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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.
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).