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Human C1-esterase inhibitor
go.drugbank.com/drugs/DB06404ApprovedInvestigational[L16586, L16606] This drug is indicated for prophylaxis and treatment of Hereditary Angioedema (HAE), a human genetic disorder caused by a shortage of C1 inhibitor activity that results in an overreaction … The disease is characterized by acute attacks of painful, and in some cases, fatal swelling of several soft tissues or edema, which may last up to five days when untreated.[L16586, L16606]
Categories: Drugs Used in Hereditary AngioedemaEfgartigimod alfa
go.drugbank.com/drugs/DB15270ApprovedInvestigationalEfgartigimod alfa for intravenous use was granted FDA approval on December 17, 2021[L39501] and European Commission approval on August 11, 2022 for use in patients with myasthenia gravis. … and a reduction in electrical nerve impulses.
Sebetralstat
go.drugbank.com/drugs/DB18305ApprovedInvestigationalFood and Drug Administration approved sebetralstat on July 7, 2025, for treating acute HAE attacks in patients aged 12 years and older [L53533, L53538]. … This plasma kallikrein inhibitor works by reducing bradykinin, the substance that causes the characteristic swelling in HAE attacks [L53533]. The U.S.
Categories: Drugs Used in Hereditary AngioedemaSynonyms: 1H-Pyrazole-4-carboxamide, N-[(3-fluoro-4-methoxy-2-pyridinyl)methyl]-3-methoxymethyl)-1-[[4-[(2-oxo-1(2H)-pyridinyl)methyl]phenyl]methyl]-, N-[(3-fluoro-4-methoxypyridin-2-yl)methyl]-3-(methoxymethyl)-1-({4-[(2-oxopyridin-1(2H)-yl)methyl]phenyl}methyl)-1H-pyrazol-4-carboxamideDebrisoquine
go.drugbank.com/drugs/DB04840ApprovedAn adrenergic neuron-blocking drug similar in effects to guanethidine. It is also noteworthy in being a substrate for a polymorphic cytochrome P-450 enzyme.
Mitapivat
go.drugbank.com/drugs/DB16236ApprovedInvestigational[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] … Mitapivat is a novel, first-in-class pyruvate kinase activator. It works to increase the activity of erythrocyte pyruvate kinase, a key enzyme involved in the survival of red blood cells.
Synonyms: Pkr-in-1Protein C
go.drugbank.com/drugs/DB11312ApprovedInvestigationalOnce in its activated form, APC functions as a serine protease with potent anticoagulant effects, especially in the presence of its cofactor protein S. … A lack of protein C in the body would lead to unchecked coagulation activation, resulting in thrombin generation and intravascular clot formation.
Mixtures: Beriplex P/n 500, Beriplex P/n 1000Rhizopus stolonifer
go.drugbank.com/drugs/DB10814ApprovedRhizopus stolonifer extract is used in allergenic testing.
Mixtures: Number Ten Mold Mixture, Number Ten Mold MixtureOlezarsen
go.drugbank.com/drugs/DB18728ApprovedInvestigationalOlezarsen is an antisense oligonucleotide that targets the messenger RNA (mRNA) for apolipoprotein C-III (APOC3, apoC-III),[L52033] which is a key regulator of triglyceride levels in plasma, in the liver … [A264888,A264893] Olezarsen is conjugated to a ligand containing three N-acetyl-galactosamine (GalNAc) residues to enable its delivery to hepatocytes.
Phenoxyethanol
go.drugbank.com/drugs/DB11304ApprovedInvestigationalIt is produced by reacting phenol (EU) and ethylene oxide (EU) at a high temperature and pressure. This substance occurs naturally in green tea (EU) [L2621]. … According to the European Union Cosmetics Regulation (EC) n.1223/2009, phenoxyethanol is authorized as a preservative in cosmetic formulations at a maximum concentration of 1.0% [L2625].
Benzatropine
go.drugbank.com/drugs/DB00245ApprovedInvestigationalThe generation of structure-activity relationships proved that benztropine derivatives with the presence of a chlorine substituent in the para position in one of the phenyl rings produces an increased … [A37914] Benztropine was developed by USL Pharma and officially approved by the FDA on 1996.[L5500]