Novartis oral Fabhalta® (iptacopan) receives positive CHMP opinion for the treatment of adults living with C3 glomerulopathy (C3G)

Core Viewpoint - Novartis has received a positive opinion from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommending marketing authorization for Fabhalta® (iptacopan), a first-in-class oral Factor B inhibitor, for treating adults with C3 glomerulopathy (C3G) [1][5][6] Group 1: Product and Clinical Data - Fabhalta is the first treatment for C3G, a rare kidney disease with no approved therapies, and aims to address the underlying cause of the disease [2][4][6] - The Phase III APPEAR-C3G study demonstrated a statistically significant 35.1% reduction in proteinuria at 6 months compared to placebo, indicating a meaningful clinical benefit [3][6][7] - The estimated glomerular filtration rate (eGFR) showed a numerical improvement of +2.2 mL/min/1.73 m² over 6 months, with stabilization observed over 12 months in the treatment group [3][6][7] Group 2: Market Context and Future Prospects - C3G is typically diagnosed in young adults, with approximately 50% of patients progressing to kidney failure within 10 years of diagnosis, highlighting the urgent need for effective treatments [2][6] - Following the CHMP's recommendation, the European Commission will make a final decision on the approval within two months, with ongoing regulatory reviews in the US, China, and Japan [5][6] - Novartis is advancing multiple potential treatments for kidney diseases, emphasizing its commitment to addressing unmet medical needs in this area [9][10]