Novel targeted C3 inhibitor pegcetacoplan for paroxysmal nocturnal hemoglobinuria
Sign Up to like & getrecommendations! Published in 2022 at "Clinical and Experimental Medicine"
DOI: 10.1007/s10238-022-00830-3
Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, life-threatening acquired blood disease characterized by chronic complement-mediated hemolysis and thrombosis. On May 14, 2021, the US FDA approved a new targeted C3 therapy Empaveli (pegcetacoplan), once called… read more here.
Keywords: paroxysmal nocturnal; nocturnal hemoglobinuria; novel targeted; pegcetacoplan ... See more keywords
Efficacy and Safety Maintained up to 3 Years in Adults with Paroxysmal Nocturnal Hemoglobinuria Receiving Pegcetacoplan
Sign Up to like & getrecommendations! Published in 2025 at "Advances in Therapy"
DOI: 10.1007/s12325-025-03310-8
Abstract: Pegcetacoplan, the first C3 and C3b inhibitor for paroxysmal nocturnal hemoglobinuria (PNH), demonstrated efficacy and safety in C5 inhibitor-experienced and -naive patients in the phase 3 studies PEGASUS (NCT03500549) and PRINCE (NCT04085601), respectively. This integrated… read more here.
Keywords: pegcetacoplan; paroxysmal nocturnal; prince; safety ... See more keywords
Comparative effectiveness of pegcetacoplan versus ravulizumab in patients with paroxysmal nocturnal hemoglobinuria previously treated with eculizumab: a matching-adjusted indirect comparison.
Sign Up to like & getrecommendations! Published in 2021 at "Current medical research and opinion"
DOI: 10.1080/03007995.2021.1971182
Abstract: OBJECTIVE In the absence of a head-to-head study, we assessed the comparative effectiveness of pegcetacoplan, a targeted C3 complement inhibitor, vs. ravulizumab, a C5 complement inhibitor, among patients with paroxysmal nocturnal hemoglobinuria (PNH) previously treated… read more here.
Keywords: treated eculizumab; ravulizumab; pegcetacoplan; comparative effectiveness ... See more keywords
#1467 Pegcetacoplan for post-transplant recurrent C3 glomerulopathy or immune complex membranoproliferative glomerulonephritis in NOBLE: 12-week evolution
Sign Up to like & getrecommendations! Published in 2024 at "Nephrology Dialysis Transplantation"
DOI: 10.1093/ndt/gfae069.442
Abstract: C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) are rare diseases caused by uncontrolled complement activation, resulting in excess deposition of C3 breakdown products in the kidneys, which leads to kidney damage and… read more here.
Keywords: week; post transplant; pegcetacoplan; histology ... See more keywords
Efficacy, Safety, and Quality of Life of Pegcetacoplan in Japanese Patients with Paroxysmal Nocturnal Hemoglobinuria Treated within the Phase 3 PEGASUS Trial
Sign Up to like & getrecommendations! Published in 2024 at "Acta Haematologica"
DOI: 10.1159/000537696
Abstract: Abstract Introduction: Pegcetacoplan, the first approved proximal complement C3 inhibitor, showed superiority to eculizumab in improving hemoglobin levels and clinical outcomes in the phase 3 PEGASUS study in patients with paroxysmal nocturnal hemoglobinuria (PNH) and… read more here.
Keywords: pegcetacoplan; japanese patients; pnh; pegasus ... See more keywords
Safety and efficacy of pegcetacoplan in paroxysmal nocturnal hemoglobinuria
Sign Up to like & getrecommendations! Published in 2022 at "Therapeutic Advances in Hematology"
DOI: 10.1177/20406207221114673
Abstract: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, hematologic disease characterized by complement-mediated hemolysis, thrombosis, and various degrees of bone marrow dysfunction. Until recently, C5 inhibition with eculizumab or ravulizumab represented the only therapies approved… read more here.
Keywords: safety; nocturnal hemoglobinuria; pegcetacoplan; paroxysmal nocturnal ... See more keywords
Real-World Adherence with Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria Compared with Previously Reported Oral Medication Adherence Rates
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DOI: 10.1182/blood-2024-203541
Abstract: Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare and severe hematologic disorder caused by somatic mutation(s) in hematopoietic stem cells, which results in dysregulation of complement activation and complement-mediated hemolysis. Several complement inhibitors are approved… read more here.
Keywords: adherence rates; pegcetacoplan; medication; real world ... See more keywords
Low risk for meningococcal and other encapsulated bacteria infections with systemically administered pegcetacoplan in paroxysmal nocturnal hemoglobinuria and C3 glomerulopathies
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DOI: 10.1182/blood-2025-6719
Abstract: Introduction: Complement inhibitors are promising therapies for complement-mediated conditions, including paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (C3G) and primary immune-complex membranoproliferative glomerulonephritis (IC-MPGN). Clinical benefits of the initially available C5 inhibitors that blocked terminal complement… read more here.
Keywords: pegcetacoplan; meningococcal encapsulated; infection; complement ... See more keywords
Optimizing PNH treatment with the complement inhibitor pegcetacoplan: A case report
Sign Up to like & getrecommendations! Published in 2025 at "Blood"
DOI: 10.1182/blood-2025-6734
Abstract: Introduction: Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, serious hematologic disorder characterized primarily by intravascular hemolysis due to excessive erythrocyte sensitivity to physiological complement C3 activation. Patients with PNH often report symptoms of anemia,… read more here.
Keywords: treatment; pegcetacoplan; pnh treatment; inhibitor ... See more keywords
Management of acute breakthrough hemolysis with intensive pegcetacoplan dosing in patients with PNH
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DOI: 10.1182/bloodadvances.2023011691
Abstract: Key Points • Intensive subcutaneous or IV pegcetacoplan treatment was effective in the management of acute BTH events in patients on pegcetacoplan.• Intensive subcutaneous or IV treatment with pegcetacoplan was safe and well tolerated. read more here.
Keywords: breakthrough hemolysis; pegcetacoplan; acute breakthrough; management acute ... See more keywords