Novartis touts surrogate endpoint win, nabs priority review for Fabhalta in IgAN

A Novartis drug with "pipeline in a pill" potential, according to one analyst group, has delivered on that promise once again. 

Monday, Novartis said treatment with Fabhalta led to a "clinically meaningful and statistically significant" 38.3% reduction of proteinuria (protein in the urine) in patients with IgA nephropathy (IgAN). The result came from a pre-specified interim analysis of the phase 3 APPLAUSE-IgAN study.

For the analysis, investigators looked at patients' 24-hour urine protein to creatinine ratio after nine months of treatment with either the Novartis drug or placebo. The data were presented Monday at the World Congress of Nephrology in Buenos Aires, Argentina.

Alongside the data drop, Novartis said the FDA has accepted its application for Fabhalta in IgAN and has granted a priority review.

Reduction of proteinuria is an "increasingly recognized" endpoint associated with improved kidney outcomes, the drugmaker said in its release. The FDA has already granted accelerated approvals to Calliditas Therapeutics' Tarpeyo and Travere Therapeutics' Filspari in IgAN based on the surrogate endpoint.

When Novartis revealed top-line interim results from the trial back in October, analysts with ODDO BHF touted the drug's performance in the study as "faultless." At the time, the analysts said the drug had "pipeline in a pill" potential, thanks to its promise in IgAN and other renal and hematological diseases.

Shortly afterward, in December, Fabhalta won its original FDA nod to treat paroxysmal nocturnal hemoglobinuria. In that field, it's going up against AstraZeneca's stalwart complement inhibitors Soliris and Ultomiris, although the Novartis drug holds one advantage in that it's an oral therapy; AZ's drugs are given via injection or infusion.

About a week after the FDA approval, Novartis revealed that Fabhalta, also known as iptacopan, was associated with a statistically significant reduction in proteinuria in patients with C3 glomerulopathy (C3G). Novartis didn't provide detailed results from the phase 3 study in C3G study at the time.

Besides those diseases, Novartis is researching iptacopan in atypical hemolytic uremic syndrome and immune complex membranoproliferative glomerulonephritis. Analysts with Jefferies have estimated the drug could generate $3.6 billion in peak sales.

As for the IgAN study, APPLAUSE-IgAN remains ongoing to examine Fabhalta's ability to slow disease progression. To do this, investigators will examine the annualized total estimated glomerular filtration rate slope over a 24-month span. Those results are expected next year.

IgAN, also known as Berger disease, is a rare progressive kidney disease that affects about 25 million new patients worldwide each year, according to Novartis. Patients with elevated levels of proteinuria may progress to kidney failure within a decade.