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Nylidrin
go.drugbank.com/drugs/DB06152ApprovedWithdrawnNylidrin is a peripheral vasodilator. … FDA has considered nylidrin as "lacking substantial evidence of effectiveness" in cerebral ischemia, cerebral arteriosclerosis, and other cerebral circulatory insufficiencies.
Zilganersen
go.drugbank.com/drugs/DB18161ApprovedInvestigational[L64096] Alexander disease is caused by variants in the *GFAP* gene that lead to overproduction and toxic accumulation of GFAP in astrocytes, and dysfunction in astrocytes can over time damage neurons … [L64096] The FDA approved zilganersen on September 3, 2026 for the treatment of Alexander disease in pediatric and adult patients, making it the first and only disease-modifying treatment for the condition
Danicopan
go.drugbank.com/drugs/DB15401ApprovedInvestigational[A263506] It was subsequently approved by the FDA in March 2024.[L50386] … [L50381] It was first approved in January 2024 in Japan for patients with PNH,[A263506,L50416] shortly after which the EMA adopted a positive opinion and recommended granting it marketing authorization
Synonyms: (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidin-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridin-2-yl)-4-fluoropyrrolidine-2-carboxamide, (2S,4R)-1-(2-(3-acetyl-5-(2-methylpyrimidine-5-yl)-1H-indazol-1-yl)acetyl)-N-(6-bromopyridine-2-yl)-4-fluoropyrrolidine-2-carboxamideTividenofusp alfa
go.drugbank.com/drugs/DB17628ApprovedInvestigationalTividenofusp alfa is a first-in-class, brain-penetrant enzyme replacement therapy (ERT) indicated for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome [L56097, L56099 … Hunter syndrome is a rare, X-linked lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase (IDS), which leads to the toxic accumulation of glycosaminoglycans (GAGs) throughout
Aducanumab
go.drugbank.com/drugs/DB12274ApprovedInvestigationalin amyloid-β plaques. … [L34420] Biogen enrolled patients in phase 3 clinical trials in 2015 but increased the size of the trials from 1350 patients to 1650 patients to maintain statistical power in the face of a high standard
Fluoroestradiol F-18
go.drugbank.com/drugs/DB15690ApprovedInvestigational[L14117] It is expected to be available in late 2020/early 2021.[L14117] … [L14096] The ability to image ER-positive tumors _in vivo_ is advantageous in that, while helping to visualize tumor progression/regression, it may also be used to assess for heterogeneity in ER expression
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.
Disulfiram
go.drugbank.com/drugs/DB00822ApprovedInvestigationalA carbamate derivative used as an alcohol deterrent. It is a relatively nontoxic substance when administered alone, but markedly alters the intermediary metabolism of alcohol. … It acts by inhibiting aldehyde dehydrogenase.
Synonyms: N,N,N',N'-tetraethylthiuram disulfide, 1,1'-dithiobis(N,N-diethylthioformamide)Categories: Drugs Used in Alcohol Dependence, Drugs Used in Addictive DisordersFluticasone furoate
go.drugbank.com/drugs/DB08906ApprovedInvestigationalFluticasone furoate is a synthetic glucocorticoid available as an inhaler and nasal spray for various inflammatory indications[FDA Label][F4364]. Fluticasone furoate was first approved in 2007[L5965].
Efgartigimod alfa
go.drugbank.com/drugs/DB15270ApprovedInvestigational[A243759,A243784] Efgartigimod alfa is a first-in-class[L39501] antagonist of the neonatal Fc receptor (FcRn) used in the treatment of MG. … and a reduction in electrical nerve impulses.