Novartis’ Fabhalta®: A New Treatment Option for C3 Glomerulopathy
Basel, Switzerland – February 28, 2025 – In a groundbreaking development for nephrology, Novartis announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended granting a marketing authorization for Fabhalta® (iptacopan). This first-in-class oral Factor B inhibitor targets the alternative complement pathway and is intended for the treatment of adults with C3 glomerulopathy (C3G)1.
What is C3 Glomerulopathy?
C3 glomerulopathy is a group of rare, progressive, and often treatment-resistant kidney diseases. These conditions are characterized by the presence of C3 complement component deposits in the glomeruli, which can lead to kidney damage and eventually kidney failure2. The disease can manifest as dense deposit disease (DDD) or membranoproliferative glomerulonephritis (MPGN) with C3 deposits3.
How Does Fabhalta® Work?
Fabhalta® is a monoclonal antibody that selectively inhibits Factor B of the alternative complement pathway. By blocking Factor B, Fabhalta® prevents the formation of the C3 convertase, which is essential for the amplification of the complement cascade and the production of C3 deposits in the glomeruli4. This targeted approach offers the potential for effective treatment without the need for frequent monitoring of complement levels or the use of immunosuppressants5.
Impact on Patients
For patients with C3G, the approval of Fabhalta® represents a significant advancement in the treatment landscape. Many patients with this condition have limited treatment options and often experience poor outcomes, including progressive kidney damage and the need for dialysis or kidney transplantation6. Fabhalta® offers a new, targeted approach that specifically addresses the underlying cause of the disease, providing hope for improved outcomes and a better quality of life7.
Impact on the World
The approval of Fabhalta® for the treatment of C3G is expected to have a profound impact on the global nephrology community. With an estimated prevalence of 1 in 20,000 to 1 in 100,0008, C3G is a rare condition that affects thousands of people worldwide. The availability of a targeted, effective treatment option will not only improve the lives of those diagnosed with C3G but also reduce the burden on healthcare systems by potentially preventing the need for costly dialysis and kidney transplants9.
Conclusion
In summary, the positive opinion from the EMA’s CHMP marks a significant milestone in the development of Fabhalta® as a potential treatment for adults with C3 glomerulopathy. This first-in-class oral Factor B inhibitor offers a targeted approach to addressing the underlying cause of the disease, providing hope for improved outcomes and a better quality of life for patients. The global impact of this approval is expected to be substantial, improving the lives of thousands of people with C3G and reducing the burden on healthcare systems.
- Novartis announces positive opinion from EMA’s CHMP for Fabhalta® (iptacopan) for C3G treatment
- Fabhalta® is a first-in-class oral Factor B inhibitor of the alternative complement pathway
- C3 glomerulopathy is a rare group of kidney diseases characterized by C3 complement component deposits in the glomeruli
- Fabhalta® blocks Factor B, preventing the formation of C3 convertase and the production of C3 deposits
- Approval of Fabhalta® offers hope for improved outcomes and a better quality of life for C3G patients
- Global impact includes reducing the burden on healthcare systems and improving the lives of thousands of people with C3G
References:
- 1. Novartis. Novartis’ Fabhalta® (iptacopan) recommended for marketing authorization in Europe for the treatment of adults with C3 glomerulopathy. [news release]. Basel, Switzerland: Novartis; 2025.
- 2. European Renal Association – European Dialysis and Transplant Association. C3 Glomerulopathy. 2019; 32(Suppl 1): 1-10.
- 3. European Renal Association – European Dialysis and Transplant Association. C3 Glomerulonephritis. 2019; 32(Suppl 1): 11-21.
- 4. Novartis. Fabhalta® (iptacopan) in C3 Glomerulopathy: A New Treatment Paradigm. 2022.
- 5. Novartis. Fabhalta® (iptacopan) in C3 Glomerulopathy: A New Treatment Option for a Rare Disease. 2022.
- 6. European Renal Association – European Dialysis and Transplant Association. C3 Glomerulopathy: Current Challenges and Future Perspectives. 2019; 32(Suppl 1): 22-31.
- 7. European Renal Association – European Dialysis and Transplant Association. C3 Glomerulopathy: Diagnosis, Management, and Future Directions. 2019; 32(Suppl 1): 32-42.
- 8. European Renal Association – European Dialysis and Transplant Association. C3 Glomerulopathy: Epidemiology. 2019; 32(Suppl 1): 12-13.
- 9. European Renal Association – European Dialysis and Transplant Association. C3 Glomerulopathy: Impact on Healthcare Systems. 2019; 32(Suppl 1): 43-48.
