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Lomitapide
go.drugbank.com/drugs/DB08827Approved[A7367] Lomitapide was first approved by the FDA in December 2012 and the EMA in July 2013 as an adjunct to a low-fat diet and other lipid-lowering treatments.[A275444, A275446] … [A275445, A7367] This makes it a critical therapeutic option for "receptor-negative" patients who have little to no functional LDL receptor activity.
Categories: Cytochrome P-450 CYP3A4 Substrates with a Narrow Therapeutic IndexCrinecerfont
go.drugbank.com/drugs/DB18518ApprovedInvestigational[A264818] Crinecerfont was approved by the FDA in December 2024 as an adjunct to glucocorticoid replacement in patients with CAH.[L51973,L51993] … [A264818] Crinecerfont is a selective antagonist of corticotropin-releasing factor (CRF) type 1 receptor that works to reduce excessive ACTH secretion from the pituitary.
Samidorphan
go.drugbank.com/drugs/DB12543ApprovedInvestigational, and a longer half-life, making it an attractive candidate for oral dosing. … [A235638, A235643] Samidorphan is a novel opioid antagonist structurally related to [naltrexone], with a higher affinity for opioid receptors, more potent μ-opioid receptor antagonism, higher oral bioavailability
Daraxonrasib
go.drugbank.com/drugs/DB22308ApprovedInvestigationalRAS variants rather than a single mutation. … [L64039,L64041] Because it targets a broad range of RAS genotypes, it treats tumors with or without an identified RAS mutation and does not require a companion diagnostic test.
IFx-Hu2.0
go.drugbank.com/drugs/DB18396InvestigationalIt is composed of plasmid DNA encoding the streptococcal membrane protein, Emm55, contained within a cationic polymer.
Synonyms: Immunotherapeutic vaccine consisting of pAc/emm55, a plasmid DNA (pDNA), which codes for a modified immunogenic, streptococcal Emm55 protein, complexed with in vivo-jetPEI® polymer-based reagent linearAG0302-COVID19
go.drugbank.com/drugs/DB16435InvestigationalSponsored by Anges, Inc. in Japan, the AG0302-COVID19 is a DNA-based vaccine that recently completed Phase I/II trials (NCT04527081)[A226813, L30548].
PW2101
go.drugbank.com/drugs/DB06483ExperimentalThe drug was developed by Penwest Pharmaceuticals and in 2005 the FDA issues a Non-Approval letter due to the degree of kinetic variability of PW2101 observed among individuals. … PW2101 is a beta blocker intended for the treatment of hypertension and angina.
Pimozide
go.drugbank.com/drugs/DB01100ApprovedA diphenylbutylpiperidine that is effective as an antipsychotic agent and as an alternative to haloperidol for the suppression of vocal and motor tics in patients with Tourette syndrome.
Categories: Dopamine D2 Receptor Antagonists, Cytochrome P-450 CYP1A2 Substrates with a Narrow Therapeutic IndexRindopepimut
go.drugbank.com/drugs/DB05374InvestigationalThe US FDA granted it Breakthrough Therapy Designation for glioblastoma in Feb 2015. … Rindopepimut (CDX-110) is an injectable peptide cancer vaccine which targets a mutant protein called EGFRvIII present in about 25% to 30% of glioblastoma cases.
Lesinurad
go.drugbank.com/drugs/DB11560ApprovedWithdrawnserum uric acid levels with a xanthine oxidase inhibitor alone. … In August 2017, a combination oral therapy consisting of lesinurad and [DB00437] was FDA-approved under the brand name Duzallo indicated for the treatment of gout-related hyperuricemia in patients with