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Mitapivat
go.drugbank.com/drugs/DB16236ApprovedInvestigationalDefects in the pyruvate kinase enzyme in various red blood cells disorders lead to the lack of energy production for red blood cells, leading to lifelong premature destruction of red blood cells or chronic … [A245478] On January 5, 2026, the FDA also approved the use of in the treatment of anemia in adults with alpha- or beta-thalassemia, making it the first oral treatment for this condition.[L54953]
Gedatolisib
go.drugbank.com/drugs/DB11896ApprovedInvestigationalGedatolisib is a kinase inhibitor that blocks all four class I PI3K isoforms together with both mTOR complexes, mTORC1 and mTORC2, producing downstream inhibition of multiple effectors including AKT. … PI3Kα, AKT, or mTORC1). Celcuity describes gedatolisib as the first FDA-approved therapy to inhibit the pathway comprehensively across all class I PI3K isoforms and both mTOR complexes.
Sapropterin
go.drugbank.com/drugs/DB00360ApprovedInvestigationalSapropterin (tetrahydrobiopterin or BH4) is a cofactor in the synthesis of nitric oxide.
Telaprevir
go.drugbank.com/drugs/DB05521ApprovedWithdrawnHCV is a single-stranded RNA virus that is categorized into nine distinct genotypes, with genotype 1 being the most common in the United States, and affecting 72% of all chronic HCV patients [L852]. … of HCV infection is associated with significant long-term health benefits including reduced liver-related damage, improved quality of life, reduced incidence of Hepatocellular Carcinoma, and reduced all-cause
Categories: Antivirals for Systemic Use, Antiinfectives for Systemic UseMethionine
go.drugbank.com/drugs/DB00134ApprovedInvestigationalNutraceuticalIt is a chelating agent for heavy metals.
Mixtures: GLAMIN SOLUTION FOR INFUSION, KABIVEN EMULSION FOR INFUSIONOmbitasvir
go.drugbank.com/drugs/DB09296ApprovedInvestigationalHCV is a single-stranded RNA virus that is categorized into nine distinct genotypes, with genotype 1 being the most common in the United States, and affecting 72% of all chronic HCV patients [L852]. … of HCV infection is associated with significant long-term health benefits including reduced liver-related damage, improved quality of life, reduced incidence of Hepatocellular Carcinoma, and reduced all-cause
Categories: Antivirals for Systemic Use, Treatments for Hepatitis CGalactose
go.drugbank.com/drugs/DB11735ApprovedInvestigationalWithdrawnThere are even proposals for its use in accelerating senescence in mice, rats, and Drosophila, for its association with ovarian cancer, or even for the potential treatment of focal segmental glomerulosclerosis … Nevertheless, none of these ongoing studies have yet provided formal elucidation for their proposals. As a naturally occurring sugar, it may be found in a number dairy products.
Categories: Tests for Liver Functional CapacityCerliponase alfa
go.drugbank.com/drugs/DB13173ApprovedInvestigational[L51319] On April 27, 2017, cerliponase alfa was first approved by the FDA as the first treatment for neuronal ceroid lipofuscinosis type 2 (CLN2), also known as TPP1 deficiency.
Zotepine
go.drugbank.com/drugs/DB09225ApprovedWithdrawnIn the EMA, by 2015 it was under pharmacovigilance studies for the potential treatment of acute renal failure.[L1314]
Elvitegravir
go.drugbank.com/drugs/DB09101ApprovedInvestigationalOn September 24, 2014, the FDA approved the single pill form of elvitegravir. … Elvitegravir is a human immunodeficiency virus type 1 (HIV-1) integrase strand transfer inhibitor (INSTI) used for the treatment of HIV-1 infection in antiretroviral treatment-experienced adults.
Categories: Antivirals for Systemic Use, Antiinfectives for Systemic Use