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
Dexpramipexole Hypereosinophilic syndrome (HES) Apr 22, 2019
Autologous enriched T-cells genetically modified with a retroviral vector to express two chimeric antigen receptors (CARs) targeting cluster of differentiation (CD) 19 and CD 22. Acute Lymphoblastic Leukemia Apr 17, 2019
adeno associated viral vector containing Padua derivative of the human coagulation factor IX cDNA Hemophilia B Apr 17, 2019
a long-acting interleukin-11 analog comprised of 178 amino acids covalently modified wiht a 40-kDa branched maleimide-polyethylene glycol at the added cysteine residue following the carboxy-terminal amino acid Acute radiation syndrome Apr 17, 2019
6-[18F] fluoro-L-m-tyrosine Diagnostic for the management of Aromatic L-Amino Acid Decarboxylase Deficiency Apr 17, 2019
a non-replicating, recombinant adeno-associated virus serotype 9 (AAV9) containing the human Methyl CpG Binding Protein 2B (MECP2B) cDNA under the control of a segment of the murine MECP2 promoter. Rett Syndrome (RTT) Apr 17, 2019
cromolyn solution Idiopathic pulmonary fibrosis Apr 11, 2019
umbralisib Nodal marginal zone lymphoma Apr 11, 2019
umbralisib Extranodal marginal zone lymphoma Apr 11, 2019
umbralisib Splenic marginal zone lymphoma Apr 11, 2019
pamrevlumab Duchenne Muscular Dystrophy Apr 11, 2019
autologous chimeric antigen receptor (CAR) T cells targeting intercellular adhesion molecule-1 (ICAM-1) Anaplastic thyroid cancer, medullary thyroid cancer, and recurrent or metastatic follicular or papillary thyroid cancer Apr 09, 2019
Marzeptacog alfa (activated) Haemophilia B Apr 01, 2019
Codon-optimised human cystic fibrosis transmembrane conductance regulator messenger ribonucleic acid complexed with lipid-based nanoparticles Cystic fibrosis Apr 01, 2019
Adeno-associated viral vector serotype rh10 containing the human cholesterol 24-hydroxylase gene Huntington's disease Apr 01, 2019
4-hydroxy-6-{2-[4-(trifluoromethyl)phenyl]ethyl}pyridazin-3(2H)-one Friedreich’s ataxia Apr 01, 2019
2-[3-(2-chloro-4-{[5-cyclopropyl-3-(2,6-dichlorophenyl)-1,2-oxazol-4-yl]methoxy}phenyl)-3-hydroxyazetidin-1-yl]pyridine-4-carboxylic acid-2-amino-2-(hydroxymethyl)propane-1,3-diol (1/1) Primary sclerosing cholangitis Apr 01, 2019
1-[(3S)-3-{4-amino-3-[(3,5-dimethoxyphenyl)ethynyl]-1H-pyrazolo[3,4-d]pyrimidin-1-yl}pyrrolidin-1-yl]-2-propen-1-one Biliary tract cancer Apr 01, 2019
alpha tocopherol and ascorbic acid Fragile X syndrome Mar 28, 2019
allogeneic ABCB5-positive limbal stem cells Limbal stem cell deficiency Mar 28, 2019