In recent reports on MSN and AdventLS, it was shared that, as a result of the pioneering efforts of Dr. Carla Nester and researchers at the Children’s Hospital of Iowa University, young adults over twelve years of age with C3G now have an approved therapy. IC-MPGN was approved earlier this year by the U.S.FDA as the first treatment for patients 12 years and older with C3G and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), a rare and severe kidney disease that is closely related.
The disease, also known as IC-MPGN, is a rare life-threatening kidney disease. The newly approved therapy was granted approval based on attacking the root cause of the body’s complement system. While previous therapies had been designed to treat C3G by attacking its most harmful inflammatory process, in this instance the opposite occurred as the newly approved therapy was based on its targeting the body’s complement system. The complement system is a key part of the body’s immune response.
About the Trial
Dr. Carla Nester, director of UI Health Care’s Clinic for Renal Diseases and senior author of the VALIANT trial (pegcetacoplan), led the study. The Phase III, randomized, double-blind, placebo-controlled trial was conducted at 122 centers in 19 countries and included 124 patients. Dr. Nester was joined by Dr. Richard Smith, an expert in complement-related kidney diseases and hearing loss.
The team saw a 68% protein reduction in the patients’ urine and stabilization of kidney function. Approximately 67% of the children who were tested attained a complete remission while 72% were clear of disease activity. Pegcetacoplan (Empaveli ) was approved earlier this year by the U.S. Food and Drug Administration (FDA) as the first treatment for patients 12 years and older with C3G and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN), a closely related rare and severe kidney disease.
As a result of the aforementioned combined clinical expertise in glomerular diseases, the Stead Hospital is now one of the few U.S. centers to provide complete care to C3G patients. The research conducted in Smith’s lab over many years has advanced the understanding of the underlying biology of C3G and led to the realization that inhibiting excessive activation of the complement pathway could be the key to effective treatment.
The UI researchers are now collaborating with pharmaceutical companies to develop and test new drugs that could inhibit complement activation. The new system blocks the malfunctioning part of the complement system.
Unlike previous treatments, which relied on broad anti-inflammatory drugs like steroids, or medications that targeted the wrong segment of the complement pathway, this new class of medication precisely blocks the malfunctioning part of the complement system.
Pegcetacoplan is administered by a twice-weekly injection, which most young patients prefer over daily oral medication. Another new drug called iptacopan, was approved earlier this year to treat adults with C3G.
For more information, please refer to the New England Journal of Medicine or the Iowa University Stead Family Children’s Hospital.
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Rose Duesterwald July 6, 2026
