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Eplontersen
go.drugbank.com/drugs/DB16199ApprovedInvestigationalThis approval was based on positive results demonstrated in the 35-week interim analysis from the Phase 3 NEURO-TTRansform study, where a consistent improvement in the Neuropathy Impairment Score +7 (mNIS … [A262975] Hereditary transthyretin-mediated amyloidosis is caused primarily by pathogenic variants of the TTR gene, leading to the formation and thus accumulation of insoluble and misfolded amyloid in
Synonyms: Ion-ttr-lrxMarstacimab
go.drugbank.com/drugs/DB17725ApprovedInvestigational[A264608] Marstacimab is used to prevent bleeding episodes in patients with hemophilia,[L51803] which is an X‐linked bleeding disorder characterized by coagulation clotting factor deficiency. … [L51803] It is a tissue factor pathway inhibitor (TFPI) antagonist, which is an endogenous anticoagulant.
Dordaviprone
go.drugbank.com/drugs/DB14844ApprovedInvestigationalDordaviprone is a first-in-class small molecule imipridone. On August 6, 2025, dordaviprone was granted accelerated approval by the FDA. … [L53838] It is used to treat patients with diffuse midline glioma harboring an H3 K27M mutation.
Modafinil
go.drugbank.com/drugs/DB00745ApprovedInvestigationalThe exact mechanism of action is unclear, although in vitro studies have shown it to inhibit the reuptake of dopamine by binding to the dopamine reuptake pump, and lead to an increase in extracellular … Modafinil is a stimulant drug marketed as a 'wakefulness promoting agent' and is one of the stimulants used in the treatment of narcolepsy.
Ravulizumab
go.drugbank.com/drugs/DB11580ApprovedInvestigationalRavulizumab is a potent and selective complement 5 (C5) inhibitor. It is a humanized monoclonal IgG2/4 kappa antibody produced in Chinese hamster ovary (CHO) cells. … [A244460] It works by blocking terminal complement-mediated inflammation, cell activation, and cell lysis in blood disorders associated with the destruction of red blood cells, thrombosis, and impaired
Bacillus calmette-guerin substrain russian BCG-I live antigen
go.drugbank.com/drugs/DB16648ApprovedInvestigationalthought to work by stimulating a local immune and inflammatory response in the bladder. … [L32729,A232264] The live Russian BCG-I substrain of BCG was granted a notice of compliance with conditions by Health Canada in February 2021.
Synonyms: Bacillus Calmette-Guérin (BCG) - strain russian BCG-I, Bacillus calmette-guerin substrain russian BCG-I live antigenDoxylamine
go.drugbank.com/drugs/DB00366ApprovedInvestigationalVet approvedIt is used in allergies and as an antitussive, antiemetic, and hypnotic. Doxylamine has also been administered in veterinary applications and was formerly used in parkinsonism.
Mixtures: Robitussin Maximum Strength Severe Multi-Symptom 7 in 1 Relief Nighttime, N - TimeSynonyms: N,N-Dimethyl-2-(1-phenyl-1-(2-pyridinyl)ethoxy)ethanamine, Phenyl-2-pyridylmethyl-beta-N,N-dimethylaminoethyl etherDoxercalciferol
go.drugbank.com/drugs/DB06410ApprovedInvestigationalDoxercalciferol is indicated for the treatment of secondary hyperparathyroidism in patients with chronic kidney disease on dialysis, as well as for the treatment of secondary hyperparathyroidism in patients … Doxercalciferol is a synthetic vitamin D2 analog that undergoes metabolic activation in vivo to form 1α,25-dihydroxyvitamin D2 (1α,25-(OH)2D2), a naturally occurring, biologically active form of vitamin
Temazepam
go.drugbank.com/drugs/DB00231ApprovedIn particular, before temazepam saw regular prescription use in civilians it was - and still is - employed by the US military as a sedative-hypnotic medication to be taken by soldiers, pilots, etc. to … capable of developing drug tolerance, physical dependence, and addiction in users.
Methyltestosterone
go.drugbank.com/drugs/DB06710ApprovedInvestigationalAlso, has antineoplastic properties and so has been used secondarily in women with advanced breast cancer. Methyltestosterone is a schedule III drug in the US.
Synonyms: 17-beta-Hydroxy-17-methylandrost-4-en-3-one, 17beta-Hydroxy-17-methylandrost-4-en-3-one