Novartis Unveils First and Only Fabhalta® (iptacopan) for the Treatment of C3 Glomerulopathy (C3G)
Novartis has introduced Fabhalta® (iptacopan) as the first and only FDA-approved treatment for adults with C3 glomerulopathy (C3G), a rare and progressive kidney disease that often leads to kidney failure.
Fabhalta® is an oral inhibitor targeting the alternative complement pathway, addressing the underlying cause of C3G and reducing proteinuria.
Symptoms include fatigue, mobility difficulties, and mental health challenges such as depression and anxiety.
Treatment options for C3G were limited to supportive care and immunosuppressive therapy. The newly approved drug works by inhibiting the alternative complement pathway, which plays a role in the development of the disease, and has been shown to reduce proteinuria, a key indicator of kidney damage.
Approval was granted based on data from the Phase III APPEAR-C3G trial, which assessed the drug’s efficacy and safety over a six-month period in a randomised, double-blind study comparing the treatment with a placebo in combination with supportive care.
Glomerulopathy (C3G) primarily affects young adults, with an average diagnosis age of 23 years. The disease leads to significant kidney damage, with approximately half of those affected progressing to kidney failure within a decade, requiring dialysis or transplantation.
Fabhalta® is the first and only FDA-approved treatment for C3G, offering a targeted option for managing this progressive kidney disease.
