FDA approves Gazyva as first new therapy for idiopathic nephrotic syndrome in decades

FDA has approved Gazyva (obinutuzumab) to reduce the risk of relapse in adults and children aged 2 years and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome (INS) who are in complete remission. The decision introduces the first FDA-approved therapy specifically for INS in approximately 70 years.

INS is a rare kidney disorder and the most common cause of nephrotic syndrome in children. Although many patients respond initially to corticosteroids, relapse rates remain high, often leading to repeated courses of steroids and other immunosuppressive therapies. Long-term disease management can be complicated by treatment-related adverse effects as well as recurrent episodes of proteinuria, edema, infection, and thrombotic complications.

The approval was supported by findings from the phase 3 INShore trial. At 52 weeks, 95% of patients treated with Gazyva had no relapses after week 8 and were in complete remission, compared with 73% of those receiving mycophenolate mofetil (MMF), a commonly used maintenance therapy. Gazyva was also associated with longer relapse-free survival, a longer time to first relapse or death, and fewer relapses overall. As a B-cell-depleting therapy, Gazyva targets a suspected driver of disease activity in INS and may offer an alternative approach to maintaining remission in patients with frequently relapsing or steroid-dependent disease.

No new safety signals were observed in INShore. The most common adverse reactions were infections, infusion-related reactions, and neutropenia. Labeling notes that patients taking Gazyva may be at higher risk for severe infections than those taking MMF. Gazyva carries boxed warnings for hepatitis B virus reactivation and progressive multifocal leukoencephalopathy, a rare and serious brain infection.

"For too long, patients living with INS have had to endure unpredictable relapses, prolonged steroid use, and the constant threat of disease progression," said Andi Callaway, founder and president of The Nephrotic Syndrome Foundation. "This approval of Gazyva marks a shift that enables patients and families facing this debilitating disease to raise expectations for care and look ahead with optimism for a brighter future."

Sources: FDA. (2026 Sep 25). FDA approves drug to treat idiopathic nephrotic syndrome in patients 2 years and older; Genentech. (2026 Sep 25). FDA approves Gazyva for idiopathic nephrotic syndrome in patients aged 2 and older