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
verdiperstat Multiple system atrophy (MSA) Feb 15, 2019
recombinant adeno-associated viral vector serotype S3 containing codon-optimized expression cassette encoding human coagulation factor IX variant Hemophilia B Feb 15, 2019
A small molecule inhibitor of the pro-survival protein Myeloid Cell Leukemia sequence 1 (MCL 1) Acute Myeloid Leukemia Feb 15, 2019
2-[(2S)-1-azabicyclo[2.2.2]oct-2-yl]-6-(3-methyl-1H-pyrazol-4-yl)thieno[3,2-d]pyrimidin-4(3H)-one hemihydrate Esophageal cancer Feb 11, 2019
2,4-dinitrophenol Huntington disease Feb 11, 2019
rose bengal disodium Ocular melanoma Feb 11, 2019
Extracts of herbs Scutellaria baicalensis Geori, Glycyrrhiza uralensis Fisch, Paeonia lactiflora Pall, and Ziziphus jujuba Mill Pancreatic cancer Feb 11, 2019
etoposide toniribate Biliary tract cancer Feb 11, 2019
budesonide Primary biliary cholangitis Feb 11, 2019
Bispecific T-cell engager (BiTE) containing two single-chain variable fragment (scFv) domains directed against the B cell maturation antigen (BCMA) surface receptor on tumor cells and the cluster of differentiation 3 (CD3) receptor on T-cells Multiple myeloma Feb 08, 2019
itolizumab Prevention of acute graft versus host disease Feb 05, 2019
rozanolixizumab Myasthenia gravis Feb 01, 2019
ketamine Complex Regional Pain Syndrome Feb 01, 2019
itolizumab Acute graft versus host disease Feb 01, 2019
efgartigimod Primary Immune Thrombocytopenia (ITP) Feb 01, 2019
budesonide Autoimmune Hepatitis Feb 01, 2019
benralizumab Hypereosinophilic syndrome Feb 01, 2019
autologous T-cells genetically engineered via lentiviral vector technology to express a synthetic receptor directed against B-cell maturation antigen (BCMA) protein target Multiple myeloma (MM) Feb 01, 2019
autologous genetically engineered T cells that express a single-domain antibody that recognizes human mesothelin fused to the cluster of differentiation 3e subunit of the T cell receptor Mesothelioma Feb 01, 2019
(E)-3-(6-Bromo-pyridin-2-yl)-2-cyano-N-((S)-1-phenyl-ethyl)-acryl amide Glioblastoma Feb 01, 2019