New Novartis Fabhalta (iptacopan) data show clinically meaningful and statistically significant proteinuria reduction of 38.3% versus placebo for patients with IgA nephropathy (IgAN)

• APPLAUSE-IgAN is first and only Phase III study to demonstrate significant proteinuria reduction by targeting the complement system in patients with IgAN¹
  
  
• IgAN is a heterogeneous, progressive, rare kidney disease and is a major cause of chronic kidney disease worldwide²; complement activation is a key driver of glomerular inflammation in IgAN^3,4
  
  
• There is a need for effective, targeted therapies for IgAN^2,5; up to 30% of patients with persistent proteinuria (≥1 g/day) may progress to kidney failure within 10 years, requiring maintenance dialysis and/or kidney transplantation⁶
  
  
• Novartis continues to advance broad renal portfolio in late-stage development, exploring the potential to slow disease progression and extend dialysis-free life  

Basel, April 15, 2024 – Novartis today presented results from a pre-specified interim analysis of the Phase III APPLAUSE-IgAN study of Fabhalta (iptacopan), an investigational Factor B inhibitor of the alternative complement pathway, in patients with IgA nephropathy (IgAN)¹. In the analysis, patients treated with Fabhalta achieved a 38.3% (p<0.0001) proteinuria reduction (as measured by 24-hour urine protein to creatinine ratio [UPCR]) at 9 months when compared to placebo on top of supportive care¹.

Proteinuria reduction is an increasingly recognized surrogate marker correlating with progression to kidney failure and has been used as an endpoint in IgAN clinical trials to support accelerated approvals⁷. The study also showed that Fabhalta was well tolerated with a favorable safety profile consistent with previously reported data^1,8. Results were presented today during a late-breaking clinical trials session at the World Congress of Nephrology (WCN) in Buenos Aires, Argentina¹.

“In IgAN, part of the immune system called the alternative complement pathway can become overly activated in the kidneys, which causes an inflammatory response, leading to progressive kidney damage and gradual loss of kidney function. The loss of kidney function, together with potential side effects of IgAN treatments available until recently, significantly impact patients’ lives,” said Professor Dana Rizk, Investigator and APPLAUSE-IgAN Steering Committee Member and professor in the UAB Division of Nephrology. “Fabhalta is the first potential treatment for IgAN that specifically targets the alternative complement pathway.”