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Eculizumab
go.drugbank.com/drugs/DB01257ApprovedInvestigationalEculizumab is a monoclonal antibody that targets complement protein C5. … [L6919] In Q1 2023, the EMA's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of two formulations of eculizumab indicated for the treatment of paroxysmal nocturnal hemoglobinuria
Pegvaliase
go.drugbank.com/drugs/DB12839ApprovedInvestigationalPhenylketonuria is a rare autosomal recessive disorder that is characterized by deficiency of the enzyme phenylalanine hydroxylase (PAH) [A33284] and affects about 1 in 10,000 to 15,000 people in the United … The presence of a PEG moiety in pegvaliase-pqpz allows a reduced immune response and improved pharmacodynamic stability [A33284].
Cannabidiol
go.drugbank.com/drugs/DB09061ApprovedInvestigationalIn addition to the well-known activity on CB1 and CB2 receptors, there is further evidence that CBD also activates 5-HT1A/2A/3A serotonergic and TRPV1–2 vanilloid receptors, antagonizes alpha-1 adrenergic … changed significantly with regards to its usefulness in a number of medical conditions ranging from anxiety to epilepsy.
Synonyms: (1'R,2'R)-5'-methyl-4-pentyl-2'-(prop-1-en-2-yl)-1',2',3',4'-tetrahydrobiphenyl-2,6-diolEnsifentrine
go.drugbank.com/drugs/DB16157ApprovedInvestigationalEnsifentrine is a first-in-class, selective dual inhibitor of the phosphodiesterase 3 (PDE3) and phosphodiesterase 4 (PDE4) enzymes. … [L50903] It works to induce bronchodilator and reduce inflammation in COPD.[A263913]
Synonyms: N-(2-((2E)-9,10-DIMETOXI-4-OXO-2-((2,4,6-TRIMETILFENIL)IMINO)-6,7-DIHIDRO-2H-PIRIMIDO(6,1-A)ISOQUINOLEIN-3(4H)-IL)ETIL)UREA, UREA, N-(2-(6,7-DIHYDRO-9,10-DIMETHOXY-4-OXO-2-((2,4,6-TRIMETHYLPHENYL)IMINO)-2H-PYRIMIDO(6,1-A)ISOQUINOLIN-3(4H)-YL)ETHYL)-Garetosmab
go.drugbank.com/drugs/DB16379ApprovedInvestigational[L63940] It is a fully human IgG4 monoclonal antibody that binds and neutralizes activin A, blocking activin A from activating the mutant ACVR1 receptor that, in FOP, drives the formation of heterotopic … Garetosmab-grts is an activin A inhibitor used to treat fibrodysplasia ossificans progressiva (FOP).
Netupitant
go.drugbank.com/drugs/DB09048ApprovedInvestigationalNetupitant is a neurokinin 1 receptor antagonist. The combination drug is marketed by Eisai Inc. and Helsinn Therapeutics (U.S.) Inc. under the brand Akynzeo. … Netupitant is an antiemitic drug approved by the FDA in October 2014 for use in combination with palonosetron for the prevention of acute and delayed vomiting and nausea associated with cancer chemotherapy
Synonyms: 2-[3,5-Bis(trifluoromethyl)phenyl]-N,2-dimethyl-N-[4-(2-methylphenyl)-6-(4-methyl-1-piperazinyl)-3-pyridinyl]propanamideLusutrombopag
go.drugbank.com/drugs/DB13125ApprovedInvestigationalLusutrombopag is currently in phase III development in various European countries including Austria, Belgium, Germany, and the UK [A36730]. … This hematological abnormality, especially in cases of severe thrombocytopenia (platelet count <50,000/μL), creates challenges to patients requiring invasive medical procedures where there is a significant
Pipradrol
go.drugbank.com/drugs/DB11584ApprovedWithdrawnPipradrol was made illegal in many countries in 1970s because of its potential for abuse. It is currently classified under the Misuse of Drugs Act as a Class C substance [L2522]. … In addition to this, it has shown favorable effects in postpartum depressive symptoms [L2516].
Metreleptin
go.drugbank.com/drugs/DB09046ApprovedInvestigationalMetreleptin, a recombinant analog of the human hormone leptin, is an orphan drug used to treat complications of leptin deficiency in people with lipodystrophy. … Metreleptin is produced in _E. coli_ and differs from native human leptin by the addition of a methionine residue at its amino terminus.
Synonyms: N-MethionylleptinBrincidofovir
go.drugbank.com/drugs/DB12151ApprovedInvestigationalBrincidofovir is an oral antiviral drug used in the treatment of human smallpox infections. … It is a lipid conjugate pro-drug of the acyclic nucleotide analogue [cidofovir][L34404,A235725] - this lipid conjugate improves drug delivery to the target cells and significantly reduces the nephrotoxicity