Novartis wins FDA approval for Fabhalta, the first treatment for C3 glomerulopathy

Novartis has achieved a significant regulatory milestone with the U.S. Food and Drug Administration (FDA) approval of Fabhalta (iptacopan) for the treatment of C3 glomerulopathy (C3G). This marks the first-ever FDA-approved therapy for the ultra-rare kidney disease, which has historically been managed only through supportive care, broad immunosuppression, and symptom control.

C3G is a progressive and debilitating renal disorder that primarily affects young adults, leading to kidney inflammation, proteinuria (excess protein in urine), and potential kidney failure. Many patients experience a rapid decline, with nearly half progressing to end-stage renal disease within a decade of diagnosis. The introduction of Fabhalta offers a groundbreaking alternative by targeting the underlying cause of C3G rather than just managing symptoms.

The approval is based on Phase III APPEAR-C3G study data, which demonstrated significant and sustained proteinuria reduction in patients treated with Fabhalta. The oral therapy is classified as an alternative complement pathway inhibitor, designed to modulate immune system overactivation, which is a core driver of C3G pathology. With its favorable safety profile and clinical efficacy, Fabhalta is set to reshape the standard of care for individuals with C3G, offering new hope to a community that has long faced limited treatment options.

What Makes Fabhalta a Breakthrough in Kidney Disease Treatment?

Fabhalta’s approval establishes a new standard in nephrology, particularly for patients suffering from complement-mediated kidney disorders. Unlike previous therapies, which relied on generalized immunosuppressants, Fabhalta selectively inhibits the alternative complement pathway, a major contributor to C3G progression.

The APPEAR-C3G Phase III study was pivotal in demonstrating Fabhalta’s impact. The trial involved a 6-month randomized, double-blind period, followed by a 6-month open-label extension, during which all participants received Fabhalta. Clinical results showed that proteinuria reduction was evident as early as 14 days into treatment and remained consistent through 12 months.

Beyond efficacy, Fabhalta maintained a strong safety profile, with no new safety concerns emerging from clinical trials. The most frequently reported adverse effects included nasopharyngitis and viral infections, which are common in immunomodulating therapies. Given the nature of Fabhalta’s mechanism, it is subject to a Risk Evaluation and Mitigation Strategy (REMS), requiring specific vaccinations prior to treatment initiation to mitigate the risk of serious infections.

Why Is the FDA Approval of Fabhalta Important for Patients and Physicians?

C3G has long posed significant challenges for both patients and healthcare providers. With no prior FDA-approved treatments, management strategies were limited to corticosteroids, immunosuppressants, and kidney transplantation in advanced cases. Physicians have struggled with unpredictable disease progression, making it difficult to prevent renal decline in younger patients.

With Fabhalta, the treatment landscape has fundamentally shifted. Nephrologists now have a targeted therapy that directly addresses the root cause of C3G, rather than relying on therapies designed for broader autoimmune kidney diseases. The oral formulation also enhances patient adherence and convenience, eliminating the need for frequent hospital visits associated with intravenous immunotherapies.

Patient advocacy groups have expressed optimism over the approval, recognizing Fabhalta as a long-awaited solution for individuals and families affected by C3G. Many patients have historically relied on off-label therapies, leaving them vulnerable to uncertain outcomes and inconsistent treatment responses. The introduction of Fabhalta as a dedicated treatment option offers newfound stability and a clearer path forward in C3G management.

How Does Fabhalta Fit Into Novartis’ Kidney Disease Portfolio?

Fabhalta represents the third FDA approval for Novartis in two years, following its prior indications for paroxysmal nocturnal hemoglobinuria (PNH) in 2023 and primary immunoglobulin A nephropathy (IgAN) in 2024. The latest approval further solidifies Novartis’ expanding presence in renal medicine, reflecting a strategic focus on rare and complex kidney disorders.

Beyond Fabhalta, Novartis is actively advancing additional kidney disease therapies, including:

• Atrasentan, an investigational endothelin A receptor antagonist, currently under FDA review for IgAN, with a decision expected in the first half of 2025.
• Zigakibart, a subcutaneous anti-APRIL monoclonal antibody, now in Phase III trials for IgAN.

The company is also exploring Fabhalta’s potential for broader nephrology indications, including atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN), and lupus nephritis (LN). If successful, Fabhalta could emerge as a leading therapy across multiple complement-driven renal diseases, reinforcing Novartis’ long-term strategy in nephrology innovation.

What’s Next for Fabhalta on the Global Stage?

With FDA approval secured, Novartis is pursuing regulatory approvals for Fabhalta in key international markets. The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has already issued a positive opinion, indicating potential EU market entry in the coming months. Regulatory submissions in China and Japan are also underway, positioning Fabhalta for wider global adoption.

As Fabhalta launches commercially, Novartis is expected to deploy a comprehensive market access strategy, ensuring affordability and reimbursement options for eligible patients. The company’s ongoing collaborations with nephrologists, patient advocacy organizations, and healthcare payers will be crucial in optimizing treatment accessibility and driving physician awareness.

How Is Novartis’ Stock Responding to Fabhalta’s FDA Approval?

The latest FDA approval has reinforced investor confidence in Novartis’ nephrology pipeline. As of March 20, 2025, Novartis shares closed at $113.23, marking a 1.50% increase following the announcement. Over the past year, the stock has appreciated by 17.50%, outperforming the broader pharmaceutical sector, which has averaged 8.87% growth.

Analysts currently maintain a “Hold” rating on Novartis, with a 12-month price target of $113.55, suggesting limited near-term upside. However, long-term prospects remain strong, driven by Fabhalta’s expansion into global markets and continued growth in renal disease treatments.

Investment Considerations for Novartis Shareholders

Financial analysts highlight three key factors for investors assessing Novartis’ growth trajectory:

• Market Adoption of Fabhalta – Strong physician uptake and global regulatory approvals could drive higher-than-expected revenue growth.
• Expansion of the Kidney Disease Portfolio – Further FDA approvals for atrasentan and zigakibart could solidify Novartis’ leadership in nephrology.
• Stock Valuation – While the current stock price reflects Fabhalta’s impact, additional catalysts may unlock further upside potential.

For now, existing shareholders may consider holding positions, while new investors might wait for a stronger entry point based on global approval milestones.