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
recombinant lentivirus vector expressing the human phenylalanine hydroxylase (PAH) gene under control of a liver-specific promoter Phenylketonuria Oct 15, 2018
reboxetine Narcolepsy Oct 15, 2018
heterodimeric fusion protein that binds to IL-1 beta consisting of human extracellular domain of IL1 receptor and a mutant Fc fragment of human IgG1 and part of human IL1 receptor accessory protein Schnitzler Syndrome Oct 15, 2018
(2S)-2-{[(2R)-2-[({[3,3-dibutyl-7-(methylthio)-1,1-dioxido-5-phenyl-2,3,4,5-tetrahydro-1,2,5-benzothiadiazepin-8-yl]oxy}acetyl)amino]-2-(4-hydroxyphenyl)acetyl]amino}butanoic acid Alagille syndrome Oct 15, 2018
Humanized IgG-1 antibody targeting human transferrin receptor conjugated to human iduronate-2-sulfatase Mucopolysaccharidosis Type II (MPS II) or Hunter Syndrome Oct 15, 2018
Monoclonal antibody targeting CCL24 Primary sclerosing cholangitis Oct 11, 2018
Lead-212 DOTAMTATE Neuroendocrine tumors Oct 11, 2018
Glucagon-Like Peptide 2 fused to hyFc Short bowel syndrome Oct 11, 2018
olaparib Pancreatic cancer Oct 11, 2018
N-[5-(6-fluoro-8-{[4-(2-hydroxypropan-2-yl)piperidin-1-yl]methyl}-2-morpholinoquinazolin-4-yl)-2-methoxypyridin-3-yl]methanesulfonamide Treatment Follicular Lymphoma Oct 10, 2018
Indolino-benzodiazepine compound (sulfonated DGN549-C) conjugated to the humanized anti-CD123 monoclonal antibody (G4723A) Acute myeloid leukemia Oct 10, 2018
bispecific T-cell engager antibody with a single chain Fc moiety that binds to B cell maturation antigen surface receptor and CD3 Multiple myeloma Oct 10, 2018
antibody-drug conjugate comprised of a human aglycosylated anti-CD74 IgG1 antibody (SP7219) and two non-cleavable maytansinoid linker warheads Multiple myeloma Oct 10, 2018
clervonafusp alfa Pompe Disease Oct 04, 2018
clenbuterol Amyotrophic lateral sclerosis Oct 02, 2018
3-(3-(3,5-Dimethyl-1H-pyrazol-4- yl)propoxy)-4-fluorobenzoic acid Transthyretin amyloidosis Oct 02, 2018
S-[+]-apomorphine Amyotrophic lateral sclerosis (ALS) Oct 02, 2018
progerinin Hutchinson-Gilford progeria syndrome Oct 02, 2018
poly (acetyl, arginyl) glucosamine Cystic fibrosis Oct 02, 2018
N-Acetyl-Leucine Ataxia Telangiectasia Oct 02, 2018