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Eculizumab is a monoclonal antibody used to treat hematological diseases, such as paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). It is a recombinant humanized IgG4 kappa monoclonal antibody that binds to and inhibits the activity of complement component 5 (C5). By blocking C5, eculizumab prevents the formation of the membrane attack complex (MAC), which is responsible for the destruction of red blood cells in PNH and aHUS.
Eculizumab is the only approved therapy for PNH and aHUS, and is the only drug that can stop the progression of these diseases. It is administered intravenously, and is typically given once every two weeks. Eculizumab is also being studied for its potential use in other hematological diseases, such as myasthenia gravis and neuromyelitis optica.
Companies in the eculizumab market include Alexion Pharmaceuticals, Inc., F. Hoffmann-La Roche Ltd., and GlaxoSmithKline plc. Show Less Read more