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.

Become a Partner.

Product Indication Date Designated
indralin Prevention of acute radiation syndrome (ARS) Jun 13, 2018
extracellular domain of Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 fused to the Fc region of human IgG1 Ectonucleotide pyrophosphatase/phosphodiesterase 1 deficiency (generalized arterial calcification of infancy and autosomal recessive hypophosphatemic rickets type 2) Jun 11, 2018
Recombinant humanized IgG1 anti-HER2 monoclonal antibody conjugated to Duostatin-5 Gastric cancer Jun 07, 2018
nirogacestat Desmoid tumor (aggressive fibromatosis) Jun 07, 2018
brincidofovir Smallpox Jun 05, 2018
olinciguat Sickle Cell Disease Jun 04, 2018
L-cystine bis(N'-methylpiperazide) Cystinuria Jun 04, 2018
TransCon Parathyroid Hormone (mPEG conjugated parathyroid hormone 1-34) Hypoparathyroidism Jun 04, 2018
apomorphine palmitate Acute Myeloid Leukemia Jun 04, 2018
7-benzyl-4-(2-methylbenzyl)-1,2,6,7,8,9-hexahydroimidazo[1,2-a]pyrido[3,4-e]pyrimidin-5(4H)-one Malignant glioma Jun 04, 2018
Three human monoclonal antibodies against the Ebola virus glycoprotein Ebola virus disease May 25, 2018
Synthetic double-stranded siRNA oligonucleotide targeted against transthyretin mRNA, with six phosphorothioate linkages in the backbone, and nine 2'-fluoro and thirty-five 2'-O-methyl nucleoside residues in the sequence, which is covalently linked via a phosphodiester group to a ligand containing three N-acetylgalactosamine residues Transthyretin-mediated amyloidosis May 25, 2018
Itraconazole Prevention of invasive aspergillosis May 25, 2018
H-Arg-Pro-Lys-Pro-Gln-Gln-Phe-2Thi-Gly-Leu-Met(O2)-NH2-DOTA-225-actinium Glioma May 25, 2018
Glucagon analogue linked to a human immunoglobulin Fc fragment Congenital hyperinsulinism May 25, 2018
Equine immunoglobulin F(ab')2fragments targeting Shiga toxin Prevention of haemolytic uraemic syndrome May 25, 2018
Daratumumab AL amyloidosis May 25, 2018
Bardoxolone methyl Alport syndrome May 25, 2018
Autologous CD4+ and CD8+ T cells expressing a CD19-specific chimeric antigen receptor Follicular lymphoma May 25, 2018
Ambroxol hydrochloride Amyotrophic lateral sclerosis May 25, 2018