Ravulizumab non-inferior to eculizumab in PNH

In patients with paroxysmal nocturnal hemoglobinuria (PNH) red blood cells are attacked by the complement system. Standard of care is treatment with the C5 complement inhibitor eculizumab, which currently is the only approved drug for PNH. However, in some patients C5 inhibition is not maintained during the two-week dosing interval, resulting in breakthrough hemolysis and risk of thrombosis.

Ravulizumab is a novel complement inhibitor with high affinity for C5 and a 3-4 times longer half life than eculizumab. The phase III noninferiority study ALXN1210 compared ravulizumab (q8w) to eculizumab (q2w) in 246 adult PNH patients who did not receive prior complement inhibitor therapy. Professor Jong Wook Lee (Catholic University of Korea, Seoul, South Korea) presented the results during the Late Breaking oral session.

“74% of patients receiving ravulizumab remained transfusion free, versus 66% of patients receiving eculizumab. LDH normalized in 53% of patients receiving ravulizumab, versus 49% with eculizumab”, Lee told.

“Ravulizumab given every eight weeks achieved statistically significant noninferiorty to two-weekly eculizumab on all coprimary and key secondary efficacy endpoints.”

The investigators observed a 2.5-fold reduction in breakthrough hemolysis with ravulizumab, and immediate and complete terminal complement inhibition that was sustained throughout the entire 26-week treatment period.

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Last Updated on Friday 13 July 2018.