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Eculizumab
go.drugbank.com/drugs/DB01257ApprovedInvestigational[L6919] In Q1 2023, the EMA's Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of two formulations of eculizumab indicated for the treatment of paroxysmal nocturnal hemoglobinuria … Eculizumab is a monoclonal antibody that targets complement protein C5.
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
Deutetrabenazine
go.drugbank.com/drugs/DB12161ApprovedInvestigationalIn clinical trials of patients with HD, 12 weeks of treatment of deutetrabenazine resulted in overall improvement in mean total maximal chorea scores and motor signs than placebo [A32046]. … It was approved by FDA in April 2017 and is marketed under the trade name Austedo as oral tablets.
Enoximone
go.drugbank.com/drugs/DB04880ApprovedInvestigationalWithdrawnIt is used in patients with congestive heart failure. Trials were halted in the U.S., but the drug is used in various countries. … Enoximone is a selective phosphodiesterase inhibitor with vasodilating and positive inotropic activity that does not cause changes in myocardial oxygen consumption.
Categories: Compounds used in a research, industrial, or household settingFlosequinan
go.drugbank.com/drugs/DB13228ApprovedWithdrawnFlosequinan was approved in the USA and the UK for a year prior to being withdrawn from the market due to increased mortality in chronic heart failure patients, found in drug trials.[A174979,L43942]
Categories: Vasodilators Used in Cardiac DiseasesDextrothyroxine
go.drugbank.com/drugs/DB00509ApprovedWithdrawnThyroxine is synthesized via the iodination of tyrosines (monoiodotyrosine) and the coupling of iodotyrosines (diiodotyrosine) in the thyroglobulin. … Thyroxine is peripherally deiodinated to form triiodothyronine which exerts a broad spectrum of stimulatory effects on cell metabolism.
Omaveloxolone
go.drugbank.com/drugs/DB12513ApprovedInvestigational[L45424] The EMA approved this drug the following year, in February 2024, and Health Canada in March 2025 for the same condition. … [A257529] In February 2023, omaveloxolone was approved by the FDA for the treatment of Friedreich's ataxia in adults and adolescents aged 16 years and older.
Synonyms: Propanamide, N-(2-cyano-3,12-dioxo-28-noroleana-1,9(11)-dien-17-yl)-2,2-difluoro-Pegunigalsidase alfa
go.drugbank.com/drugs/DB14992ApprovedInvestigational[A259347,A259352] In May 2023, the EMA granted marketing authorization to pegunigalsidase alfa in the European Union (EU) for the treatment of adult patients with Fabry disease. … Pegunigalsidase alfa (PRX-102) is a recombinant form of human α-galactosidase-A indicated for long-term enzyme replacement therapy in patients with Fabry disease, a rare genetic disorder characterized
Cladribine
go.drugbank.com/drugs/DB00242ApprovedInvestigational[A263733] In 2017 in Europe and in 2019 in the United States, cladribine was also approved for the treatment multiple sclerosis.[A263718] … [A350] Cladribine was first approved in the United States in 1993 [A263713] initially as a treatment for a number of hematological malignancies; currently, it is approved for the treatment of hairy cell
Ravulizumab
go.drugbank.com/drugs/DB11580ApprovedInvestigationalRavulizumab is a potent and selective complement 5 (C5) inhibitor. It is a humanized monoclonal IgG2/4 kappa antibody produced in Chinese hamster ovary (CHO) cells. … [A244460] It works by blocking terminal complement-mediated inflammation, cell activation, and cell lysis in blood disorders associated with the destruction of red blood cells, thrombosis, and impaired