Information about Eculizumab
Eculizumab is a humanized monoclonal antibody to complement factor 5 which acts to block complement activation and is used to treat paroxysmal nocturnal hemoglobinuria and hemolytic uremic syndrome.
Liver safety of Eculizumab
Eculizumab has been linked to several instances of serum enzyme elevations after repeated infusions and to rare instances of clinically apparent acute liver injury.
Mechanism of action of Eculizumab
Eculizumab (e" kue liz' ue mab) is a recombinant, humanized IgG monoclonal antibody to complement factor 5, which inhibits its enzymatic cleavage and activation. Activated complement is an important mediator of immune damage including hemolysis of red blood cells and plays an essential role in the hemolysis and tissue damage that accompanies paroxysmal nocturnal hemoglobinuria (PNH) and hemolytic uremic syndrome (HUS). In clinical trials in PNH, eculizumab was found to reduce hemolysis and the need for blood transfusions with subsequent improvement in symptoms and quality of life.
FDA approval information for Eculizumab
Eculizumab was approved for use in PNH in the United States in 2007. The indications were later broadened to include atypical hemolytic uremic syndrome with complement-mediated thrombotic events in 2011.
Dosage and administration for Eculizumab
Eculizumab is available as a solution in single dose vials of 300 mg in 30 mL (10 mg/mL) under the commercial name Soliris. The recommended dose varies by body weight and indication, but it is typically given by intravenous infusion (over 35 minutes) weekly for 5 weeks and every two weeks thereafter.
Side effects of Eculizumab
Side effects are not common, but can include headache, diarrhea, nausea, fatigue and upper respiratory tract infections. Rare, but potentially severe adverse reactions include serious infections, including meningococcal infections, for which reason eculizumab is available only as a part of a risk evaluation and mitigation strategy (REMS) that requires physician training in its use and enrollment of the patient in a surveillance program.
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