Pegcetacoplan (Empaveli) is approved for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) who are treatment naïve and those who are switching from the C5 inhibitors eculizumab (Soliris) and ravulizumab (Ultomiris).

It is the first approved targeted C3 therapy for PNH.



Phase 3 PEGASUS study, demonstrated superiority to eculizumab for the change from baseline in hemoglobin level at week 16, with an adjusted mean increase of 3.84 g/dL of hemoglobin.



Pegcetacoplan was found to have noninferiority to eculizumab on the end point of transfusion avoidance: 85% of patients who received pegcetacoplan were transfusion free over 16 weeks compared with 15% of those who received eculizumab.



The agent may increase the risk of meningococcal and other serious infections caused by encapsulated bacteria that may become rapidly life threatening or fatal if not recognized and treated early.



The most common serious adverse effect in patients treated with pegcetacoplan was infections (5%). 



The most common toxicities: injection site reactions (39%), infections (29%), diarrhea (22%), abdominal pain (20%), respiratory tract infection (15%), viral infection (12%), and fatigue (12%). 



No cases of meningitis and no deaths were reported in patients treated with pegcetacoplan.



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