Kidney Week

Abstract: FR-PO0413

Renal Safety of Pegcetacoplan in Paroxysmal Nocturnal Hemoglobinuria (PNH): Findings from Clinical Trials and Global Real-World Experience

Session Information

• AKI: Biomarkers, Diagnostics, and Risk Prediction
  October 23, 2026 | Location: Exhibit Hall A, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Acute Kidney Injury

• 102 AKI: Clinical, Outcomes, and Trials

Authors

• Mastrangelo, Antonio, Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico, Milan, Lombardy, Italy

Antonio Mastrangelo

• Ariceta Iraola, María Gema, Hospital Universitari Vall d'Hebron, Barcelona, CT, Spain

María Gema Ariceta Iraola, MD, PhD

• Nester, Carla M., The University of Iowa Stead Family Children's Hospital, Iowa City, Iowa, United States

Carla M. Nester, MD, MS, FASN

• Mukherjee, Anwesha, Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States

Anwesha Mukherjee, PhD

• Hillmen, Peter, Apellis Pharmaceuticals Inc, Waltham, Massachusetts, United States

Peter Hillmen, MBChB, PhD

• Szamosi, Johan, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden

Johan Szamosi, MSc

• Franzén, Liza, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden

Liza Franzén

• Taliadouros, Ginny, Swedish Orphan Biovitrum AB publ, Stockholm, Stockholm County, Sweden

Ginny Taliadouros

• de Castro, Carlos M., Duke University, Durham, North Carolina, United States

Carlos M. de Castro, DrMed

• Peffault de Latour, Regis, French Reference Center for Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria, Paris, France

Regis Peffault de Latour, MD

Background

Paroxysmal nocturnal hemoglobinuria (PNH) is a complement-mediated hematologic disorder that may be associated with renal morbidity. Both acute kidney injury (AKI) and chronic kidney disease (CKD) may occur in PNH patients as consequences of chronic and acute hemolysis, iron deposition, microvascular thrombosis, and other factors.
Pegcetacoplan, a targeted C3/C3b inhibitor, demonstrated sustained hematologic and clinical benefits in the Phase 3 PEGASUS and PRINCE studies in patients with PNH, with long-term follow-up from their extension study supporting maintained efficacy and a favorable safety profile.
This analysis evaluated renal safety outcomes observed in PEGASUS, PRINCE, their extension study, and in real-world post-marketing data.

Methods

Adult PNH patients received pegcetacoplan 1,080 mg via subcutaneous infusion twice weekly. The combined safety set of included clinical trials was used to assess estimated glomerular filtration rate (eGFR) over time and adverse events (AEs) under the high-level term of renal failure and impairment. Safety reporting from post-marketing data of real-world pegcetacoplan use was assessed for serious renal AEs.

Results

132 patients were included in the clinical trial safety set. Mean eGFR remained stable over up to 312 weeks follow-up, with no evidence of progressive kidney function decline (Figure).
Renal AEs were infrequent, reported in only 12.9% of patients, the most common being new diagnoses of AKI (6.8%) and CKD (2.3%). Serious AKI events were typically secondary to PNH (e.g. due to hemolysis) or consistent with known complications of PNH (e.g. infection, dehydration) and resolved with treatment.
Serious renal AEs reported in the post-marketing setting are consistent with those observed in clinical trials, predominantly not related or unlikely related to pegcetacoplan, and resolved without dose change.

Conclusion

Across clinical trials and in the real world, reported renal AEs were consistent with PNH and typical intercurrent illnesses, with no pattern identified over long-term follow-up. eGFR remained stable over up to 6 years in clinical trials, with no evidence of progressive kidney function decline.

Funding

• Commercial Support – Swedish Orphan Biovitrum AB (Sobi) and Apellis Pharmaceuticals, Inc.