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Iptacopan
go.drugbank.com/drugs/DB16200ApprovedInvestigationalIptacopan is a small-molecule factor B inhibitor previously investigated as a potential treatment for the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) by inhibiting the complement factor … [A262566] Factor B is a positive regulator of the alternative complement pathway, where it activates C3 convertase and subsequently C5 convertase.
Categories: Compounds used in a research, industrial, or household settingOdevixibat
go.drugbank.com/drugs/DB16261ApprovedInvestigational[L34803] Previous therapies for PFIC included a bile acid sequestrant such as [ursodeoxycholic acid].
Orelabrutinib
go.drugbank.com/drugs/DB16272InvestigationalOrelabrutinib is under investigation in clinical trial NCT04305197 (A Study of ICP-022 in Patients With Systemic Lupus Erythematosus (SLE)).
Veliflapon
go.drugbank.com/drugs/DB16346InvestigationalVeliflapon is under investigation in clinical trial NCT00353067 (Veliflapon (Dg-031)to Prevent Heart Attacks or Stroke in Patients With a History of Heart Attack or Unstable Angina).
Deucravacitinib
go.drugbank.com/drugs/DB16650ApprovedInvestigationalDeucravacitinib is a novel oral selective tyrosine kinase 2 (TYK2) inhibitor. … [A246938, A246943] This selectivity towards TYK2 may lead to an improved safety profile of deucravacitinib, as nonselective JAK inhibitors are associated with a range of adverse effects such as altered
L-Acetylleucine
go.drugbank.com/drugs/DB16956ApprovedInvestigationalLevacetylleucine (L-acetylleucine) is a modified acetylated derivative of the amino acid [leucine]. The racemic (i.e. DL-) form has been available in France since 1957 for the treatment of vertigo. … , after which animal studies showed that the L-enantiomer (i.e. levacetylleucine) has potential clinical benefits compared to the racemic mixture.
Synonyms: Acetyl-l-leucine, Acetylleucine, l-Nutlin-3
go.drugbank.com/drugs/DB17039ExperimentalNutlin-3 is a small molecule inhibitor that targets p53-Mdm2 interaction.[A253057]
Vorasidenib
go.drugbank.com/drugs/DB17097ApprovedInvestigationalVorasidenib is a first-in-class dual isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor. … [A264209] Vorasidenib was first approved by the FDA on August 6, 2024, for the treatment of Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation.[L51144]
Acoramidis
go.drugbank.com/drugs/DB17999ApprovedInvestigational[A264768] Although they share a mechanism of action, acoramidis is more selective for TTR and is a more potent stabilizer when compared to tafamidis. … Acoramidis is a small molecule stabilizer of transthyretin (TTR) for use in patients with TTR amyloidosis.