Kidney Week

Abstract: SA-PO0674

Long-Term Managed Access Program (MAP) Data Confirm Sustained eGFR Stabilization with Iptacopan up to 24 Months in C3 Glomerulopathy (C3G)

Session Information

• Glomerular Diseases: Clinical, Outcomes, and Therapeutics Research - Other
  October 24, 2026 | Location: Exhibit Hall A, Convention Center
  Abstract Time: 10:00 AM - 12:00 PM

Category: Glomerular Diseases

• 1402 Glomerular Diseases: Clinical, Outcomes, and Therapeutics

Authors

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

Carla M. Nester, MD, MS, FASN

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

Luigi Biancone, MD, PhD

• Blasco Pelicano, Josep Miquel, Department of Nephrology and Renal Transplantation, Nephrology and Urology Institute Hospital Clinic, 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

• Raha, Sohini, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States

Sohini Raha, PhD

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

Sviatlana Rizk, PhD

• Kamboj, Shantanu, Novartis Healthcare Pvt. Ltd., Hyderabad, India

Shantanu Kamboj, MD

• Khan, Abdul Faheem, Novartis Healthcare Pvt. Ltd., Hyderabad, India

Abdul Faheem Khan, MD, MBBS

• Aldea, Anna, Novartis Farmaceutica SA, Barcelona, CT, Spain

Anna Aldea

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

Serge Smeets, PhD

Background

C3G is an ultra-rare, progressive kidney disease caused by overactivation of the alternative complement pathway (AP). Up to 50% of patients (pts) with C3G develop kidney failure within 10 years (yrs). Iptacopan is an oral, selective complement factor B inhibitor that targets AP overactivation. The iptacopan MAP established by Novartis included adult pts with native and recurrent C3G not eligible for clinical trials. A previous analysis at 12 months (mo) on a similar cohort showed stabilization of estimated glomerular filtration rate (eGFR) slope (ASN 2025), compared to the historical rapid eGFR decline. This analysis aimed to confirm the 12-mo data with a larger patient number and to present longer term effectiveness and safety for up to 24 mo.

Methods

Iptacopan 200 mg b.i.d. was provided in response to unsolicited requests from physicians and resupplied every 3 mo upon request. eGFR data were collected 12, 6 and 3 mo prior to and every 3 mo up to 24 mo after iptacopan initiation. A longitudinal mixed-effects model was used to estimate pre-treatment eGFR slope and slope change after iptacopan initiation.

Results

87 pts (native: n=65; recurrent: n=22) were evaluable for sensitivity eGFR slope analysis. Following iptacopan initiation, eGFR trajectories stabilized in both cohorts from a steep negative pre-treatment slope at both 12 and 24 mo. At 12 and 24 mo, the change in eGFR slope was +6.89 (95%CI: -1.24, 15.02; p=0.0951) and +7.10 (95%CI: -0.39, 14.60; p=0.0629) mL/min/1.73m²/yr in native C3G, and +18.85 (95%CI: 11.35, 26.36; <0.0001) and +21.07 (95%CI: 14.99, 27.14; p<0.0001) mL/min/1.73m²/yr in recurrent C3G, respectively (Table). During the 24-mo period, 114 adverse events (AEs) in 39 native C3G pts and 73 in 15 recurrent C3G pts were reported; most AEs were non-serious.

Conclusion

This long-term MAP data corroborates the benefit of iptacopan treatment demonstrated in the clinical trials, showing sustained eGFR stabilization in pts with native and recurrent C3G up to 24 mo in the real world. Iptacopan was well tolerated with no new safety signals identified.

Acknowledgment

Dyuti Coomar (Novartis, UK) performed the statistical analysis of the data. Roohi Chopra (Novartis, Spain) contributed to the analysis, interpretation, and review of the data. Rajeeb Ghosh (Novartis, India) provided medical writing support, funded by Novartis Pharma AG.

eGFR slope (mL/min/1.73 m2/yr)

Funding

• Commercial Support – Novartis Pharma AG