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Alpelisib
go.drugbank.com/drugs/DB12015ApprovedInvestigational[L6652] In April 2022, the FDA granted the use of alpelisib in the treatment of PIK3CA-Related Overgrowth Spectrum (PROS) in adults and children who require systemic therapy.[L41384] … It works by selectively inhibiting class I PI3K p110α [A179203], which is the catalytic subunit of PI3K, a lipid kinase that plays a role in various biological processes, including proliferation, survival
Categories: Class I Phosphatidylinositol 3-Kinases, antagonists & inhibitorsExemestane
go.drugbank.com/drugs/DB00990ApprovedInvestigationalExemestane is an oral steroidal aromatase inhibitor used in the adjuvant treatment of hormonally-responsive (also called hormone-receptor-positive, estrogen-responsive) breast cancer in postmenopausal … It irreversibly binds to the active site of the enzyme resulting in permanent inhibition.
Fluticasone furoate
go.drugbank.com/drugs/DB08906ApprovedInvestigationalFluticasone furoate is a synthetic glucocorticoid available as an inhaler and nasal spray for various inflammatory indications[FDA Label][F4364]. Fluticasone furoate was first approved in 2007[L5965].
mRNA-1345
go.drugbank.com/drugs/DB18739ApprovedInvestigationalmRESVIA was first approved in the EU and US in 2024 for use in patients 60 years of age and older. … [L53403,L53393] It is additionally under investigation for maternal use and use in children.[A274028,L53438,L53443]
Nylidrin
go.drugbank.com/drugs/DB06152ApprovedWithdrawnNylidrin is a peripheral vasodilator. … FDA has considered nylidrin as "lacking substantial evidence of effectiveness" in cerebral ischemia, cerebral arteriosclerosis, and other cerebral circulatory insufficiencies.
Fluoroestradiol F-18
go.drugbank.com/drugs/DB15690ApprovedInvestigational[L14117] It is expected to be available in late 2020/early 2021.[L14117] … [L14096] The ability to image ER-positive tumors _in vivo_ is advantageous in that, while helping to visualize tumor progression/regression, it may also be used to assess for heterogeneity in ER expression
Zilganersen
go.drugbank.com/drugs/DB18161ApprovedInvestigational[L64096] Alexander disease is caused by variants in the *GFAP* gene that lead to overproduction and toxic accumulation of GFAP in astrocytes, and dysfunction in astrocytes can over time damage neurons … [L64096] The FDA approved zilganersen on September 3, 2026 for the treatment of Alexander disease in pediatric and adult patients, making it the first and only disease-modifying treatment for the condition
Tividenofusp alfa
go.drugbank.com/drugs/DB17628ApprovedInvestigationalTividenofusp alfa is a first-in-class, brain-penetrant enzyme replacement therapy (ERT) indicated for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome [L56097, L56099 … Hunter syndrome is a rare, X-linked lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase (IDS), which leads to the toxic accumulation of glycosaminoglycans (GAGs) throughout
Nitisinone
go.drugbank.com/drugs/DB00348ApprovedNitisinone is a synthetic reversible inhibitor of 4-hydroxyphenylpyruvate dioxygenase. It is used in the treatment of hereditary tyrosinemia type 1 and Alkaptonuria. … It is sold under the brand names Orfadin and Harliku. [L53253, L53258]
Efgartigimod alfa
go.drugbank.com/drugs/DB15270ApprovedInvestigational[A243759,A243784] Efgartigimod alfa is a first-in-class[L39501] antagonist of the neonatal Fc receptor (FcRn) used in the treatment of MG. … and a reduction in electrical nerve impulses.