Novartis has spent considerable time and energy building its presence in the IgA nephropathy (IgAN) disease area, and now it can celebrate a key trial win for its first drug to gain an FDA approval in the indication.
At the final analysis of the phase 3 Applause-IgAN trial, Novartis’ complement factor B inhibitor Fabhalta showed that it can slow kidney function decline and IgAN progression, the company said in an Oct. 16 release.
The placebo-controlled trial measured patients’ kidney function by looking at their estimated glomerular filtration rate (eGFR) slopes over the span of two years. An eGFR blood test is a straightforward process to assess kidney functioning, and doing the tests over a period of time can show how patients’ conditions are progressing.
Novartis plans to leverage the data for Fabhalta regulatory submissions next year, the company said in its release. The Swiss drugmaker didn’t share detailed results from the phase 3 trial but said they would be presented at future medical meetings.
The final analysis showing follows Fabhalta’s prior win in the same study on a separate surrogate endpoint that measured the reduction in patients’ proteinuria (protein in the urine) at nine months versus placebo. That endpoint is an “increasingly recognized surrogate marker correlating with progression to kidney failure,” Novartis said at the time of the trial readout.
Those data allowed the drug to secure an accelerated FDA approval in IgAN in April of last year. Now, Novartis believes it has the evidence to support a traditional FDA approval for the medicine.
A debilitating autoimmune kidney disease, IgAN is diagnosed in about 25 people per million each year worldwide, according to Novartis. Many patients eventually progress to kidney failure. Supportive care and symptom management alone usually don't slow progression.
Fabhalta won its original FDA approval in late 2023 as a treatment for the rare blood disease paroxysmal nocturnal hemoglobinuria. And, since its IgAN approval last year, the med picked up yet another approval in March as a treatment for complement 3 glomerulopathy.
The medicine, which pulled down $201 million in the first half of 2025, forms part of Novartis’ ambitious positioning in IgAN. Besides Fabhalta, the company in April won an IgAN approval for Vanrafia, a medicine picked up in its $2.7 billion buyout of Chinook Therapeutics.
In addition to those two medicines, the company’s investigational anti-APRIL antibody, zigakibart, is in phase 3 testing in IgAN.
In this disease area, the Swiss drugmaker is going up against Travere Therapeutics and its Filspari, which is approved to slow kidney function decline in adults with primary IgAN who are at risk of disease progression. Calliditas Therapeutics’ Tarpeyo is also cleared to treat patients at risk of disease progression. All of the FDA approvals in IgAN so far are for oral medications.