Basel & Cambridge, MA – Novartis has announced that the U.S. Food and Drug Administration (FDA) has granted approval for oral Fabhalta, a groundbreaking treatment for adults with C3 glomerulopathy (C3G) aimed at reducing proteinuria. This approval marks Fabhalta as the first and only FDA-approved therapy for C3G, a rare and debilitating kidney disease.
“C3G is a devastating disease, particularly affecting young individuals and significantly impacting both their physical and emotional well-being,” said Carla Nester, M.D., M.S.A., F.A.S.N., Professor of Pediatrics-Nephrology at the University of Iowa and co-investigator of the Fabhalta APPEAR-C3G study. “Historically, treatment options have been limited and often come with significant challenges. This approval of Fabhalta is a historic milestone for the C3G community, as it offers, for the first time, a treatment believed to address the underlying cause of the disease, creating the potential for a new standard of care for C3G patients.”
Fabhalta is the only oral therapy that inhibits the alternative complement pathway, selectively targeting the suspected root cause of C3G. Prior to this approval, patients were reliant on supportive care, broad immunosuppressive treatments, and symptomatic management to cope with the disease’s progression and complications.
“As someone whose family has experienced the impact of C3G across generations, the physical and emotional toll this disease takes on individuals is hard to fully describe,” said Lindsey Fuller, a C3G patient and co-leader of C3G Warriors. “The approval of Fabhalta, especially in an oral form, represents a long-awaited breakthrough for those of us living with C3G. This approval offers hope not just for me and my family, but for countless others who have been yearning for an effective treatment.”
C3G is a rare, progressive kidney disease with no FDA-approved treatments until now. Diagnoses typically occur around the age of 23, and the prognosis is poor, with approximately 50% of those affected progressing to kidney failure within 10 years, necessitating lifelong dialysis or a kidney transplant. Beyond its physical effects, C3G also causes severe fatigue, mobility impairments, and mental health challenges, including depression and anxiety, profoundly impacting the quality of life for those living with the condition.
The approval of Fabhalta offers hope to patients and families grappling with C3G, providing a potential new path to better manage and treat this challenging and often debilitating condition.
