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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
Trofinetide
go.drugbank.com/drugs/DB06045Approved[A258453] Trofinetide is marketed as DAYBUE and DAYBUE STIX in the United States, as DAYBUE in Canada, and as DAYBU in the European Union. … Trofinetide is a novel synthetic analog of [glypromate], also known as glycine–proline–glutamate (GPE), a naturally occurring protein in the brain and the N-terminal tripeptide of insulin-like growth factor
Metyrapone
go.drugbank.com/drugs/DB01011ApprovedInvestigationalAn inhibitor of the enzyme steroid 11-beta-monooxygenase. It is used as a test of the feedback hypothalamic-pituitary mechanism in the diagnosis of cushing syndrome.
Trichlormethiazide
go.drugbank.com/drugs/DB01021ApprovedVet approved(From Martindale, The Extra Pharmacopoeia, 30th ed, p830)
Nirmatrelvir
go.drugbank.com/drugs/DB16691ApprovedInvestigationalCommission on January 27, 2022. … [L39544] It was fully approved by the FDA on May 25, 2023.
Lurasidone
go.drugbank.com/drugs/DB08815ApprovedInvestigationalFood and Drug Administration (FDA) for treatment of schizophrenia on October 29, 2010 and is currently pending approval for the treatment of bipolar disorder in the United States. … Lurasidone is an atypical antipsychotic developed by Dainippon Sumitomo Pharma. It was approved by the U.S.
Ferric maltol
go.drugbank.com/drugs/DB15598ApprovedInvestigationalFerric maltol is an iron(III) atom complexed with 3 maltol molecules to increase the bioavailability compared to iron(II), without depositing it in the duodenum as insoluble ferric hydroxide and phosphate … [A189291] Ferric maltol was initially granted FDA approval on July 25, 2019, for use in adults.
Etranacogene dezaparvovec
go.drugbank.com/drugs/DB16791ApprovedInvestigationalHemophilia B - also called factor IX deficiency or Christmas disease - is an X-linked genetic disorder resulting in an absence or deficiency of clotting factor IX. … [L44156] It delivers a copy of the deficient gene that results in cell transduction and an eventual increase in circulating factor IX activity.
Categories: Complex MixturesLazertinib
go.drugbank.com/drugs/DB16216ApprovedInvestigational[A264289] It was approved by the FDA on August 19, 2024.[L51189] Lazertinib is used alone or in combination with other chemotherapeutic agents.[L51184] … [A264299, A264304] Lazertinib was first approved in South Korea on January 18, 2021, for the treatment of _EGFR_ T790M mutation-positive non-small cell lung cancer (NSCLC) with EGFR mutations.
Synonyms: N-(5-((4-(4-((DIMETHYLAMINO)METHYL)-3-PHENYL-1H-PYRAZOL-1-YL)PYRIMIDIN-2-YL)AMINO)-4-METHOXY-2-MORPHOLINOPHENYL)ACRYLAMIDE, 2-PROPENAMIDE, N-(5-((4-(4-((DIMETHYLAMINO)METHYL)-3-PHENYL-1H-PYRAZOL-1-YL)-2-PYRIMIDINYL)AMINO)-4-METHOXY-2-(4-MORPHOLINYL)PHENYL)-Ipecac
go.drugbank.com/drugs/DB13293ApprovedWithdrawnuse to cause vomiting in poisoning. … [L1113] The FDA does not have currently any approved product containing ipecac, however, ipecac as an ingredient is accepted to be sold over the counter in packages of 1 fluid ounce (30 ml) for the emergency
Categories: Complex Mixtures