Kidney Week

Abstract: SA-PO0811

Iptacopan Treatment in Patients with C3 Glomerulopathy (C3G): 12-Month Results from the Early Access Program

Session Information

• Glomerular Management: Real-World Lessons and Emerging Therapies
  November 08, 2025 | Location: Exhibit Hall, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Glomerular Diseases

• 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics

Authors

• Biancone, Luigi, Division of Nephrology Dialysis and Transplantation, Città Della Salute e Della Scienza Hospital, Turin, Italy

Luigi Biancone, MD, PhD

• Nester, Carla M., Division of Pediatric Nephrology, Stead Family Children’s Hospital, Iowa City, Iowa, United States

Carla M. Nester, MD, MS, FASN

• Blasco Pelicano, Josep Miquel, Department of Nephrology and Renal Transplantation, Nephrology and Urology Institute Hospital Clínic, Barcelona, Spain

Josep Miquel Blasco Pelicano, MD, PhD

• Cavero, Teresa, Department of Nephrology, Hospital Universitario 12 de Octubre, Madrid, Spain

Teresa Cavero, PhD

• Ardissino, Gianluigi, Center for Hemolytic Uremic Syndrome (HUS) Prevention, Ospedale Maggiore Policlinico, Milan, Italy

Gianluigi Ardissino, MD, PhD

• Ansari, Soudeh, Novartis Pharmaceuticals Corporation, Cambridge, Massachusetts, United States

Soudeh Ansari, PhD

• Rizk, Sviatlana, Novartis Pharma AG, Basel, Switzerland

Sviatlana Rizk, PhD

• Chopra, Roohi, Novartis Farmaceutica, S.A., Barcelona, Spain

Roohi Chopra, MD, MBBS

• Smeets, Serge, Novartis Pharma AG, Basel, Switzerland

Serge Smeets, PhD

Background

C3 glomerulopathy (C3G) is an ultra-rare, progressive kidney disease caused by overactivation of the alternative complement pathway (AP). Around 50% of affected patients develop kidney failure within 10 years of diagnosis. Iptacopan is an oral proximal complement inhibitor that targets factor B to selectively inhibit the AP. Here, we report 12-month results of iptacopan treatment through the early access program in patients with native and recurrent C3G post-transplantation who are not eligible or able to participate in clinical trials.

Methods

Iptacopan 200 mg twice daily was provided on approval of an unsolicited request. Resupply requests were typically made every 3 months and estimated glomerular filtration rate (eGFR) values collected.

Results

At baseline the mean (SD) eGFR was 69.18 mL/min/1.73m² (34.22) and 41.45 mL/min/1.73m² (15.95) for native (n=93) and recurrent (n=35) C3G patients, respectively. eGFR data evaluable for change analysis were available for 26 patients with native and 11 with recurrent C3G. Over the 12 months prior to iptacopan treatment, native patients showed an eGFR slope of -10.7 mL/min/1.73m²/year (95% CI: -17.6, -3.8) compared with -23.0 mL/min/1.73m²/year (95% CI: -28.4, -17.6) in the recurrent patients. After 12 months of iptacopan treatment, the eGFR slope was -4.7 mL/min/1.73m²/year (change in slope +6.0 mL/min/1.73m²/year; 95% CI: -8.8, 20.7; p=0.411) in native patients and -0.1 mL/min/1.73m²/year (change in slope +22.9 mL/min/1.73m²/year; 95% CI: 11.2, 34.6; p=0.001) in recurrent patients (Figure).

Conclusion

In the early access program, 12 months of iptacopan treatment resulted in slowing of disease progression in native and recurrent patients with C3G.

Funding

• Commercial Support – Novartis Pharma AG, Basel, Switzerland

Digital Object Identifier (DOI)

• doi: 10.1681/ASN.2025tptpa76d