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Garetosmab
go.drugbank.com/drugs/DB16379ApprovedInvestigational[L63940,L64047] It is the second therapy approved for FOP, following [palovarotene] (Sohonos), which was approved in 2023, and is the first FOP therapy shown to reduce clinician-assessed flare-ups. … [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
Ensifentrine
go.drugbank.com/drugs/DB16157ApprovedInvestigational[L50903] It works to induce bronchodilator and reduce inflammation in COPD.[A263913] … Ensifentrine is a first-in-class, selective dual inhibitor of the phosphodiesterase 3 (PDE3) and phosphodiesterase 4 (PDE4) enzymes.
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)-Praziquantel
go.drugbank.com/drugs/DB01058ApprovedInvestigationalVet approved[A263201] The efficacy of praziquantel in treating parasitic flatworms infection with low cost (~US$0.20 drug cost to treat a child) makes it an integral to WHO's plan to eliminate schistosomiasis by 2030 … Specifically, it is known as a treatment of trematodes and cestodes infections such as schistosomiasis, taeniasis, and cysticercosis.
Vernakalant
go.drugbank.com/drugs/DB06217ApprovedInvestigationalIntravenous formulation was approved in Europe in September 2010 as Brinavess and in Canada in April 2017. It is an investigational drug under regulatory review by FDA. … It acts as an atypical class III antiarrhythmic drug that potentiates its effect in higher heart rates.
Vibegron
go.drugbank.com/drugs/DB14895ApprovedInvestigationalOn December 23, 2020, vibegron was approved for the same indication in adults. It is available as oral tablets under the market name GEMTESA. … [L28305] Vibegron was first approved in Japan in September 2018 for the treatment of overactive bladder,[A226050] a condition associated with distressing symptoms of urge urinary incontinence, urgency,
Dabrafenib
go.drugbank.com/drugs/DB08912ApprovedInvestigational[L41955] In May 2018, Tafinlar (dabrafenib), in combination with Mekinist ([DB08911]), was approved to treat anaplastic thyroid cancer caused by an abnormal BRAF V600E gene.[L41955] … It was approved on May 29, 2013, for the treatment of melanoma with V600E or V6000K mutation.[L41955] It was also used for metastatic non-small cell lung cancer with the same mutation.
Acoramidis
go.drugbank.com/drugs/DB17999ApprovedInvestigationalAcoramidis is a small molecule stabilizer of transthyretin (TTR) for use in patients with TTR amyloidosis. … [A264808] It was brought to market by BridgeBio Pharma and approved by the FDA in November 2024 to reduce negative cardiovascular outcomes in patients with cardiomyopathy caused by TTR amyloidosis.
Opicapone
go.drugbank.com/drugs/DB11632ApprovedInvestigationalOpicapone was approved for use by the European Commission in June 2016 [L2339] and the FDA in April 2020.[L13772] It is marketed under the brand name Ongentys as once-daily oral capsules. … Opicapone is a potent, reversible, and peripherally-acting third-generation inhibitor of catechol-o-methyltransferase (COMT), an enzyme involved in the breakdown of various catecholamines including dopamine
Netupitant
go.drugbank.com/drugs/DB09048ApprovedInvestigationalNetupitant 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 … Netupitant is a neurokinin 1 receptor antagonist. The combination drug is marketed by Eisai Inc. and Helsinn Therapeutics (U.S.) Inc. under the brand Akynzeo.
Synonyms: 2-[3,5-Bis(trifluoromethyl)phenyl]-N,2-dimethyl-N-[4-(2-methylphenyl)-6-(4-methyl-1-piperazinyl)-3-pyridinyl]propanamidePegvaliase
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 … Individuals with PKU also need to be under a strictly restricted diet as Phe is present in foods and products with high-intensity sweeteners [L2925].