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Voxelotor
go.drugbank.com/drugs/DB14975ApprovedVoxelotor is a novel hemoglobin S polymerization inhibitor for the treatment of sickle cell disease. … This is a genetically inherited condition most prevalent in the Middle East, Africa, and certain parts of India.
Categories: Cytochrome P-450 Substrates, Cytochrome P-450 CYP2B6 SubstratesMiransertib
go.drugbank.com/drugs/DB14982InvestigationalMiransertib is under investigation in clinical trial NCT01473095 (Phase 1 Dose Escalation Study of ARQ 092 in Adult Subjects With Advanced Solid Tumors and Recurrent Malignant Lymphoma).
Categories: Heterocyclic Compounds, 1-RingSynonyms: 3-{3-[4-(1-aminocyclobutyl)phenyl]-5-phenyl-3H-imidazo[4,5-b]pyridin-2-yl}pyridin-2-amineCimlanod
go.drugbank.com/drugs/DB14983InvestigationalCimlanod is under investigation in clinical trial NCT02819271 (A First-in-Human Study of the Safety of Single Continuous Intravenous (IV) Infusions of CXL-1427 for up to 48 Hours in Healthy Volunteers)
Casimersen
go.drugbank.com/drugs/DB14984ApprovedInvestigationala six-membered morpholino ring, and the phosphodiester links between nucleotides are replaced with a phosphorodiamidate linkage. … Duchenne muscular dystrophy (DMD) is an X-linked recessive allelic disorder characterized by a lack of functional dystrophin protein, which leads to progressive impairment of ambulatory, pulmonary, and
Categories: Heterocyclic Compounds, 1-Ring, Cytochrome P-450 CYP2C19 InhibitorsEnarodustat
go.drugbank.com/drugs/DB14985InvestigationalEnarodustat is under investigation in clinical trial NCT02581124 (Study to Evaluate Effect of Lapatinib on Pharmacokinetics of JTZ-951 in Subjects With End-stage Renal Disease).
Categories: Heterocyclic Compounds, 1-RingLinrodostat
go.drugbank.com/drugs/DB14986InvestigationalLinrodostat is under investigation in clinical trial NCT03247283 (Pharmacokinetics and Metabolism Study in Healthy Male Participants).
Categories: Heterocyclic Compounds, 2-RingNP-G2-044
go.drugbank.com/drugs/DB14995InvestigationalNP-G2-044 is under investigation in clinical trial NCT03199586 (Phase 1 Clinical Trial of Metastasis Inhibitor NP-G2-044 in Patients With Advanced or Metastatic Solid Tumors (Including Lymphoma)).
Categories: Heterocyclic Compounds, 1-Ring, Heterocyclic Compounds, 2-RingFitusiran
go.drugbank.com/drugs/DB15002ApprovedInvestigationalFitusiran is an antithrombin-directed double-stranded small interfering ribonucleic acid (siRNA) that is covalently linked to a ligand containing a triantennary N-acetylgalactosamine (GalNAc) moiety. … [L52860] Fitusiran was first approved by the FDA on March 28, 2025, as routine prophylaxis therapy to prevent or reduce the frequency of bleeding episodes associated with hemophilia A or B.
Synonyms: SMALL INTERFERING RNA (SIRNA) INHIBITING ANTITHROMBIN LIVER PRODUCTION: DUPLEX OF ((2S,4R)-1-(1-((2-ACETAMIDO-2-DEOXY-.BETA.-D-GALACTOPYRANOSYL)OXY)-16,16-BIS((3-((3-(5-((2-ACETAMIDO-2-DEOXY-.BETA., -D-GALACTOPYRANOSYL)OXY)PENTANAMIDO)PROPYL)AMINO)-3-OXOPRAdrulipase alfa
go.drugbank.com/drugs/DB15004InvestigationalMS-1819 is under investigation in clinical trial NCT03481803 (A Phase IIa Study With Escalating Dose of MS1819-SD).
Edasalonexent
go.drugbank.com/drugs/DB15010InvestigationalEdasalonexent is under investigation in clinical trial NCT01511900 (A Multiple Ascending Dose Study of CAT-1004 in Patients With Type 2 Diabetes).