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Dichloroacetic acid
go.drugbank.com/drugs/DB08809ApprovedInvestigationalWithdrawnDCA was approved for use in Canada in 1989 (as a topical formulation for treatment of warts and for cauterization and removal of a wide variety of skin and tissue lesions), but was cancelled post market … Salts of DCA are used as drugs since they inhibit the enzyme pyruvate dehydrogenase kinase.
Tefidsogene civaparvovec
go.drugbank.com/drugs/DB16789InvestigationalIt consists of an adeno-associated virus (AAV) type 2/6 vector and a zinc finger nuclease (ZFN) system that inserts a correct copy of the Iduronate 2-Sulfatase (IDS) gene into the albumin locus in hepatocytes … Tefidsogene civaparvovec, formerly known as SB-913, is an investigational therapy for genome editing.
Synonyms: EXONS 2-9 FROM HIDS AND A SPLICE ACCEPTOR SITE (SA), A RECOMBINANT NON-REPLICATING ADENO-ASSOCIATED VIRUS TYPE 2/6 (RAAV REP2-CAP6) VECTOR, WHICH CONTAINS A PROMOTER-LESS HUMAN IDURONATE 2-SULFATASE (HIDS) TRANSGENE CASSETTE, ENCODING PARTS OF EXON 1 PLUSCIGB 300
go.drugbank.com/drugs/DB16464InvestigationalCIGB-300, is under investigation in clinical trials for its potential to treat various conditions, including NCT01639638 (recurrent and nonrecurrent condyloma) and NCT01639625 (squamous cell carcinoma and adenocarcinoma of the cervix)
Oftasceine
go.drugbank.com/drugs/DB11184ApprovedOftasceine is also referred to as Fluorexon. It is used in ophthalmic solutions as a staining agent when fitting soft and hard lenses. It is a fluorescent dye or luminescent agent.
Drotrecogin alfa
go.drugbank.com/drugs/DB00055ApprovedWithdrawnIt is a glycoprotein of approximately 55 kilodalton molecular weight, consisting of a heavy chain and a light chain linked by a disulfide bond. … Drotrecogin alfa was withdrawn from the market after a major study indicated that it was not effective in improving outcomes in patients with sepsis.
Idebenone
go.drugbank.com/drugs/DB09081ApprovedInvestigationalIdebenone is a synthetic analogue of ubiquinone (also known as Coenzyme Q10), a vital cell antioxidant and essential component of the Electron Transport Chain (ETC). … LHON is a mitochondrially inherited degeneration of retinal ganglion cells, resulting in acute central vision loss.
Categories: Compounds used in a research, industrial, or household settingSynonyms: 2-(10-hydroxydecyl)-5,6-dimethoxy-3-methyl-p-benzoquinone, 6-(10-hydroxydecyl)-2,3-dimethoxy-5-methyl-p-benzoquinoneOmacetaxine mepesuccinate
go.drugbank.com/drugs/DB04865ApprovedInvestigationalWithdrawnOmacetaxine mepesuccinate (formerly known as HHT or Homoharringtonine), is a cephalotaxine ester and protein synthesis inhibitor with established clinical activity as a single agent in hematological malignancies … Omacetaxine mepesuccinate is synthesized from cephalotaxine, which is an extract from the leaves of the plant, Cephalotaxus species.
Synonyms: 1-[(1S,3aR,14bS)-2-Methoxy-1,5,6,8,9,14b-hexahydro-4H-cyclopenta[a][1,3]dioxolo[4,5-H]pyrrolo[2,1-b][3]benzazepin-1-yl] 4-methyl (2R)-2-hydroxy-2-(4-hydroxy-4-methylpentyl)butanedioateAtaluren
go.drugbank.com/drugs/DB05016ApprovedWithdrawnAtaluren is a novel, orally administered drug that targets nonsense mutations. … More specifically, ataluren is used in the small group of patients whose disease is caused by a specific genetic defect (called a ‘nonsense mutation’) in the dystrophin gene.
Macitentan
go.drugbank.com/drugs/DB08932ApprovedInvestigationalMacitentan is a dual endothelin receptor antagonist used in the treatment of pulmonary arterial hypertension (PAH).[L35890] It was first approved by the FDA in 2013. … A combination product (Opsynvi) comprising macitentan and [tadalafil] was approved in Canada in October 2021 for the treatment of PAH.[L39105] It was subsequently approved by the FDA in March 2024.
Categories: Endothelin A Receptor Antagonists, Cytochrome P-450 Substrates