Amid a steady advance of kidney-focused label expansions, Novartis’ Fabhalta is slowing its roll in a key potential autoimmune niche.
After wrapping up the first section of the study, the Swiss drugmaker has decided not to go onto part 2 of its midstage K12201 trial of Fabhalta, also known as iptacopan, in lupus nephritis (LN). The decision came on the heels of “strategic considerations” and, importantly, “was not driven by any interim efficacy or safety findings,” a Novartis representative said told Fierce in an email.
The study, which enrolled 81 participants according to the Federal clinical trials database, was hoping to assess the safety and efficacy of Fabhalta in adult patients with active LN also taking standard-of-care treatment, with or without oral corticosteroids.
The first phase of the study, which Novartis completed, pitted Fabhalta and standard-of-care against a placebo-SoC control, while the second phase was set to compare Fabhalta and standard-of-care, Fabhalta plus placebo alone and placebo plus standard-of-care.
Novartis told Fierce that study investigators have been notified of the decision to halt the trial and are currently working with study participants on next steps.
Novartis said that future development plans for Fabhalta in LN “will be evaluated” based on the data it accrued in part one of its study. The company representative also flagged Novartis’ ongoing commitment to the disease space by way of its late-stage study of dual-mechanism antibody drug ianalumab in LN.
Lupus nephritis is an inflammatory complications that affects patients living with systemic lupus erythematosus, in which autoantibodies attack the kidneys, causing swelling and irritation that can impair the waste-removing organs’ function or even lead to failure if left unaddressed.
Shortly before the drug’s initial FDA green light in paroxysmal nocturnal hemoglobinuria (PNH) back in Dec. 2023, analysts at ODDO BHF hailed Fabhalta as a potential “pipeline in a pill” for Novartis, which has indeed gone on to significantly expand the medicine’s pedigree of renal indications since then.
Notably, Fabhalta scooped up its second nod in immunoglobulin A nephropathy (IgAN) in the summer of 2024, rounding things out with an approval in the ultrarare kidney disease complement 3 glomerulopathy (C3G) the following March, specifically to reduce excess protein in the urine in a phenomenon known as proteinuria.
More recently, the drug in July converted the accelerated approval it had first won in IgAN into a full, formal endorsement from the FDA, which has signed off on the medicine to slow kidney function decline in adults with the disease who are at risk of progression.
Novartis has pinned blockbuster hopes on Fabhalta, which reeled in sales of $505 million for all of last year.
Though lupus nephritis would have marked another feather in the medicine’s cap, the disease area has grown increasingly crowded thanks to a number of new approvals in recent years, including for Roche’s Gazyva, a monoclonal antibody targeting CD20, last October.