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CXD101
go.drugbank.com/drugs/DB16182InvestigationalCXD101 is under investigation in clinical trial NCT03873025 (A Study of CXD101 in Combination With Pembrolizumab for Relapsed or Refractory Diffuse Large B-cell Lymphoma).
Eplontersen
go.drugbank.com/drugs/DB16199ApprovedInvestigationalIt was previously investigated as a potential treatment for hereditary transthyretin-mediated amyloidosis. … [A262985] As eplontersen targets both the WT and mutant TTR, it poses tremendous promises as a treatment.
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 settingBepirovirsen
go.drugbank.com/drugs/DB16202InvestigationalIONIS-HBVRx is under investigation in clinical trial NCT04544956 (A Mechanistic Study of GSK3228836 With Fine Needle Aspiration (FNA) in Participants With Chronic Hepatitis B).
Ivuxolimab
go.drugbank.com/drugs/DB16208InvestigationalIvuxolimab is under investigation in clinical trial NCT02554812 (A Study of Avelumab in Combination With Other Cancer Immunotherapies in Advanced Malignancies (JAVELIN Medley)).
Leniolisib
go.drugbank.com/drugs/DB16217ApprovedInvestigationalLeniolisib is a potent and selective inhibitor of phosphoinositide 3-kinase δ (PI3Kδ). … [L45758] APDS is a primary immunodeficiency caused by mutations in genes encoding the PI3Kδ, thereby increasing the activity of PI3Kδ, causing immune dysfunction, and elevating susceptibility to infections
Maralixibat
go.drugbank.com/drugs/DB16226ApprovedInvestigational[A239249] No clinical trials have been performed to assess the efficacy of these treatments for cholestatic pruritus and treatments were given based on a prescriber's clinical experience.
Apinocaltamide
go.drugbank.com/drugs/DB16241InvestigationalApinocaltamide is under investigation in clinical trial NCT03239691 (A Study to Evaluate the Effect of ACT-709478 in Photosensitive Epilepsy Patients).
MK-0773
go.drugbank.com/drugs/DB16244InvestigationalMK-0773 is under investigation in clinical trial NCT00529659 (A Study of the Safety and Efficacy of MK-0773 in Women With Sarcopenia (Loss of Muscle Mass)(mk-0773-005)).
Odevixibat
go.drugbank.com/drugs/DB16261ApprovedInvestigational[L34803] Previous therapies for PFIC included a bile acid sequestrant such as [ursodeoxycholic acid].