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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
concizumab Hemophilia B Jul 22, 2019
autologous T-cells genetically modified to express a CD123-specific, hinge-optimized, CD28-costimulatory chimeric receptor and a truncated human epidermal growth factor receptor Acute myeloid leukemia Jul 22, 2019
artesunate Malaria Jul 22, 2019
Aripiprazole N-glycanase 1 (NGLY1) deficiency Jul 22, 2019
(S)-4-methyl-5-((4-((2-(methylamino)-6-(2,2,2-trifluoroethyl)thieno[2,3-d]pyrimidin-4-yl)amino)piperidin-1-yl)methyl)-1-(2-(4-(methylsulfonyl)piperazin-1-yl)propyl)-1H-indole-2-carbonitrile Acute myeloid leukemia Jul 22, 2019
(R)-N-(1-(1-(1H-indazol-5-yl)-3-methyl-2,4-dioxo-1,3,8-triazaspiro[4,5]decan-8-yl)-3-methyl-1-oxobutan-2-yl)-2-fluoro-5-(trifluoromethyl)benzamide Idiopathic pulmonary fibrosis Jul 18, 2019
PEGylated 24-amino acid peptide inhibitor of Interleukin-2, Interleukin-9 and Interleukin-15 Cutaneous T-cell lymphoma Jul 15, 2019
Double stranded oligomer RNA interference-based liver targeted therapeutic directed against the ANGPTL3 gene Homozygous familial hypercholesterolemia Jul 15, 2019
Zamicastat Pulmonary arterial hypertension (PAH) (pulmonary hypertension WHO Group 1) Jul 15, 2019
afatinib Pancreatic cancer Jul 09, 2019
bispecific T-cell engager (BiTE) antibody targeting CD33 and CD3 Acute Myeloid Leukemia Jul 01, 2019
Regorafenib Glioblastoma multiforme Jul 01, 2019
non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) containing the short hairpin RNA (shRNA) sequence against human SOD1 under the H1 promoter Amyotrophic Lateral Sclerosis Jul 01, 2019
apilimod dimesylate Follicular lymphoma Jul 01, 2019
Ornithine transcarbamylase messenger RNA Ornithine Transcarbamylase Deficiency Jun 26, 2019
mometasone furoate Eosinophilic esophagitis Jun 26, 2019
Kallikrein 7 and elastase 2 inhibitor Netherton Syndrome Jun 25, 2019
Human monoclonal IgG2 antibody against tissue factor pathway inhibitor Hemophilia B Jun 25, 2019
5-iodo-2-pyrimidinone-2�deoxyribose Glioblastoma Jun 25, 2019
gallium citrate Lung infections in patients with cystic fibrosis Jun 24, 2019