A rare and life-threatening kidney disease affecting young adults and children finally has an effective and approved therapy due to the pioneering and leadership of researchers at the Iowa University’s Children’s Hospital.
Unlike previous treatments for C3G that focused on alleviating the most damaging inflammatory process of the disease, Pegdetacoplan directly targets the root cause found in the body’s complement system which is defined as a vital part of an immune response.
About the Trial
Dr. Carla Nester the director of UI Health Care’s Clinic for Renal Diseases led the trial investigating Pegcetacoplan. The end result was a 68% reduction of protein in patients’ urine with kidney function stabilization.
Approximately 67% of the children tested attained a complete remission while 72% were clear of disease activity. The Phase III, randomized, double-blind, placebo-controlled trial reported by MedicalExpress, was conducted at 122 centers in 19 countries and involved 124 patients.
About the Results
The FDA had approved Pegcetacoplan earlier in 2025. It is the first treatment for patients twelve years old or older diagnosed with C3G and closely related IC-MPGN kidney disease.
The latest advance is based on research involving the complement system under the direction of Dr. Richard Smith, professor of pediatrics at Iowa University and well established as an expert in complement-related kidney diseases.
The team at Iowa University has earned the trust of people affected by C3G who are willing to travel from all over the world to receive care at Iowa University’s Renal Disease Center.
It was the research conducted at Dr. Smith’s lab suggesting that activation of the complement pathway may lead to effective treatment. This motivated the UI researchers to convert this knowledge into therapies that they tested and confirmed would inhibit complement activation.
Previous treatments relied on anti-inflammatory drugs or even drugs that focused incorrectly on the complement pathway. Now the new medication blocks malfunctioning parts of the complement system.
Iptacopan, medicine approved earlier this year to treat adults diagnosed with C3G also inhibits the complement system. The impact on patients is profound. Dr. Nester ended the interview by saying that it was a long road but “we’re here”.
