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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. This study reports the 12-month results of iptacopan treatment through the managed access program in patients with native and recurrent C3G post-transplantation, who are not eligible or able to participate in clinical trials.
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 were collected.
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 C3G 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% Cl: −17.6, −3.8) compared with −23.0 mL/min/1.73m2/year (95% Cl: −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% Cl: −8.8, 20.7; p=0.411) in native patients and −0.1 mL/min/1.73m2/year (improvement in slope +22.9 mL/min/1.73m2/year; 95% Cl: 11.2, 34.6; p=0.001) in recurrent patients (Figure).
In the managed access program, 12 months of iptacopan treatment resulted in the slowing of disease progression in native and recurrent patients with C3G.
This abstract was also submitted to the American Society of Nephrology (ASN) Kidney Week 2025 Congress, November 6-9, in Houston, TX, USA. Encore submission of this abstract to WCN 2026 is permitted by the organizers of the original meeting (ASN 2025).