Summary of Clinical Use

Used to treat paroxysmal nocturnal hemoglobinuria (PNH). In the UK, NICE (National Institute for Health and Care Excellence) has approved eculizumab as a treatment for the rare, but fatal blood disorder, atypical haemolytic uraemic syndrome (aHUS). This decision was reached because eculizumab represents a significant innovation for a disease with a high unmet clinical need. Eculizumab treatment is expected to increase life expectancy of aHUS patients by decades. The FDA expanded approval in October 2017, to include treatment of patients with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AchR) antibody-positive.

Mechanism Of Action and Pharmacodynamic Effects

This antibody blocks the cleavage of the complement protein C5 and thereby prevents the formation of C5a and the membrane attack complex, which lowers the risk of blood clot formation and intravascular hemolysis, the most common thrombotic complications underlying mortality in PNH patients. Chronic uncontrolled activation of the complement system also plays a major role in the debilitating symptoms and potentially life-threatening complications for anti-AchR antibody-positive gMG patients. Ecilizumab effectively inhibits the terminal complement cascade and directly targets this critical underlying cause of gMG.

External links

For extended ADME data see the following: Electronic Medicines Compendium (eMC) Drugs.com European Medicines Agency (EMA)