Novartis releases new data for innovative rare kidney disease treatment

Novartis has released new data from the first and only Phase III study to demonstrate significant proteinuria reduction by targeting the complement system in patients with the rare kidney disease IgA nephropathy (IgAN). Pre-specified interim analysis results from the Phase III APPLAUSE-IgAN clinical trial were presented at the 2024 World Congress of Nephrology (WCN). Fabhalta ^® (iptacopan) is a Factor B inhibitor of the alternative complement pathway. It was investigated as a twice-daily oral treatment in the trial.

The small molecule treatment enabled a 38.3 percent proteinuria reduction in the IgAN at nine months compared to placebo on top of supportive care, the data showed.

Significance of the results from Novartis

“Fabhalta is the first potential treatment for IgAN that specifically targets the alternative complement pathway,” Professor Dana Rizk, Investigator and APPLAUSE-IgAN Steering Committee Member and professor in the UAB Division of Nephrology commented.

In December 2023, the US Food and Drug Administration (FDA) approved the treatment for adults with the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH). Fabhalta was given a positive opinion from the CHMP of the EMA in March 2024, according to Novartis. 

“IgAN progresses over many years, and patients’ needs may evolve such that different therapies may be best used at different times,” shared Dr David Soergel, Global Head, Cardiovascular, Renal and Metabolism Development Unit, Novartis. “Our renal pipeline includes medicines with a variety of mechanisms which may allow them to be targeted to patients based on their clinical characteristics.”

The APPLAUSE-IgAN clinical trial is ongoing and only limited interim analysis results can be presented. Fabhalta was well tolerated with a favorable safety profile consistent with previously reported data, Novartis confirmed.

The company’s acquisition of Chinook Therapeutics in June last year was anticipated to significantly expand Novartis’ renal portfolio. The deal included rights to Chinook Therapeutics’ therapeutic candidates for IgA nephropathy.