Kidney Week

Abstract: SA-OR078

Sustained Efficacy and Safety in C3 Glomerulopathy Following 36 Months of Oral Treatment with Iptacopan: Interim Participant Results from the APPEAR-C3G Extension Study

Session Information

• Late-Breaking Research Orals: Pediatrics, Genetics, and Glomerular Diseases
  October 24, 2026 | Location: Four Seasons Ballroom 2, Convention Center
  Abstract Time: 05:30 PM - 05:42 PM

Category: Glomerular Diseases

• 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics

Authors

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

Carla M. Nester, MD, MS, FASN

• Smith, Richard J., Carver College of Medicine, University of Iowa, Iowa City, Iowa, United States

Richard J. Smith, MD

• Kavanagh, David, National Renal Complement Therapeutics Centre, Royal Victoria Infirmary, Newcastle upon Tyne, England, United Kingdom

David Kavanagh, MBChB, PhD

• Vivarelli, Marina, Ospedale Pediatrico Bambino Gesu IRCCS, Rome, Lazio, Italy

Marina Vivarelli, MD

• Remuzzi, Giuseppe, Istituto di Ricerche Farmacologiche Mario Negri IRCCS, Bergamo, Italy

Giuseppe Remuzzi, MD

• Zhao, Minghui, Peking University First Hospital, Beijing, China

Minghui Zhao, PhD

• Wong, Edwin Kwan Soon, National Renal Complement Therapeutics Centre, Royal Victoria Infirmary, Newcastle upon Tyne, England, United Kingdom

Edwin Kwan Soon Wong, MBChB, PhD

• Cao, Shiwei, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States

Shiwei Cao

• Israni, Rubeen K., Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States

Rubeen K. Israni, MD, MPH

• Luna Yolba, Raquel, Novartis Pharma AG, Basel, BS, Switzerland

Raquel Luna Yolba, PhD

• Jacinto-Sanders, Severina, Novartis Pharma AG, Basel, BS, Switzerland

Severina Jacinto-Sanders, PhD

• Grisouard, Jean, Novartis Pharma AG, Basel, BS, Switzerland

Jean Grisouard, PhD

• Bomback, Andrew S., David Koch Jr Glomerular Kidney Center, Columbia University Irving Medical Center, New York, New York, United States

Andrew S. Bomback, MD, MPH

Background

The Phase 3 open-label extension study evaluates iptacopan long-term efficacy and safety in participants with C3 glomerulopathy (C3G). Here we report the 36-month (M) follow-up data from APPEAR-C3G participants.

Methods

Participants with C3G received oral iptacopan 200 mg twice-daily (b.i.d.) for up to 12M in the Phase 3 APPEAR-C3G (NCT04817618) study, before entering the open-label extension study (NCT03955445). Long-term efficacy is assessed by change in proteinuria and eGFR based on summary statistics. Safety and tolerability of iptacopan is continually monitored.

Results

Of 73 participants completing the 12M APPEAR-C3G study, 66 entered the extension study for treatment with iptacopan 200 mg b.i.d; 57 completed the 36M follow-up (12M plus 24M extension). There was a reduction of 45% (95% CI: 23%, 61%) from baseline in FMV UPCR (n=45, Figure) and a mean (SD) change of –0.63 (13.05) mL/min/1.73m² from baseline in eGFR (n=47) after 36M treatment with iptacopan. Iptacopan demonstrated a safety profile consistent with studies across other indications with no new safety findings and no meningococcal infections reported in clinical trials with C3G participants.

Conclusion

This analysis confirms previous findings demonstrating sustained efficacy and safety of oral iptacopan over 3 years. To date, this is the largest cohort with long-term follow up data in participants with C3G receiving iptacopan in clinical trials, addressing a gap that may inform clinical practice in this population.

Acknowledgment

Professional medical writing assistance was provided by Carol Crawford (Novartis Ireland Ltd.) and funded by Novartis Pharma AG.

Mean (95% CI) percentage change from iptacopan (200 mg b.i.d.) baseline in FMV UPCR (g/g) over time up to 36 months.

Funding

• Commercial Support – Novartis Pharma AG