EU Experts Recommend Iptacopan for C3 Glomerulopathy Treatment

C3 glomerulopathy (C3G) is characterized by the buildup of C3 and its fragments within the kidney's glomeruli, leading to inflammation and potential kidney damage. This condition, similar to paroxysmal nocturnal hemoglobinuria (PNH), stems from a dysregulation of the alternative complement pathway in the immune system. Patients typically exhibit symptoms such as hematuria, proteinuria, and hypertension. Alarmingly, around 50% of individuals diagnosed with C3G may experience kidney failure within a decade, necessitating lifelong dialysis or even kidney transplantation.

Currently, there are no approved targeted treatments for C3G. However, this situation may soon change with Iptacopan, an oral Factor B inhibitor that has been under investigation for complement-mediated disorders, including both PNH and C3G. Iptacopan was introduced to the German market for PNH treatment in autumn 2024.

The European Commission is now evaluating a new recommendation from experts at the European Medicines Agency (EMA) regarding the use of Iptacopan for adult patients with C3G. The recommendation suggests that Iptacopan could be administered in conjunction with a Renin-Angiotensin-System (RAS) inhibitor, or as an alternative for patients who cannot tolerate RAS inhibitors or for whom these inhibitors are contraindicated.

This recommendation is based on findings from the APPEAR-C3G study, which demonstrated that patients receiving Iptacopan alongside supportive therapy experienced a significant reduction in proteinuria, as well as stabilization and numerical improvement in their estimated glomerular filtration rate (eGFR). The European Commission is expected to announce its decision regarding the approval within 67 days.