FDA Approves Gazyva (Obinutuzumab) for Idiopathic Nephrotic Syndrome in Patients 2 Years and Older

The U.S. Food and Drug Administration (FDA) has approved Gazyva (obinutuzumab) injection to reduce the risk of relapse in adult and pediatric patients 2 years of age and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome (INS) who are in complete remission. Gazyva was previously approved for certain cancers and for adults with active lupus nephritis receiving standard therapy.

A Kidney Disorder That Often Begins in Childhood

Idiopathic nephrotic syndrome is a kidney disorder of unknown cause characterized by the leakage of large amounts of protein into the urine. This can lead to low blood albumin levels and swelling, as well as complications such as high cholesterol, infections, and blood clots. INS is the most common cause of nephrotic syndrome in children and usually begins between 2 and 7 years of age. The FDA estimates its annual incidence at 1 to 4 cases per 100,000 children, varying by age, race, and geography.

Phase 3 INSHORE Trial Supported Approval

The approval was supported by INSHORE (NCT05627557), a Phase 3, randomized, controlled, open-label, multicenter study involving 85 patients aged 2 years and older with childhood-onset, frequently relapsing or steroid-dependent INS. Patients were required to be in complete remission at study entry.

Participants were randomized to intravenous Gazyva, administered according to body weight on Days 1 and 15 and Weeks 24 and 26, or twice-daily oral mycophenolate mofetil (MMF), a commonly used standard-of-care therapy. The primary endpoint assessed the proportion of patients with a first-morning urine protein-to-creatinine ratio of no more than 0.2 g/g at Week 52 without relapse from Week 8 through Week 52.

Gazyva Reduced Relapses and Sustained Remission

Genentech reported that 95% of patients receiving Gazyva achieved the primary endpoint compared with 73% of those receiving MMF at Week 52. The result reflected a greater proportion of patients who remained free of relapse after Week 8 while maintaining complete remission at one year.

Analyses of key secondary endpoints also showed statistically significant and clinically meaningful benefits with Gazyva compared with MMF. These included an increase in overall relapse-free survival, a longer median time to first relapse or death, and fewer relapses from baseline through Week 52. Genentech reported that no new safety signals were observed.

Safety and Regulatory Designations

Gazyva’s prescribing information carries a boxed warning for hepatitis B virus reactivation and progressive multifocal leukoencephalopathy, a rare and serious brain infection caused by a virus in people with weakened immune systems. The most common side effects reported in patients with INS include infections, infusion-related reactions, and neutropenia.

Gazyva received Breakthrough Therapy, Orphan Drug, and Priority Review designations for this approval. Genentech described the INS approval as the second FDA approval of Gazyva for an immune-mediated disease, following its approval for adults with active lupus nephritis in 2025.

Genentech also described Gazyva as the first FDA-approved treatment option for idiopathic nephrotic syndrome in 70 years.

With this approval, Gazyva provides a new treatment option for reducing relapse risk in children and adults with childhood-onset, frequently relapsing or steroid-dependent INS who are in complete remission.

Reference

FDA Approves Gazyva for Idiopathic Nephrotic Syndrome in Patients Aged 2 And Older, Genentech, 25 September 2026

FDA Approves Drug to Treat Idiopathic Nephrotic Syndrome in Patients 2 Years and Older, US FDA, 25 September 2026

A Study to Evaluate the Efficacy and Safety of Obinutuzumab Versus MMF in Participants with Childhood Onset Idiopathic Nephrotic Syndrome (INShore), ClinicalTrials.gov ID NCT05627557