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CHGN111
go.drugbank.com/drugs/DB06376ExperimentalNumerous parameters of mitochondrial function are altered when the activity of CLK-1 is reduced, which results in a decrease of ROS at critical cellular sites, as well as in a decrease of systemic oxidative … CHGN111 is an inhibitor of the mitochondrial enzyme CLK-1 which is a demethoxyubiquinone hydroxylase.
Vonicog alfa
go.drugbank.com/drugs/DB12872ApprovedInvestigational[A264389] Vonicog alfa works to reduce bleeding events and bleeding times in patients with vWD, who have deficiencies in VWF.[A264389] … [A264444] Used to treat von Willebrand Disease (vWD), it mimics the biological actions of endogenous VWF,[L51274] which is a critical plasma glycoprotein involved in platelet adhesion and aggregation.
Difluocortolone
go.drugbank.com/drugs/DB09095ApprovedWithdrawnIt is commonly used in dermatology for the reduction of inflammation and itching. It was submitted to the FDA in July 1984 by the pharmaceutical company Schering AG.[L1082]
Pegcetacoplan
go.drugbank.com/drugs/DB16694ApprovedInvestigationalPegcetacoplan is a complement inhibitor indicated in the treatment of paroxysmal nocturnal hemoglobinuria (PNH). … [L34095] In February 2023, pegcetacoplan for intravitreal use was approved by the FDA for the treatment of geographic atrophy (GA) secondary to age-related macular degeneration.[L45354]
Ethionamide
go.drugbank.com/drugs/DB00609ApprovedInvestigationalWithdrawnA second-line antitubercular agent that inhibits mycolic acid synthesis. It also may be used for treatment of leprosy. (From Smith and Reynard, Textbook of Pharmacology, 1992, p868)
Etripamil
go.drugbank.com/drugs/DB12605ApprovedInvestigational) to sinus rhythm in adults. … [A275243, A275248] The nasal formulation of etripamil was first approved by the FDA on December 12, 2025 for the conversion of acute symptomatic episodes of paroxysmal supraventricular tachycardia (PSVT
Romiplostim
go.drugbank.com/drugs/DB05332ApprovedInvestigationalRomiplostim is produced by recombinant DNA technology in Escherichia coli. FDA approved on August 22, 2008. … Each subunit consists of an IgG1 Fc carrier domain that is covalently attached to a polypeptide sequence that contains two binding domains to interact with thrombopoietin receptor c-Mpl.
Imetelstat
go.drugbank.com/drugs/DB14909ApprovedInvestigational[A187838] Imetelstat received FDA approval in June 2024 for use in certain patients with MDS who have transfusion-dependent anemia and are unable to use ESAs.[L50883,L50888] … Imetelstat is a first-in-class 18-amino acid oligonucleotide that directly and competitively inhibits human telomerase.
Quinidine barbiturate
go.drugbank.com/drugs/DB01346ApprovedA papulopurpuric eruption in a patient (without thrombopenia) can be developed who is taking quinidine phenylethyl barbiturate intermittently and at reintroduction.(PMID: 9739909) … The administration of quinidine derivatives helps to observe various skin and mucosal reactions.
Isosorbide
go.drugbank.com/drugs/DB09401ApprovedInvestigationalIsosorbide was previously available in an oral formulation for the reduction of intraocular pressure. It was approved by the FDA in 1980, but has since been discontinued. … Currently, isosorbide is an organic nitrate currently available in the [isosorbide mononitrate] and [isosorbide dinitrate] forms, and is used for the prevention of angina.