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Setmelanotide
go.drugbank.com/drugs/DB11700ApprovedInvestigational[A224449] Earlier attempts at agonizing MC4R (such as LY2112688) lead to successful weight loss, but also an increase in blood pressure and heart rate. … [L24474] It is an agonist of the melanocortin 4 receptor.
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.
Linzagolix
go.drugbank.com/drugs/DB17083ApprovedInvestigational[A253667] Linzagolix was approved for use in the European Union in June 2022 for the management of symptoms caused by uterine fibroids.[L43627] … Uterine fibroids occur in >70% of women of reproductive age, and when symptomatic are associated with heavy menstrual bleeding, anemia, abdominal pain and pressure, bloating, increased urinary frequency
CTS-21166
go.drugbank.com/drugs/DB06073Investigational, and oral availability; all of these have met the requirements of an ideal oral drug candidate. … In 2008, CoMentis revealed this small compound as a transition-state analog inhibitor (structure is currently undisclosed) with excellent properties in brain penetration, selectivity, metabolic stability
Efanesoctocog alfa
go.drugbank.com/drugs/DB16662ApprovedInvestigationalepisodes in patients with hemophilia A. … [L45379] The use of FVIII replacement products is beneficial in patients with hemophilia A; however, their quality of life can be affected due to frequent doses.
Myrrh
go.drugbank.com/drugs/DB11605ApprovedInvestigationalExtractives and their physically modified derivatives such as tinctures, concretes, absolutes, essential oils, oleoresins, terpenes, terpene-free fractions, distillates, residues, obtained from Commiphora abyssinica, Burseraceae.
Acetarsol
go.drugbank.com/drugs/DB13268ApprovedWithdrawn[L2622] It was first discovered in 1921 by Ernest Fourneau at the Pasteur Institute. It was developed by Neolab Inc, and approved by Health Canada as an antifungal on December 31, 1964. … Acetarsol, with the molecular formula N-acetyl-4-hydroxy-m-arsanilic acid, is a pentavalent arsenical compound with antiprotozoal and antihelmintic properties.
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-oneLeniolisib
go.drugbank.com/drugs/DB16217ApprovedInvestigational[L45753] Investigations for using leniolisib in primary Sjögren’s syndrome are ongoing.[A258468] … [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
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.