Novartis Secures Landmark FDA Approval for Fabhalta® in Rare Kidney Disease
Novartis has received its third U.S. Food and Drug Administration (FDA) approval for Fabhalta® (iptacopan), marking a major milestone as the first and only approved treatment for adults with C3 glomerulopathy (C3G), an ultra-rare kidney disease. The once-daily oral medication is approved to reduce proteinuria in patients living with this progressive condition.
Oral Treatment Becomes First-ever Approved Therapy for C3 Glomerulopathy
This latest approval adds to Fabhalta’s growing therapeutic footprint, following previous FDA nods for paroxysmal nocturnal hemoglobinuria (PNH) and primary immunoglobulin A nephropathy (IgAN).
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 stated:
“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. 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.”
C3G affects approximately 1 to 2 individuals per million annually and is typically diagnosed around age 23. Characterized by overactivation of the alternative complement pathway, the disease causes inflammation and damage in the glomeruli—kidney structures vital to blood filtration. Nearly 50% of those diagnosed progress to kidney failure within a decade, often requiring lifelong dialysis or transplant.
Pivotal Trial Data from the Phase III APPEAR-C3G Study
The approval is based on positive results from the Phase III APPEAR-C3G study, which evaluated the efficacy and safety of twice-daily oral Fabhalta in adult C3G patients. The study included a six-month randomized, double-blind phase comparing Fabhalta to placebo, followed by a six-month open-label period.
Clinically meaningful reductions in proteinuria were observed as early as 14 days into treatment and sustained through 12 months. Those who switched from placebo to Fabhalta during the open-label phase also demonstrated similar improvements. The therapy showed a favorable safety profile, with nasopharyngitis and viral infections among the most common adverse reactions. Fabhalta will be available under a Risk Evaluation and Mitigation Strategy (REMS), due to the risk of serious infections from encapsulated bacteria.
Lindsey Fuller, C3G patient and Co-Leader of C3G Warriors stated:
“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. To finally have an approved treatment – and one that can be taken orally – is something people with C3G have been waiting for.”
Global Approval Momentum
The U.S. approval follows a positive opinion issued last month by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP), with regulatory reviews currently underway in China and Japan.
Victor Bultó, President US, Novartis said:
“We extend our deepest gratitude to the patients and investigators who participated in our clinical trials, without whom this first FDA approval in C3G wouldn’t have been possible. With this additional approval for Fabhalta – the second in kidney disease – we will leverage our established capabilities and expertise to bring this innovative treatment to patients in need as we work to help transform care for people living with kidney diseases.”
This latest approval strengthens Novartis’ commitment to nephrology, with Fabhalta now approved across three indications. The company also continues to advance other therapies for kidney conditions with high unmet need, including atrasentan and zigakibart, both currently in late-stage development for IgAN.
Advancing Kidney Disease Therapies
With a four-decade legacy in kidney disease, Novartis is aiming to shift the paradigm from reactive treatment to proactive, disease-modifying therapies. The company’s pipeline targets underlying mechanisms of kidney conditions in an effort to slow or prevent progression to end-stage renal disease. Through collaborations with stakeholders across the healthcare ecosystem, Novartis is also focused on accelerating diagnosis and improving access to treatment.
About APPEAR-C3G
The APPEAR-C3G study (NCT04817618) is a multicenter, randomized, double-blind, placebo-controlled Phase III trial evaluating oral Fabhalta (200 mg, twice daily) in adult patients with native kidney C3G. The primary endpoint was proteinuria reduction from baseline at six months, measured by 24-hour urine protein-creatinine ratio. A separate adolescent cohort is currently enrolling.
About C3G
C3G is a form of membranoproliferative glomerulonephritis driven by dysregulation of the alternative complement pathway. This leads to abnormal C3 protein deposits in kidney glomeruli, causing inflammation, hematuria, proteinuria, and a decline in kidney function. The disease is rare, chronic, and typically diagnosed in young adulthood.
Original Source: Press Release, Basel, March 20, 2025, Novartis: Novartis receives third FDA approval for oral Fabhalta® (iptacopan) – the first and only treatment approved in C3 glomerulopathy (C3G)
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