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Pegcetacoplan for PNH More Cost-Effective Than Anti-C5 Monoclonal Antibodies


A cost-utility analysis conducted from the perspective of the Italian health system found that pegcetacoplan was more effective and less costly than 2 complement 5 (C5) inhibitors for the treatment of paroxysmal nocturnal hemoglobinuria (PNH).

Pegcetacoplan, a complement 3 (C3) inhibitor, was shown to be more cost-effective for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) than eculizumab and ravulizmab, both complement 5 (C5) inhibitors, in a recent cost-utility analysis. The findings, published in ClinicoEconomics and Outcomes Research, reflect the perspective of Italy’s national health system.1

Blood samples | Image credit: © Daniel CHETRONI – stock.adobe.com

Blood samples | Image credit: © Daniel CHETRONI – stock.adobe.com

PNH is a rare and potentially fatal blood disorder that causes episodes of hemolysis and can carry associated complications including renal failure, pulmonary arterial hypertension, and myelodysplastic syndromes, as well as detrimental impacts on quality of life. Allogeneic hematopoietic stem cell transplantation is the only cure, but due to the risks inherent to the procedure, clinicians and patients may opt for medication management instead. Therapies have historically been monoclonal antibody C5 inhibitors such as eculizumab and ravulizumab, but the C3 inhibitor pegcetacoplan was approved by US and European regulators in 2021 for the treatment of PNH based on its superiority to eculizumab in clinical trials.

Considering this new treatment option, the investigators conducted a cost-utility analysis using a Markov model to compare pegcetacoplan with eculizumab and ravulizumab for PNH from the perspective of the Italian National Health Service. The modeled patient population matched pegcetacoplan’s indication in Europe—adults with PNH who experience anemia (hemoglobin levels < 10.5 g/dL) despite treatment with eculizumab—and patient characteristics were based on the participants in the phase 3 PEGASUS trial2 that led to the C3 inhibitor’s approval.

Data on pegcetacoplan’s effectiveness vs eculizumab were drawn from PEGASUS, whereas comparisons vs ravulizumab were based on a noninferiority trial for eculizumab and ravulizumab.1 Prices were extracted from Tunnel software, and quality of life was calculated using the European Organisation for Research and Treatment of Cancer Core Quality of Life Questionnaire, or EORTC QLQ-30, scale.

Investigators calculated 5-year total per-patient costs of €1,483,454 for pegcetacoplan, €1,585,763 for eculizumab, and €1,574,826 for ravulizumab, which are roughly equivalent to $1,581,050, $1,690,090, and $1,678,434, respectively. These totals reflect the costs of the drugs themselves, supportive treatments, administration, and health state (ie, costs of managing complications, blood transfusions, and other resource use). Although the lower total costs for pegcetacoplan were primarily driven by its lower treatment cost, the C3 inhibitor also incurred less spending on complications and transfusions.

Pegcetacoplan also resulted in more quality-adjusted life-years (QALYs) than eculizumab and ravulizumab, at 3.31 vs 2.90 and 3.05 QALYs, respectively. These QALY gains were largely due to transfusion avoidance with fewer adverse events and no need for intravenous infusion, according to the authors.

Based on these calculations, the investigators concluded that pegcetacoplan dominates the comparators in that it is more effective, less costly, and leads to better quality of life. They highlighted pegcetacoplan’s ability to avert the need for transfusions by boosting hemoglobin levels; in PEGASUS, the mean hemoglobin level reached 11.5 g/dL by week 16.

“Moreover, the lower occurrence of complications reported with pegcetacoplan, in the study, [led] to cost savings contributing to its better cost-effectiveness profile,” the authors wrote. “It should be noted that pegcetacoplan dominates both eculizumab and ravulizumab with greater QALY gain and a reduction in total health care cost.”

The patent on eculizumab is still active, but the authors also conducted a hypothetical analysis reflecting a potential biosimilar launch. Even if all incident patients received a biosimilar of eculizumab at a 40% discount, pegcetacoplan would still be dominant, they found.

Limitations include that the use of the PEGASUS trial cohort restricted to the analysis to patients who continued to experience anemia after treatment with eculizumab, meaning that the analysis applies only to a pretreated population, not to newly diagnosed patients. Also, the perspective focusing on costs incurred to a national health system may restrict the findings’ generalizability to the US landscape.

Nonetheless, the authors concluded that “pegcetacoplan may represent significant clinical and economic value within the PNH treatment pathway” and emphasized its potential role in managing PNH in the years to come.


1. Di Matteo S, Freilone R, Bruno GM, et al. Cost-utility analysis comparing pegcetacoplan to anti-C5 monoclonal antibodies in the treatment of paroxysmal nocturnal hemoglobinuria. Clinicoecon Outcomes Res. 2024;16:225-232. doi:10.2147/CEOR.S442906

2. McNulty R. Hemoglobin improvement correlates with better HRQOL in paroxysmal nocturnal hemoglobinuria. AJMC. September 1, 2022. Accessed April 18, 2024. https://www.ajmc.com/view/hemoglobin-improvement-correlates-with-better-hrqol-in-paroxysmal-nocturnal-hemoglobinuria

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