Novartis is building out its rare kidney disease portfolio with two batches of Phase 3 data at the European Renal Association conference, touting positive results for an investigational drug in IgA nephropathy and for its drug Fabhalta in C3 glomerulopathy.
In an interim analysis of the IgA nephropathy trial, called ALIGN, patients on Novartis’ experimental drug atrasentan saw a 36.1% reduction in proteinuria, a measure of protein in the urine (p<0.0001). Proteinuria levels are a biomarker used to track the kidney disease progression, and have been used in IgAN trials to support approval, according to Novartis.
The company plans to apply for approval for atrasentan, an oral selective endothelin A receptor antagonist, in the first half of this year. Secondary endpoints from the trial, including the eGFR rate that measures the kidneys’ ability to filter toxins from the blood, aren’t expected until 2026.
David Soergel, Novartis’ global head of cardiovascular, renal and metabolic drug development, told Endpoints News that just five years ago, there were no options for patients with IgAN, and now there is “a whole repertoire of potential therapeutic options.”
Rare kidney disease trial
The second batch of Phase 3 data was from a trial of Novartis’ drug Fabhalta in patients with the ultra-rare kidney disease C3 glomerulopathy, which currently has no approved treatments. In combination with supportive care, Fabhalta showed a 35.1% reduction in proteinuria. Novartis plans to submit the data for approval in the US and the EU in the second half of this year.
David SoergelIn an April 26 note, Jefferies analysts called Fabhalta an “underappreciated blockbuster,” adding that with a $550,000 US list price, they’re predicting up to $6 billion in worldwide peak sales — including $1.75 billion in C3 glomerulopathy. Fabhalta is already approved in paroxysmal nocturnal hemoglobinuria (PNH), a chronic and rare blood disorder, and also read out positive data in IgAN in April.
Soergel added that while Novartis has a 40-year history in kidney disease, their earlier renal medicines were transplant-focused. But there are three things “paving the way” for more improvements in treating kidney disease: growing unmet need, better understanding of the diseases and more open-minded regulators who will approve drugs on surrogate endpoints like proteinuria reduction.
“We’re very committed to this space and developing innovative therapies for patients with rare kidney diseases,” Soergel said. “We believe this space is opening up even more.”