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
4-{[4-Amino-3-(4-cyclohexylpiperazin-1-yl)-9,10-dioxo-9,10-dihydroanthracen-1-yl]amino}benzoic acid Pancreatic cancer Apr 11, 2018
human glial restricted progenitor cells and their progeny Transverse myelitis Apr 11, 2018
C1 esterase inhibitor, human Antibody mediated rejection (AMR) of solid organ transplants Apr 05, 2018
humanized anti-CD40LG IgG1, kappa monoclonal antibody with heavy chain C220S/C226S/C229S/P238S substitutions to attenuate effector function Amyotrophic Lateral Sclerosis (ALS) Apr 04, 2018
6-(4-(4-(2,3-Dicholorophenyl) piperazin-1-yl) butoxy)-2H-benzo [b] [1,4] oxazin-3(4H)-one hydrochloride Idiopathic Pulmonary Fibrosis Apr 04, 2018
tenalisib Cutaneous T-Cell Lymphoma Apr 04, 2018
pomalidomide Kaposi sarcoma Apr 04, 2018
tinostamustine hydrochloride Multiple myeloma Apr 02, 2018
anti-EGFRvIII x anti-CD3 bispecific T cell engager antibody construct Glioblastoma Apr 02, 2018
Brevenal Cystic fibrosis Apr 02, 2018
Autologous Activated T Lymphocytes (ATL) Hepatocellular carcinoma (HCC) Mar 28, 2018
(3R)-3-Methyl-6-[2-({5-methyl-2-[4-(trifluoromethyl)phenyl]-1H-imidazol-1-yl}methyl)phenoxy]hexanoic acid hemisulfate Duchenne muscular dystrophy Mar 28, 2018
Nanoparticle suspension containing biosynthetic codon-optimized human ornithine transcarbamylase messenger RNA Ornithine transcarbamylase (OTC) deficiency Mar 28, 2018
Humanized IgG4 monoclonal antibody against total complement component 1, subcomponent s Immune thrombocytopenia Mar 28, 2018
dexamethasone Multiple myeloma (MM) Mar 26, 2018
Small molecule allosteric inhibitor of the phosphodiesterase-4D enzyme Fragile X Syndrome Mar 26, 2018
Allogeneic mesenchymoangioblast-derived mesenchymal stem cells Acute graft versus host disease Mar 26, 2018
7-(4-fluoro-2-methoxyphenyl)-6-methyl-N-(1-(piperidin-4-yl)-1H-pyrazol-4-yl) thieno [3,2-d]pyrimidin-2-amine semi-fumarate Acute myeloid leukemia Mar 26, 2018
fully human anti-proMyostatin monoclonal antibody of the IgG4/lambda isotype that binds to human pro- and latent myostatin with high affinity Spinal muscular atrophy Mar 22, 2018
elamipretide Barth syndrome Mar 22, 2018