Novartis has received US FDA accelerated approval for its Fabhalta, a medication designed to alleviate proteinuria in adults diagnosed with primary immunoglobulin A nephropathy (IgAN), a rapidly progressive disease. The pricing details were not disclosed.

Fabhalta is an innovative treatment that inhibits the alternative complement pathway of the immune system that contributes to the pathogenesis of IgAN. This indication is granted under accelerated approval based on the prespecified interim analysis of the Phase III APPLAUSE-IgAN study, which measured a reduction in proteinuria at nine months compared to placebo. However, it has not been established whether Fabhalta slows kidney function decline in patients with IgAN. 

Continued approval of Fabhalta may depend on verification and description of clinical benefits from the ongoing Phase III APPLAUSE-IgAN study. This study evaluates whether Fabhalta slows disease progression as measured by the decline in estimated glomerular filtration rate (eGFR) over 24 months. The eGFR data, expected upon study completion in 2025, are intended to support traditional FDA approval.

Analyst QuickTake: In May 2024 , Novartis presented results from the six-month, double-blind period of the Phase III APPEAR-C3G study of Fabhalta (iptacopan) at the late-breaking clinical trials session of the European Renal Association (ERA) congress. Patients treated with Fabhalta, in addition to supportive care, achieved a 35.1% reduction in proteinuria (measured by the 24-hour urine protein to creatinine ratio [UPCR]) at six months, compared to placebo with supportive care. In many kidney diseases, reducing proteinuria was increasingly recognized as a surrogate marker for delaying progression to kidney failure.