University of Iowa

Novartis presented 12-month APPEAR-C3G study data at ASN Kidney Week 2024 showing Fabhalta (iptacopan) sustained clinically meaningful proteinuria reduction and eGFR improvement in C3G patients, with a favorable safety profile. Fabhalta, an oral complement inhibitor, could be the first FDA-approved treatment for C3G, with regulatory submissions completed in the EU, China, and Japan, and expected in the US by year-end.

By Brooklyn Draisey, Iowa Capital Dispatch, Higher Ed, Oct. 25, 2024 7:38 am.

The APPEAR-C3G trial's 12-month data show sustained proteinuria reduction with iptacopan in C3 glomerulopathy (C3G), presented at ASN Kidney Week 2024. Iptacopan, an oral Factor B inhibitor, met its primary endpoint of a significant reduction in 24-hour urine protein-to-creatinine ratio (UPCR), with a 35.1% reduction sustained over 12 months. Novartis has completed regulatory submissions for iptacopan in the EU, China, Japan, and expects US approval by year-end.

Novartis presents 12-month APPEAR-C3G data showing Fabhalta sustained proteinuria reduction, improved eGFR slope, and favorable safety profile in C3G patients, positioning it as the only oral alternative complement pathway inhibitor for this ultra-rare kidney disease.

VALIANT trial results show pegcetacoplan (Empaveli) significantly reduces proteinuria, C3c staining, and stabilizes eGFR in C3G and IC-MPGN patients, with favorable safety profiles.

ASN Kidney Week 2024 will present results from high-impact phase 3 trials on hyponatremia correction, pegcetacoplan efficacy, semaglutide benefits, pain coping skills training, acellular tissue engineered vessel use, and dapagliflozin in advanced CKD.

Pegcetacoplan (Empaveli) demonstrated significant benefits in proteinuria, C3c staining, and eGFR stabilization in C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN) patients, according to the VALIANT trial. The trial, the largest in these populations, included adolescents and post-transplant patients, showing consistent efficacy and safety across subgroups. Sobi and Apellis plan to file a supplemental New Drug Application with the FDA in early 2025.

Novartis presents 12-month APPEAR-C3G data showing Fabhalta sustained proteinuria reduction in C3G patients, improved eGFR slope, and favorable safety profile. Fabhalta is the only oral alternative complement pathway inhibitor for C3G, with regulatory submissions completed in EU, China, and Japan, and expected in the US by year-end.