Pegcetacoplan's Approval and Shifting C3G Landscape, with Sayna Norouzi, MD

After years of limited treatment options, the care landscape for C3 glomerulopathy (C3G) is undergoing a drastic transformation. Once defined by off-label therapies and uncertainty, the rare kidney disease obtained its second US Food and Drug Administration-approved treatment in July 2025 in the form of pegcetacoplan (Empaveli).

In March 2025, iptacopan (Fabhalta) became the first FDA-approved treatment for C3G, offering long-awaited hope to patients and providers. Now, with the approval of pegcetacoplan, 2 approved therapies are available—an unprecedented shift for a rare kidney disease that had long relied on off-label or trial-and-error approaches.

“For years and years, patients with C3G came to our clinics and we didn't have any specific FDA-approved medications for them,” Sayna Norouzi, MD, an assistant professor of medicine, clinical nephrologist, and founder and director of the glomerular disease and polycystic kidney disease clinics at Loma Linda University Medical Center, explained to HCPLive. “We were treating them with medications that we were hoping would help control the disease, control the proteinuria, but we didn't have precise clinical trial results for any of those medications.”

Now, with 2 FDA-approved options, she says clinicians can have informed, hopeful discussions with their patients about the treatment options available to them. However, Norouzi cautions that this new era brings more than hope—it brings complexity.

“Having access to 2 really good medications as treatment options for our patients with C3G is exciting, but at the same time, it's bringing in a lot of responsibility on our side that we have to keep ourselves updated and navigate the treatment options for our patients in a really smart way to make sure that they are receiving the medication that is appropriate for the kind of disease that they're dealing with, for the situation that they are dealing with, because all patients are different,” Norouzi explained.

While these approvals mark major progress, she notes the need for long-term safety data, post-marketing experience, and future trials—including combination strategies—to optimize care.

Editors’ note: Norouzi has relevant disclosures with Calliditas, Novartis, Otsuka, and Boehringer Ingelheim.

References

1. Brooks A. FDA Approves Pegcetacoplan (Empaveli) for C3 Glomerulopathy, IC-MPGN. HCPLive. July 28, 2025. Accessed July 31, 2025. https://www.hcplive.com/view/fda-approves-pegcetacoplan-empaveli-for-c3-glomerulopathy-ic-mpgn

2. Campbell P. FDA Approves Oral Iptacopan (Fabhalta) as First C3 Glomerulopathy Therapy. HCPLive. March 20, 2025. Accessed July 31, 2025. https://www.hcplive.com/view/fda-approves-oral-iptacopan-fabhalta-as-first-c3-glomerulopathy-therapy