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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.
Vanzacaftor
go.drugbank.com/drugs/DB18373ApprovedInvestigational], a CFTR potentiator, for the treatment of patients with responsive CFTR mutations. … Vanzacaftor is a small molecule cystic fibrosis transmembrane conductance regulator (CFTR) corrector.
Taletrectinib
go.drugbank.com/drugs/DB18711ApprovedInvestigational[L53308] Gene fusions involving _ROS1_, a proto-oncogene, have been identified as oncogenic drivers in 1-2% of patients with non-small cell lung cancer (NSCLC), in addition to other cancers at variable
Enmetazobactam
go.drugbank.com/drugs/DB18716ApprovedInvestigationalEnmetazobactam is a penicillanic acid sulfone extended-spectrum beta (β)-lactamase (ESBL) inhibitor.
Sonrotoclax
go.drugbank.com/drugs/DB18753ApprovedInvestigational[L56193,A275521] Sonrotoclax was rationally designed as a BH3 mimetic with a shallow and broad interaction with the P2 pocket of BCL-2, achieved through a novel 7-azaspiro[3.5]nonane linker; this binding … Sonrotoclax is a selective, potent, small-molecule inhibitor of BCL-2, an antiapoptotic protein overexpressed in B-cell malignancies.
Ompenaclid
go.drugbank.com/drugs/DB18819InvestigationalRGX-202 is a recombinant AAV8 that contains a vector genome encoding a miniaturized dystrophin protein (microdystrophin).
Elinzanetant
go.drugbank.com/drugs/DB18968ApprovedInvestigationalElinzanetant is a non-hormonal, selective, dual neurokinin 1 (NK-1) and 3 (NK-3) receptor antagonist.
Florquinitau (18F)
go.drugbank.com/drugs/DB19215ApprovedFlorquinitau F 18 is a radioactive diagnostic drug used as a fluorine-18–labeled positron emission tomography (PET) tracer. … [L64052] It binds to aggregated tau protein, which in Alzheimer's disease forms the neurofibrillary tangles (NFTs) that are one of the two components required for a neuropathological diagnosis of the disease
Bleximenib
go.drugbank.com/drugs/DB19328InvestigationalBleximenib is under investigation in clinical trials NCT04811560 (A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia) and NCT05453903 (A Study of Bleximenib in Combination With Acute Myeloid