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Iptacopan Treatment in Patients with C3 Glomerulopathy (C3G): 12-Month Results from the Early Access Program: SA-PO0811
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Iptacopan Treatment in Patients with C3 Glomerulopathy (C3G): 12-Month Results from the Early Access Program: SA-PO0811

Luigi Biancone, Carla M. Nester, Josep Miquel Blasco Pelicano, Teresa Cavero, Gianluigi Ardissino, Soudeh Ansari, Sviatlana Rizk, Roohi Chopra and Serge Smeets
Journal of the American Society of Nephrology, Vol.36(10S), 10.1681/ASN.2025tptpa76d
10/2025
DOI: 10.1681/ASN.2025tptpa76d

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Abstract

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.

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