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Sepiapterin
go.drugbank.com/drugs/DB16326ApprovedInvestigational[L53548,L53593] Sepiapterin was granted marketing authorization by the European Commission in June 2025 and by the US FDA in July 2025 for the treatment of adults and children with phenylketonuria. … It is a natural precursor of the enzymatic co-factor tetrahydrobiopterin (BH4), which activates PAH and helps reduce blood phenylalanine levels by enhancing the conformational stability of misfolded PAH
Categories: Heterocyclic Compounds, 2-RingSynonyms: L-sepiapterinLaronidase
go.drugbank.com/drugs/DB00090ApprovedInvestigationalThe predicted amino acid sequence of the recombinant form, as well as the nucleotide sequence that encodes it, are identical to a polymorphic form of human a-L-iduronidase. … Laronidase is a glycoprotein with a molecular weight of approximately 83 kD.
Synonyms: alpha-L-Idosiduronase, Human Recombinant alpha-L-iduronidaseProducts: ALDURAZYME ®, ALDURAZYME 100 U/ML IV INFUZYON ICIN KONSANTRE COZELTI, 1 ADETAminosalicylic acid
go.drugbank.com/drugs/DB00233ApprovedInvestigationalThe sodium salt of the drug is better tolerated than the free acid.
Categories: 4-aminosalicylic acidSynonyms: 4-amino-2-hydroxybenzoic acid, 4-aminosalicylateSulopenem etzadroxil
go.drugbank.com/drugs/DB16335ApprovedInvestigationalSulopenem etzadroxil is a prodrug of the penem antibacterial [sulopenem]. Like other beta-lactam antibacterials, it is a time-dependent inhibitor of bacterial cell wall synthesis. … [L51858] It is administered in combination with [probenecid] in order to increase antibiotic exposure.[L51768]
Interferon gamma-1b
go.drugbank.com/drugs/DB00033ApprovedInvestigationalThe sequence displayed is a cDNA sequence which codes for human interferon gamma, as described by Gray et. al. and not specifically interferon gamma 1b. … Production of Actimmune is achieved by fermentation of a genetically engineered Escherichia coli bacterium containing the DNA which encodes for the human protein.
Categories: Cytochrome P-450 CYP1A2 Inhibitors, Cytochrome P-450 Enzyme InhibitorsSynonyms: Interferon gamma 1-bLeniolisib
go.drugbank.com/drugs/DB16217ApprovedInvestigational[L45758] APDS is a primary immunodeficiency caused by mutations in genes encoding the PI3Kδ, thereby increasing the activity of PI3Kδ, causing immune dysfunction, and elevating susceptibility to infections … The FDA approved leniolisib on March 24, 2023, making it the first treatment for activated phosphoinositide 3-kinase delta syndrome (APDS).
Categories: P-glycoprotein substrates, Cytochrome P-450 SubstratesParitaprevir
go.drugbank.com/drugs/DB09297ApprovedInvestigationalAs a newer generation and directly acting HCV antiviral, paritaprevir products have better Sustained Virological Response (SVR) rates, higher barriers to resistance, fewer side effects, and a reduced pill … In a joint recommendation published in 2016, the American Association for the Study of Liver Diseases (AASLD) and the Infectious Diseases Society of America (IDSA) recommend Paritaprevir as a first line
Synonyms: (2R,6S,12Z,13aR,14aR,16aS)-N-(cyclopropanesulfonyl)-6-[(5-methylpyrazine-2-carbonyl)amino]-5,16-dioxo-2-[(phenanthridin-6-yl)oxy]-1,2,3,6,7,8,9,10,11,13a,14,15,16,16a-tetradecahydrocyclopropa[e]pyrrolo, [1,2-a][1,4]diazacyclopentadecine-14a(5H)-carboxamideCategories: Cytochrome P-450 Substrates, P-glycoprotein substratesCholecystokinin
go.drugbank.com/drugs/DB08862ApprovedInvestigationalWithdrawnCholecystokinin ( also known as _CCK or CCK-PZ_) is a peptide hormone of the gastrointestinal system which is responsible for stimulating the digestion of fat and protein. … Cholecystokinin, previously called _pancreozymin_, is synthesized and secreted by enteroendocrine cells in the duodenum (the first portion of the small intestine) and leads to the release of bile and digestive
Olaparib
go.drugbank.com/drugs/DB09074ApprovedInvestigational[A246015] Olaparib is used to treat a number of BRCA-associated tumours, including ovarian cancer, breast cancer, pancreatic cancer, and prostate cancer. … [L41100, L40908, L43792] It was first approved by the FDA and EU in December 2014,[A246020] and by Health Canada in April 2016.[L42820]
Categories: MATE 2-K Inhibitors, P-glycoprotein substrates with a Narrow Therapeutic IndexSynonyms: 4-(3-{[4-(cyclopropylcarbonyl)piperazin-1-yl]carbonyl}-4-fluorobenzyl)phthalazin-1(2H)-oneSofpironium
go.drugbank.com/drugs/DB19325Approved[A264153] Sofpironium is rapidly converted to a less active metabolite, BBI-4010, after exerting its activity, thereby limiting the incidence and/or severity of systemic anticholinergic effects. … [L51109] Soft drugs are designed to reduce systemic toxicity while enhancing local efficacy by employing retrometabolic drug design, in which a molecule contains a metabolically sensitive moiety that promotes
Synonyms: Pyrrolidinium, 3-(((2r)-2-cyclopentyl-2-hydroxy-2-phenylacetyl)oxy)-1-(2-ethoxy-2-oxoethyl)-1-methyl-, (3r)-Categories: MATE 1 Inhibitors, Cytochrome P-450 Substrates