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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
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
recombinant adeno-associated viral (AAV) vector that contains a bio-engineered capsid (AAV-Spark100) and a codon-optimized expression cassette to drive expression of a secretable form of human acid a-glucosidase (GAA) Pompe Disease Feb 01, 2019
pegcetacoplan Autoimmune hemolytic anemia Feb 01, 2019
N-(4-Fluoro-benzoyl)-L-arginyl-L-arginyl-[L-3-(naphthyl)alanyl]-L-cysteinyl-L-tyrosyl-L-citrullinyl-L-lysyl-D-lysyl-L-prolyl-L-trosyl-L-arginyl-L-ctrullinyl-L-cysteinyl-L-aginine amide,cyclic (4-13)-disulfide Pancreatic cancer Feb 01, 2019
Fully human immunoglobulin G1 monoclonal antibody that targets CD32b Mantle cell lymphoma Jan 29, 2019