosphoryloxy]ethyl]-2-nitrophenoxy]-N,N-dimethylbenzamide Hepatocellular carcinoma Jul 03, 2018
allogeneic anti-CD19 chimeric antigen receptor (CAR)-T cells Acute lymphoblastic leukemia (ALL) Jul 02, 2018
Recombinant adeno-associated viral vector serotype 9 containing human iduronidase gene Mucopolysaccharidosis type I Jun 27, 2018
Omaveloxolone Friedreich's ataxia Jun 27, 2018
L-cystine bis(N'-methylpiperazide) Cystinuria Jun 27, 2018
Efpegsomatropin Growth hormone deficiency Jun 27, 2018
Deferiprone Neurodegeneration with brain iron accumulation Jun 27, 2018
Codon-optimised human ornithine transcarbamylase mRNA complexed with lipid-based nanoparticles Ornithine transcarbamylase deficiency Jun 27, 2018
Argon Perinatal asphyxia Jun 27, 2018
20-hydroxyecdysone Duchenne muscular dystrophy Jun 27, 2018
(R)-1-(3-(aminomethyl) phenyl)-N-(5-((3-cyanophenyl)(cyclopropylmethylamino)methyl)-2-fluorophenyl)-3-(trifluoromethyl)-1H-pyrazole-5-carboxamide dihydrochloride Hereditary angioedema Jun 27, 2018
2-(tert-butylamino)-4-{[(1R, 3R, 4R)-3-hydroxy-4-methylcyclohexyl] amino} pyrimidine-5-carboxamide Idiopathic pulmonary fibrosis Jun 27, 2018
cannabidiol Autoimmune hepatitis Jun 25, 2018
L-alanoyl-D-glutamate endopeptidase from Bacillus-infected bacteriophages Inhalational anthrax Jun 21, 2018