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Vosoritide
go.drugbank.com/drugs/DB11928ApprovedInvestigational[A242273] While the remarkably short half-life of endogenous CNP - 2 to 3 minutes due to its rapid degradation by endopeptidases - makes it ineffective as a therapeutic intervention,[A242273] the development … [L39229] It was approved for use under the brand name Voxzogo (BioMarin Pharmaceutical Inc.) in the EU in August 2021 and the US in November 2021,[L39245,L39241] becoming the first pharmacological intervention
Piperaquine
go.drugbank.com/drugs/DB13941ApprovedInvestigationalEurartesim was first authorized for market by the European Medicines Agency in October 2011.
Ruxolitinib
go.drugbank.com/drugs/DB08877ApprovedInvestigational[A229938] Ruxolitinib was first approved for the treatment of adult patients with myelofibrosis by the FDA in 2011, followed by EMA's approval in 2012. … By inhibiting JAK1 and JAK2, ruxolitinib works to block the dysregulated cell signalling pathways and prevents abnormal blood cell proliferation.
Bilastine
go.drugbank.com/drugs/DB11591ApprovedBilastine is a novel new-generation antihistamine that is highly selective for the H1 histamine receptor, has a rapid onset and prolonged duration of action.
Technetium Tc-99m sestamibi
go.drugbank.com/drugs/DB09161ApprovedInvestigationalFollowing intravenous injection of the drug, Technetium Tc-99m sestamibi is taken up by the myocardium, parathyroid, and/or breast tissue. … The mechanism by which sestamibi localizes to these tissues has not been established.
Mavorixafor
go.drugbank.com/drugs/DB05501ApprovedInvestigationalby a reduced number of mature neutrophils and lymphocytes. … [L50647] WHIM syndrome is caused by mutations in the _CXCR4_ gene, which leads to overactivation of CXCR4 signalling pathways.[A263652] Mavorixafor prevents the activation of CXCR4.
Niclosamide
go.drugbank.com/drugs/DB06803ApprovedInvestigationalVet approvedNiclosamide, once marketed in the US under the brand name Niclocide, was voluntarily withdrawn from market by Bayer in 1996.[L11860]
Brivaracetam
go.drugbank.com/drugs/DB05541ApprovedInvestigationalIt is available under the brand name Briviact made by UCB. Briviact received FDA approval on February 19, 2016 [L760].
Vamorolone
go.drugbank.com/drugs/DB15114ApprovedInvestigationalVamorolone is a novel and fully synthetic glucocorticoid developed by Santhera Pharmaceuticals. … [L48676] Vamorolone was approved by the FDA in October 2023 for the management of DMD in patients ≥2 years of age.
Tezacaftor
go.drugbank.com/drugs/DB11712ApprovedInvestigational[L1595] It was developed by Vertex Pharmaceuticals and FDA approved in combination with [ivacaftor] to manage cystic fibrosis.[L6814] This drug was approved by the FDA on February 12, 2018. … [L4894] Cystic Fibrosis is an autosomal recessive disorder caused by one of several different mutations in the gene for the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) protein, an ion