IPTACOPAN TREATMENT IN PATIENTS WITH C3 GLOMERULOPATHY (C3G): 12-MONTH RESULTS FROM THE MANAGED ACCESS PROGRAM

 

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IPTACOPAN TREATMENT IN PATIENTS WITH C3 GLOMERULOPATHY (C3G): 12-MONTH RESULTS FROM THE MANAGED ACCESS PROGRAM

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Carla M
Nester
Luigi Biancone luigi.biancone@gmail.com Città Della Salute e Della Scienza Hospital Division of Nephrology Dialysis and Transplantation Turin Italy -
Carla M Nester cnester@uiowa.edu Stead Family Children’s Hospital Division of Pediatric Nephrology Iowa City United States *
Miquel Blasco MIBLASCO@clinic.cat Nephrology and Urology Institute Hospital Clínic Department of Nephrology and Renal Transplantation Barcelona Spain -
Teresa Cavero tcaveroescribano@gmail.com Hospital Universitario 12 de Octubre Department of Nephrology Madrid Spain -
Gianluigi Ardissino ardissino@centroseu.org Ospedale Maggiore Policlinico Center for Hemolytic Uremic Syndrome (HUS) Prevention Milan Italy -
Soudeh Ansari soudeh.ansari@novartis.com Novartis Pharmaceuticals Corporation Global Drug Development Cambridge United States -
Sviatlana Rizk sviatlana.rizk@novartis.com Novartis Pharma AG Global Drug Development Basel Switzerland -
Roohi Chopra roohi.chopra@novartis.com Novartis Farmaceutica S.A. Global Drug Development Barcelona Spain -
Serge Smeets serge.smeets@novartis.com Novartis Pharma AG Global Drug Development Basel Switzerland -
 
 
 
 
 
 

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). 

Kewords