Eculizumab is a monoclonal antibody, which is prescribed for two conditions:
• Paroxysmal nocturnal hemoglobinuria (PNH) in which red blood cells (RBCs) are broken down resulting in anemia and decreased oxygen supply to the body.
• Atypical hemolytic uremic syndrome (aHUS), where small blood clots are formed which block blood supply and cause damage to kidney and other organs.
Eculizumab is an orphan drug since it is used only for the above rare conditions. It acts by blocking a part of the immune system called the complement system. In paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome, the complement system acts abnormally and destroys the red blood cells and increases the risk of blood clots respectively.
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