Year: 2025
Type: article
Abstract: ews that the US Food and Drug Administration (FDA) approved iptacopan in late March to reduce proteinuria in patients with C3 glomerulopathy (C3G) marked a watershed moment for patients with the condition and their nephrologists (1).It is the first drug ever approved to treat the rare condition."C3G is a debilitating disease often affecting young people, impacting many aspects of their physical and emotional health, and our previous treatment options came with significant challenges," said Carla Nester, MD, MS, FASN, professor of pediatrics-nephrology at The University of Iowa in Iowa City, in a statement from the drug's manufacturer, Novartis (1)."This approval of [iptacopan] is historic for the entire C3G community, as now, for the first time, we have a therapy that is believed to treat the underlying cause of the disease, providing the potential for a new standard of care for patients," said Nester, who was the principal investigator on the phase 3 trial of the drug.Iptacopan is an oral medication that helped reduce proteinuria by approximately one-third of patients with C3G in the phase 3 study reported by Novartis (2).It directly targets factor 3, which is part of a feedback loop in the alternative complement pathway and, in doing so, stops a buildup of C3 in the glomeruli that leads to damage and progressive kidney disease, often leading to kidney failure within 10 years.Previously, the standard treatment was steroids and mycophenolate, which often failed to provide relief and was associated with adverse effects, noted Matthew Sparks, MD, FASN, associate professor and director of the Nephrology Fellowship Program at Duke University School of Medicine in Durham, NC.When those options failed, he explained, clinicians would then move on to an infusion therapy like eculizumab, which required regular infusions and did not have much evidence supporting a benefit.The availability of a twice-daily oral medication that reduces
Source: Kidney News
Author: Bridget M. Kuehn
Language: English
Subfield: Immunology
Field: Immunology and Microbiology
Domain: Life Sciences
Open Access status: bronze
OpenAlex ID: W4411079792