CHMP Endorses Fabhalta for C3G

Novartis announced  that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion and recommended granting a marketing authorization for Fabhalta  (iptacopan) – a first-in-class oral Factor B inhibitor of the alternative complement pathway – for the treatment of adults with C3 glomerulopathy (C3G)

There are currently no approved treatments for patients living with C3G, a progressive ultra-rare kidney disease, which often strikes when people are young. The prognosis for people living with C3G is poor, with around half of patients progressing to kidney failure within 10 years of being diagnosed, at which point they require lifelong dialysis and/or kidney transplantation^{2-5, 17}.

“C3G is a debilitating condition, often affecting young people and severely impacting their physical and mental health,” said Marianne Silkjær Nielsen, Founder of CompCure, a Danish non-profit association committed to improving outcomes for individuals with C3G and immune complex membranoproliferative glomerulonephritis (IC-MPGN). “Screening to secure timely diagnosis and access to targeted treatments are critical for patients, their families and society. This milestone is highly welcomed by the patient community, marking progress toward better patient care for people living with C3G.”

The CHMP’s opinion is based on robust data from APPEAR-C3G, the first randomized, placebo-controlled Phase III study in C3G. The study showed patients treated with Fabhalta, in addition to supportive care, achieved a statistically significant and clinically meaningful 35.1% (p=0.0014) reduction in proteinuria (as measured by 24-hour urine protein to creatinine ratio [UPCR]) at 6 months when compared to placebo. In many kidney diseases, proteinuria reduction is an increasingly recognized surrogate marker correlating with delaying progression to kidney failure. Additional data on the secondary endpoint of estimated glomerular filtration rate (eGFR), a measure of kidney function, showed a numerical improvement of +2.2 mL/min/1.73 m² (p=0.3241) over 6 months with Fabhalta compared to placebo. The eGFR remained stable during the 12 months duration of the study in the iptacopan treatment arm (+0.4 ml/min/1.73 m² from baseline). In a long-term extension study, the initial UPCR reduction was maintained and stabilization of eGFR was observed over more than 3 years after initiation of the treatment with Fabhalta

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