The FDA Approves FABHALTA, First Treatment Option for Adults with C3G

A historic milestone has been reached for the rare kidney disease (RKD) community — the FDA has approved the drug FABHALTA as the first-ever treatment option for adults with C3 glomerulopathy (C3G). This long-awaited approval brings new hope to patients and families affected by this devastating disease.

C3G is an ultra-rare, debilitating kidney disease that damages the glomeruli (the filtering units in the kidneys that help remove toxins from the blood) which can ultimately lead to kidney failure. Until now, there were no approved targeted treatment options available for C3G. 

“This approval marks a significant step forward in our collective goal to improve the outcomes of those affected by rare kidney diseases. For the first time, the C3G community has a therapy approved and designed specifically for their condition. This comes on the heels of groundbreaking advancements in IgAN, which underscores the current exciting new era of innovation in RKD treatments,” Josh Tarnoff, NephCure’s CEO said. 

FABHALTA’s approval for FABHALTA in C3G is based off Novartis’ Phase 3 study, APARECER-C3G, which evaluated the efficacy and safety of the drug and demonstrated reduction in proteinuria, which was seen as early as 14 days. 

This marks the third FDA approval, in the U.S., for FABHALTA and its second within the Novartis’ kidney disease portfolio in less than a year. In August 2024, the drug received accelerated approval para Nefropatía por IgA (NIgA). 

A New Era of Kidney Care

With this latest breakthrough, there are now four FDA-approved therapies for rare kidney disease:

• FABHALTA (iptacopan) – Approved for C3G and IgAN
• FILSPARI (sparsentan) – Approved for IgAN
• TARPEYO (budesonide) – Approved for IgAN

This approval marks an important advancement, but our work is far from over. NephCure remains committed to advancing research, advocacy, and patient education to further improve treatment options and outcomes for those with C3G and other rare kidney diseases. With more than 60 clinical trials in the RKD space, we anticipate even more new treatments to be approved in the near future. 

Now, the focus shifts to ensuring timely and equitable access to these new and better treatments for all patients.

If you or a loved one has C3G, we encourage you to speak with your doctor to determine if FABHALTA may be an appropriate treatment option for you. 

To read Novartis’ press release about FABHALTA’s approval for C3G, haga clic aquí.

For more information on C3G and available resources, please visit NephCure.org.