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August 8, 2024: Today marks a promising day for the rare kidney disease (RKD) community: the FDA has announced that Novartis’ drug, Fabhalta (iptacopan), has been granted accelerated approval for the treatment of IgA nephropathy in adults.
As background, the FDA’s accelerated approval program allows for earlier approval of drugs that treat serious conditions and fill an unmet medical need. Fabhalta is granted accelerated approval based on reduction of proteinuria. Further evaluations will determine if the drug slows kidney function decline in IgAN patients.
Over the next several months, Novartis will share more information about this new treatment and explain when and how those who need the drug can access it.
“The FDA’s accelerated approval of Fabhalta represents a critical advancement in addressing the serious and debilitating symptoms of IgA nephropathy. It adds another key option for the rare kidney disease community, as patients respond differently to various therapeutic agents, and we need more options offering different mechanisms of action,” said Josh Tarnoff, NephCure’s CEO.
“This is a momentous occasion that underscores the significant progress our community continues to make in developing effective treatments for this devastating disease. We know that our work is far from over, but these breakthroughs are critical to ensuring our patient community has increasing access to life-saving treatments. This is not just hope – this is a path paved forward.”
The continued approval of Fabhalta may be dependent upon the data from Novartis’ ongoing phase 3 APPLAUSE-IgAN study, which evaluates whether Fabhalta slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline over 2 years. The eGFR data are expected at study completion in 2025 and are intended to support traditional FDA approval.
Key takeaways regarding Fabhalta:
- Fabhalta achieved a 44% proteinuria reduction from baseline in Phase III APPLAUSE-IgAN interim analysis, compared with 9% in placebo arm, demonstrating a clinically meaningful reduction of 38% vs. placebo.
- Fabhalta is an inhibitor of the alternative complement pathway, activation of which is thought to contribute to the pathogenesis of IgAN.
- Despite current standard of care, up to 50% of IgAN patients with persistent proteinuria progress to kidney failure within 10 to 20 years of diagnosis.
- This marks the first approval from Novartis’ renal pipeline, which also includes atrasentan and zigakibart.
Fabhalta is also being developed to treat several other rare diseases beyond IgA nephropathy (IgAN), such as C3 glomerulopathy (C3G), atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN), and lupus nephritis (LN). Ongoing studies are assessing its safety and effectiveness for these conditions to support potential regulatory approvals. The company plans to submit Fabhalta to the FDA and EMA for C3G treatment by the end of the year.
While we celebrate the FDA’s accelerated approval of Fabhalta, we also continue to move forward to ensure continued access to treatments like this. Our focus now turns to ensuring all patients have timely and equitable access to this new and promising treatment.
NephCure remains steadfast in our mission to empower people with protein-spilling kidney conditions to take charge of their health, while leading the revolution in research, new treatments, and care.
We are deeply grateful for the incredible support that has helped our community reach this pivotal moment, and we are more committed than ever to the important work ahead. There is still much to be done, but today’s news gives us hope and determination to deliver on our promise to the rare kidney disease community.
