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• W4311387653 abstract "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. Paroxysmal nocturnal haemoglobinuria (PNH) is a rare haematological disorder that is characterized by complement-mediated haemolysis, fatigue and thrombotic events. Complement component 5 (C5) inhibitors eculizumab and ravulizumab have demonstrated substantial efficacy in reducing intravascular haemolysis and improving quality of life (QOL) in patients with PNH; however, many patients continue to exhibit anaemia and require transfusions because of uncontrolled extravascular haemolysis. Subcutaneous pegcetacoplan (EMPAVELI® in the USA and ASPAVELI® in the EU) is the first C3 inhibitor approved in the USA and EU for the treatment of PNH. Pegcetacoplan targets C3 in the complement cascade, upstream of C5, thereby providing control over both intravascular and extravascular haemolysis. Pegcetacoplan was superior to supportive care in improving clinical and haematological outcomes in patients with PNH who were naïve to a complement inhibitor therapy. Similarly, pegcetacoplan was superior to eculizumab in improving clinical and haematological outcomes in patients with uncontrolled PNH despite eculizumab therapy. Pegcetacoplan also improved QOL and fatigue symptoms. Pegcetacoplan was generally well tolerated, with most adverse events being mild to moderate in severity. Current evidence indicates that pegcetacoplan is a valuable treatment option for adults with PNH." @default.
• W4311387653 created "2022-12-25" @default.
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• W4311387653 date "2022-12-01" @default.
• W4311387653 modified "2023-10-10" @default.
• W4311387653 title "Pegcetacoplan: A Review in Paroxysmal Nocturnal Haemoglobinuria" @default.
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• W4311387653 doi "https://doi.org/10.1007/s40265-022-01809-w" @default.
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