Abstract: FR-PO0854
Real-World Treatment Patterns and Clinical Response Among US Patients with C3 Glomerulopathy (C3G) Receiving Iptacopan: APPRISE-C3G Data Platform
Session Information
- Glomerular Diseases: Practice and New Concepts Shaping Modern Care
October 23, 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
- Narayanan, Mohanram, Baylor Scott & White Health, Dallas, Texas, United States
- Ndife, Briana C., Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States
- Trenz, Helen, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States
- Bose, Anirban, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States
- Stephens, Neema, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, United States
- Buchan, Claire, Asclepius Analytics, New York, New York, United States
- Buchan, Tayler, Asclepius Analytics, New York, New York, United States
- Baraka, Eugene, Asclepius Analytics, New York, New York, United States
- Gargano, Peter, Oncology Pharmacy, Portland, Oregon, United States
- Canetta, Pietro A., Columbia University, New York, New York, United States
Background
C3G is an ultra-rare, progressive kidney disease caused by overactivation of the alternative complement pathway. Real-world data characterizing early clinical response to complement inhibition in patients (pts) with C3G are limited. Iptacopan is a highly selective, oral factor B inhibitor that was FDA-approved for the treatment of adults with C3G, to reduce proteinuria, in Mar 2025. A Patient Platform for Real-world data on Iptacopan in the United StatEs for pts with C3G (APPRISE-C3G) is a platform capturing data on US adults with C3G prescribed iptacopan. This interim analysis describes real-world treatment (tx) patterns and early proteinuria response in pts enrolled in APPRISE-C3G to date.
Methods
Included pts provided electronic consent for enrollment (Sep 2024 to Mar 2026). Data were sourced from specialty pharmacy and medical records. Tx duration was from iptacopan initiation (index) to first instance of discontinuation or data cutoff. Tx adherence was assessed by proportion of days covered (PDC). Clinical response was assessed as change in urine protein-creatinine ratio (UPCR) from pre-index value.
Results
Among 27 pts enrolled, median (interquartile range [IQR]) age was 27.6 (24.4 to 42.5) years, 44% were male, and 67% had commercial insurance; 21 of 24 pts with clinical data had a confirmatory biopsy. Median (IQR) iptacopan tx duration was 7.0 (3.0 to 8.7) months; median (IQR) PDC was 95.8% (88.4% to 99.1%). At data cutoff, 4 pts had discontinued therapy. All pts with clinical data had prior tx (any therapy) at index. Five pts had prior kidney transplant, 4 of whom had disease recurrence before index. Median (IQR) pre-index UPCR (n=21) was 1.9 (1.3 to 3.4) g/g and median (IQR) pre-index eGFR slope (n=21) was −4.3 (−16.0 to −1.0) mL/min/1.73 m2/year. Early clinical response was favorable (Table).
Conclusion
Tx-experienced patients with C3G receiving iptacopan demonstrated high tx adherence; data trended toward early proteinuria improvement with iptacopan in routine practice. Enrollment is ongoing and additional data will be available in the near future.
Acknowledgment
Medical writing support and editorial support were provided by Elizabeth Murray, PhD (BOLDSCIENCE Ltd., UK), and were funded by Novartis Pharmaceuticals Corporation. This abstract was developed in accordance with Good Publication Practice (GPP) guidelines. The authors had full control of the content and made the final decision on all aspects of this publication.
Funding
- Commercial Support – Novartis Pharmaceuticals Corporation