Novartis Secures Third FDA Approval for Fabhalta, Transforming C3G Treatment Landscape

Novartis Achieves Milestone with Approval of Fabhalta

Novartis Pharmaceuticals Corporation has announced a significant advancement in the treatment of C3 glomerulopathy (C3G), securing its third FDA approval for the oral medication Fabhalta® (iptacopan). On March 20, 2025, the U.S. Food and Drug Administration granted approval for Fabhalta, making it the first and only treatment to specifically address this rare kidney disease in adults. This groundbreaking development aims to reduce proteinuria, which is a clinical hallmark of C3G, ultimately transforming the treatment landscape for patients suffering from this debilitating condition.

C3G is an ultra-rare disease characterized by the build-up of complement component 3 (C3) protein in the kidneys, leading to inflammation and potential failure of renal function. Traditionally, treatment options were limited, and patients often relied on supportive care or broad immunosuppressive therapies. Fabhalta stands out as the only oral alternative complement pathway inhibitor that targets the underlying cause of C3G, providing hope for improved clinical outcomes for patients.

Dr. Carla Nester, a prominent pediatric nephrologist at the University of Iowa, emphasized the historical significance of this approval. Recognizing the physical and emotional toll that C3G exerts on patients, especially young adults, she stated, "With the approval of Fabhalta, we now have a therapy that addresses the root cause of C3G, offering potential for a new standard of care."

The pivotal Phase III APPEAR-C3G study played a crucial role in demonstrating Fabhalta's efficacy and safety. In this randomized, double-blind trial involving adult patients with C3G, the results showed a significant reduction in proteinuria from as early as 14 days after starting treatment. By the end of the 12-month period, patients continued to experience clinically meaningful reductions, showcasing Fabhalta's potential to make a real difference in patients' lives. Additionally, the trial demonstrated a favorable safety profile, with no new safety signals identified among participants.

Lindsey Fuller, a C3G patient and advocate, expressed her relief and optimism about the approval, stating, "Having an approved treatment that can be taken orally is something we have long awaited. This approval is a beacon of hope for myself and many others living with this unrelenting disease."

C3G has a poor prognosis, with approximately half of those diagnosed progressing to kidney failure within a decade, highlighting the urgent need for effective treatment options. The median age of diagnosis is around 23, and the associated challenges transcend physical health, often leading to mental health issues like anxiety and depression. By introducing Fabhalta, Novartis aims to alleviate not only the clinical symptoms of C3G but also the broader emotional and psychological burdens faced by patients.

The road to FDA approval was marked by a thoughtful collaboration between Novartis and dedicated clinical trial participants. Victor Bultó, President of Novartis US, acknowledged the invaluable contributions of patients and investigators, stating, "Without their involvement, this groundbreaking approval for C3G would not have been possible. Our goal is to continue to innovate and advance care for individuals living with kidney diseases."

Fabhalta has previously received FDA approvals for other conditions, including paroxysmal nocturnal hemoglobinuria (PNH) and primary immunoglobulin A nephropathy (IgAN), depicting Novartis' commitment to addressing a spectrum of kidney-related health issues. The ongoing research into other rare kidney diseases, such as atypical hemolytic uremic syndrome (aHUS) and lupus nephritis (LN), further solidifies the company’s leadership in the nephrology space.

Future Outlook

Looking ahead, regulatory reviews for Fabhalta are ongoing in global markets, including China and Japan. Furthermore, Novartis is advancing the late-stage development of two additional therapies for IgAN, showcasing its dedication to redefining kidney disease management. By enhancing the treatment landscape for kidney diseases, Novartis intends to empower patients to regain control of their lives, mitigating the significant physical and emotional challenges posed by chronic conditions.

Fabhalta is set to revolutionize the standard of care for C3G, and with continuous advancements in nephrology, Novartis is committed to fostering a brighter future for individuals battling kidney diseases worldwide.