Novartis Gains FDA Approval for Fabhalta in C3G

Novartis today announced that oral Fabhalta (iptacopan) has received U.S. Food and Drug Administration (FDA) approval for the treatment of adults with C3 glomerulopathy (C3G), to reduce proteinuria, making it the first and only treatment approved for this condition.

“C3G is a debilitating disease often affecting young people, impacting many aspects of their physical and emotional health, and our previous treatment options came with significant challenges,” said Carla Nester, M.D., M.S.A., F.A.S.N., Professor of Pediatrics-Nephrology at the University of Iowa and Fabhalta APPEAR-C3G Study Co-Investigator. “This approval of Fabhalta is historic for the entire C3G community as now, for the first time, we have a therapy that is believed to treat the underlying cause of the disease, providing the potential for a new standard of care for patients.”

Fabhalta is the only oral inhibitor of the alternative complement pathway to selectively target what is thought to be the underlying cause of the disease. Before the approval of Fabhalta, patients had to rely on supportive care, broad immunosuppression, and symptom management.

“As someone whose family has suffered from C3G across multiple generations, it is difficult to fully express the physical and emotional challenges of living with this unrelenting disease,” said Lindsey Fuller, C3G patient and Co-Leader of C3G Warriors. “To finally have an approved treatment – and one that can be taken orally – is something people with C3G have been waiting for. Today’s approval brings new hope for me, my family, and so many others.”

C3G is a progressive and ultra-rare kidney disease that, until now, has had no approved treatments. The average age of diagnosis is around 23 years old. Prognosis is poor, with approximately half of people living with C3G progressing to kidney failure within 10 years of diagnosis, requiring lifelong dialysis and/or kidney transplantation. People living with C3G may experience high levels of fatigue, mobility issues affecting everyday life activities, and mental health symptoms, including depression and anxiety.