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Clofazimine
go.drugbank.com/drugs/DB00845ApprovedInvestigationalWithdrawnClofazimine is a highly lipophilic antimicrobial riminophenazine dye used in combination with other agents, such as dapsone, for the treatment of leprosy. It was originally described in 1957 and was the prototypical riminophenazine dye -...
Lumacaftor
go.drugbank.com/drugs/DB09280ApprovedInvestigationalLumacaftor is a drug used in combination with DB08820 as the fixed dose combination product Orkambi for the management of Cystic Fibrosis (CF) in patients aged 6 years and older. Cystic Fibrosis is an autosomal recessive disorder caused ...
Fluoxetine
go.drugbank.com/drugs/DB00472ApprovedInvestigationalVet approvedFluoxetine is a 2nd generation antidepressant categorized as a selective serotonin reuptake inhibitor (SSRI). It gained FDA approval in 1987 and although it was initially intended for the treatment of depression, today it is commonly pre...
Gemcabene
go.drugbank.com/drugs/DB05123InvestigationalGemcabene is a new drug that lowers low-density lipoprotein cholesterol (LDL-C), decreases triglycerides, and raises high-density lipoprotein cholesterol (HDL-C).
Avalglucosidase alfa
go.drugbank.com/drugs/DB16099ApprovedInvestigationalPompe disease is characterized by progressive muscle weakness and loss of motor function, including respiratory muscle weakness, which leads to premature death and debilitating effects on people’s lives … [L35160] Late-onset Pompe disease is associated with a range of debilitating physical symptoms, such as progressive muscle weakness, including respiratory muscle weakness, and loss of motor function.
Desmopressin
go.drugbank.com/drugs/DB00035ApprovedInvestigationalOn January 15, 2026, the FDA approved a new low-dose sublingual film formulation specifically for the treatment of nocturia in patients at high risk for fluid overload, utilizing a specialized delivery … Despite its versatility, desmopressin carries a Boxed Warning regarding the risk of hyponatremia (critically low blood sodium levels), which can lead to seizures and death if fluid intake is not properly
LB-100
go.drugbank.com/drugs/DB15412InvestigationalLB-100 is under investigation in clinical trial NCT03886662 (A Study of LB-100 in Patients With Low or Intermediate-1 Risk Myelodysplastic Syndromes (MDS)).
Omidubicel
go.drugbank.com/drugs/DB17132ApprovedInvestigationalIn addition, the fact that 39% of the study's cohort were non-White patients highlights the importance of this type of treatment in key demographics with low donor availability. … In this group of patients, umbilical cord blood can be used as an alternative cell source since there is a lower risk of graft failure or graft-versus-host disease, even with low-grade HLA-matching.
Trofinetide
go.drugbank.com/drugs/DB06045ApprovedTrofinetide is a novel synthetic analog of glypromate, also known as glycine–proline–glutamate (GPE), a naturally occurring protein in the brain and the N-terminal tripeptide of insulin-like growth factor 1 (IGF-1). Trofinetide was appro...
Acipimox
go.drugbank.com/drugs/DB09055ApprovedInvestigationalIt is used in low doses and may have less marked adverse effects, although it is unclear whether the recommended dose is as effective as are standard doses of nicotinic acid.