Summary

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• Pegcetacoplan (Empaveli) is indicated for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH). It targets complement 3 (C3), offering an alternative mechanism to control both intravascular and extravascular hemolysis. This makes it a strategic option for patients refractory to eculizumab. The drug has shown effectiveness in increasing hemoglobin levels, achieving transfusion independence, and reducing lactate dehydrogenase (LDH) levels.
• Four studies provided information on the safety and efficacy of pegcetacoplan compared to other treatments like eculizumab and ravulizumab.
• Compared to first-line treatments such as eculizumab and ravulizumab, which act as C5 inhibitors, pegcetacoplan offers an alternate approach by targeting C3. This makes it particularly beneficial for those who develop refractoriness to eculizumab.
• A pooled data analysis from 27 studies showed significant reduction in LDH levels, increase in hemoglobin level, and transfusion avoidance among treatment-naïve PNH patients treated with complement inhibitors including pegcetacoplan over varying durations.
• Subgroup considerations suggest that individualized treatment plans are crucial, especially for patients unresponsive or refractory to eculizumab. In these cases, due to its action on C3 inhibition rather than C5 inhibition, pegcetacoplan might be more effective.
• Despite promising results showing substantial benefits of using Empaveli (pegcetacoplan), there is a call for more randomized controlled trials to provide robust evidence to support its use.