FDA approval for oral Fabhalta® (iptacopan) - the first and only treatment approved in C3 glomerulopathy

发布时间： 2025-03-28 阅读：31次

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On March 22, 2025--Novartis today announced that oral Fabhalta® (iptacopan) has received U.S. Food and Drug Administration (FDA) approval for the treatment of adults with C3 glomerulopathy (C3G), to reduce proteinuria, making it the first and only treatment approved for this condition.

C3G is an ultra-rare kidney disease typically diagnosed in young adults and often progresses to kidney failure. Fabhalta, an oral Factor B inhibitor of the alternative complement pathway, selectively targets the underlying cause of C3G. Before the approval of Fabhalta, patients had to rely on supportive care, broad immunosuppression, and symptom management.

This FDA approval is based on results from the pivotal Phase III APPEAR-C3G study. Patients treated with Fabhalta in addition to supportive care achieved a 35.1% reduction in proteinuria (as measured by 24-hour urine protein to creatinine ratio [UPCR]) at 6 months when compared to placebo on top of supportive care1. In many kidney diseases, proteinuria reduction is an increasingly recognized surrogate marker correlating with delaying progression to kidney failure.