FDA approves Novartis' Fabhalta for rare kidney disease C3G

Novartis has achieved a significant milestone with the approval of its drug, Fabhalta (iptacopan), by the US Food and Drug Administration (FDA) for the treatment of complement 3 glomerulopathy (C3G), a rare kidney disease. This marks the first time a specific treatment has been approved for C3G, providing hope for patients living with this debilitating condition. C3G affects approximately one to two people per million annually worldwide. It is characterized by the accumulation of C3 protein deposits in the kidney glomeruli, which are responsible for filtering waste and excess fluids from the blood. The buildup leads to inflammation and damage, resulting in proteinuria, haematuria, and impaired kidney function. Tragically, about half of those diagnosed with C3G progress to kidney failure within ten years.

Fabhalta is specifically designed to reduce proteinuria in adults suffering from C3G. By selectively targeting the presumed cause of the disease, it offers a novel approach to managing this condition. The drug is already recognized in the US for the treatment of other rare diseases, including immunoglobulin A nephropathy and paroxysmal nocturnal haemoglobinuria, a rare blood disorder. The FDA's decision to approve Fabhalta for C3G was based on promising results from the phase 3 APPEAR-C3G clinical trial. The study demonstrated a statistically significant 35.1% reduction in proteinuria for patients who received Fabhalta along with supportive care, compared to those given a placebo, over a six-month period.

Moreover, improvements were observed in the secondary endpoint, estimated glomerular filtration rate, which measures kidney function. These findings underscore the potential of Fabhalta to improve kidney health and function for C3G patients. The drug also exhibited a favorable safety profile, with no new safety signals noted during the trial. Carla Nester, a co-investigator of the APPEAR-C3G study, emphasized the importance of this approval, noting the impact C3G has on young individuals’ physical and emotional well-being. She highlighted the historical significance of Fabhalta's approval, as it presents the first therapy believed to address the root cause of C3G, offering a potential new standard of care for patients.

The approval of Fabhalta in the US follows a recent recommendation from the European Medicines Agency’s human medicines committee for the treatment of adults with C3G. The European Commission is now set to review this recommendation, signaling progress towards broader acceptance and availability of this innovative treatment in different regions. With Fabhalta, Novartis has opened new avenues for those affected by C3G, potentially transforming the standard of care and improving the quality of life for patients facing this rare kidney disease. As regulators continue to evaluate and endorse this treatment, the prospects for C3G patients are poised to improve significantly, offering them renewed hope and the possibility of better health outcomes.