FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

The FDA has approved obinutuzumab (Gazyva, Genentech) injection to reduce the risk of relapse in patients 2 years and older with childhood-onset idiopathic nephrotic syndrome (INS) who are in remission. The indication covers adults and children whose disease is frequently relapsing or steroid-dependent. Obinutuzumab was already approved for certain cancers and for adults with active lupus nephritis who are receiving standard therapy.

The FDA describes INS as a kidney disorder of unknown cause. Large amounts of protein leak into the urine, which lowers blood albumin and often causes swelling. Complications can include high cholesterol, infections, and blood clots. INS is the most common cause of nephrotic syndrome in children and usually starts between ages 2 and 7 years. According to Genentech, steroids are the mainstay of treatment, but relapse rates are above 70%. The company called obinutuzumab the first FDA-approved treatment for INS in 70 years.

The approval was based on INShore (NCT05627557), a phase 3, randomized, open-label, multicenter trial in 85 patients who were in complete remission at enrollment. Patients received either weight-based intravenous obinutuzumab on days 1 and 15 and weeks 24 and 26, or twice-daily oral mycophenolate mofetil (MMF). The primary endpoint was a first-morning urine protein-creatinine ratio of 0.2 g/g or less at week 52, with no relapse from week 8 to week 52. Genentech reported that 95% of patients in the obinutuzumab group met this endpoint, compared with 73% in the MMF group. The FDA called the difference significant. Genentech also reported statistically significant benefits on key secondary endpoints, including relapse-free survival, time to first relapse or death, and number of relapses through week 52.

The obinutuzumab label has a boxed warning for hepatitis B virus reactivation and progressive multifocal leukoencephalopathy. The FDA said the most common adverse reactions in INS were infections, infusion-related reactions, and neutropenia. Genentech's safety information notes a possibly higher risk of severe infection than with MMF, and says live vaccines should be given at least 28 days before treatment. The company reported no new safety signals.

The approval carried Breakthrough Therapy, Orphan Drug, and Priority Review designations. According to Genentech, obinutuzumab is under FDA review for primary membranous nephropathy and systemic lupus erythematosus, and is being studied in pediatric lupus nephritis.

Sources: US Food and Drug Administration. FDA approves drug to treat idiopathic nephrotic syndrome in patients 2 years and older. News release. US Food and Drug Administration; September 25, 2026. Accessed September 28, 2026.

Genentech. FDA approves Gazyva for idiopathic nephrotic syndrome in patients aged 2 and older. Press release. Genentech; September 25, 2026. Accessed September 28, 2026.