Early Insights Into Characteristics and Treatment Patterns of US Patients With Complement 3 Glomerulopathy Who Were Prescribed Iptacopan: The APPRISE-C3G Data Platform

Iptacopan, an oral, first-in-class, potent alternative complement pathway factor B inhibitor, received US Food and Drug Administration (FDA) approval in March 2025 to reduce proteinuria in adults with complement 3 glomerulopathy (C3G). A Patient Platform for Real-world data on Iptacopan in the United StatEs for patients with C3G (APPRISE-C3G) is a data platform capturing real-world data on US patients with C3G treated with iptacopan. This interim analysis provides early insights into demographic and clinical characteristics and treatment patterns of patients enrolled in APPRISE-C3G to date. 

The APPRISE data platform captures retrospective, longitudinal, deidentified patient-level data from patients with C3G, immunoglobulin A nephropathy (IgAN), or paroxysmal nocturnal hemoglobinuria treated with iptacopan and/or atrasentan. Renal cohorts began enrollment in August 2024 with FDA approval of iptacopan for IgAN. APPRISE-C3G comprises adults with C3G who are treated with iptacopan and provide electronic consent for enrollment. Data cutoff for this analysis was September 2025. Patient demographic and clinical characteristics and treatment pattern data were collected from US specialty pharmacy and medical records. Therapy duration was calculated from the date of iptacopan initiation (index) to the first instance of discontinuation or data cutoff. Treatment adherence was assessed by the proportion of days covered (PDC; ratio of days with supply to total days in the period) and medication possession ratio (MPR; total days’ supply to total days in the period). 

As of data cutoff, 15 patients were enrolled in APPRISE-C3G. At index, median (interquartile range [IQR]) patient age was 26.3 (24.6–42.5) years, 47% were male, and the most common race was White (40%). Nearly all (87%) patients had received ≥1 prior treatment for C3G; corticosteroids (40%) were the most common of the agents assessed. Median (IQR) duration of iptacopan therapy at time of data cutoff was 3.0 (2.9–3.0) months. By data cutoff, 2 patients had discontinued treatment due to provider decision and dialysis initiation, and 1 patient had paused treatment due to provider switch. Median (IQR) PDC and MPR were 99% (93%–100%) and 130% (105%–135%), respectively. Among the 8 (53%) patients with C3G with clinical data available at time of analysis (C3 glomerulonephritis, n=4; dense deposit disease, n=0; mixed, n=1; unknown subtype, n=3), median (IQR) time since initial diagnosis was 2.4 (1.6–6.1) years. Two patients had a history of kidney transplant, 1 of whom had post-transplant C3G recurrence. Median (IQR) latest reported pre-index estimated glomerular filtration rate (eGFR; n=7) was 26.8 (18.2–30.2) mL/min/1.73 m2 and median (IQR) pre-index eGFR slope was −8.4 (−22.2–−5.3) mL/min/1.73 m2/year. Median (IQR) latest reported pre-index urine protein-to-creatinine ratio (n=7) was 4.2 (2.7–7.3) g/g.

This interim analysis of APPRISE-C3G provides first insights into characteristics and treatment patterns of US patients with C3G prescribed iptacopan in a real-world setting. Recruitment for APPRISE-C3G is ongoing and will provide further valuable information about this patient population over time.