Subcutaneous
pegcetacoplan (EMPAVELI® in the USA and ASPAVELI® in the EU) is the first
complement component 3 (C3) inhibitor approved for the treatment of adults with paroxysmal nocturnal haemoglobinuria (PNH) in the USA, and in adults with PNH who are anaemic after ≥ 3 months of treatment with a C5 inhibitor in the EU. In the phase III PRINCE trial in adults with PNH who were anaemic and naïve to a
complement inhibitor therapy,
pegcetacoplan was superior to the control group (supportive care, excluding
complement inhibitors) in achieving haemoglobin stabilization and reducing
lactate dehydrogenase levels. Similarly, in the phase III PEGASUS trial in adults with PNH who had a haemoglobin level < 10.5 g/dL despite
eculizumab therapy,
pegcetacoplan was superior to
eculizumab in improving haemoglobin levels. In both trials,
pegcetacoplan also improved other clinical and haematological parameters of
haemolysis, as well as quality of life (QOL) outcomes. Clinical benefits of
pegcetacoplan were sustained for up to 48 weeks of treatment.
Pegcetacoplan was generally well tolerated in patients with PNH, with its tolerability profile being similar in patients previously treated with
eculizumab and in
complement inhibitor-naïve patients. Long-term data would be beneficial to further support the safety profile of
pegcetacoplan. Current evidence indicates that
pegcetacoplan is a valuable treatment option for adults with PNH.