"Novartis has received approval from the US Food and Drug Administration (FDA) for its oral treatment Fabhalta® (iptacopan) for adults with C3 glomerulopathy (C3G).
C3G is a rare and progressive kidney disease that often affects young adults, with an average age of diagnosis of 23 years. The condition leads to significant kidney damage, with around half of those affected progressing to kidney failure within a decade, necessitating dialysis or transplantation.
Symptoms include fatigue, mobility issues, and mental health challenges such as depression and anxiety.
Before Fabhalta’s approval, treatment options were limited to supportive care and immunosuppression. The newly approved therapy is the only oral inhibitor targeting the alternative complement pathway, which is believed to be the underlying cause of the disease.
This marks the first and only approved treatment for the condition, designed to reduce proteinuria.
The FDA approval is based on results from the Phase III APPEAR-C3G study, which assessed the efficacy and safety of Fabhalta. The trial included a six-month randomised, double-blind treatment period comparing Fabhalta with placebo alongside supportive care.
In addition to the FDA approval, Fabhalta recently received a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for its use in C3G. Regulatory reviews are ongoing in China and Japan.
This latest approval marks the third for Fabhalta in the US and the second within the Novartis kidney disease portfolio. The drug was granted accelerated FDA approval in August 2024 for reducing proteinuria in adults with primary immunoglobulin A nephropathy (IgAN).
Its first FDA approval was in December 2023 for the treatment of paroxysmal nocturnal haemoglobinuria (PNH).
Novartis is also advancing research into other rare kidney diseases, including atypical haemolytic uraemic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN), and lupus nephritis (LN).
Alongside Fabhalta, Novartis is developing two additional investigational treatments for IgAN. Atrasentan, an oral endothelin A receptor antagonist, is under FDA review with a decision expected in the first half of 2025. Zigakibart, a subcutaneous anti-APRIL monoclonal antibody, is currently in Phase III trials.
Ongoing studies aim to further evaluate the safety and efficacy of Fabhalta in these conditions.