FDA Approves Obinutuzumab (Gazyva) for Idiopathic Nephrotic Syndrome

The US Food and Drug Administration (FDA) has approved obinutuzumab (Gazyva) for idiopathic nephrotic syndrome (INS), a rare kidney disease usually diagnosed in early childhood.¹ Genentech said it is the first FDA approved treatment for INS in 70 years.

The approval covers adult and pediatric patients aged ≥2 years with frequently relapsing or steroid dependent childhood onset INS who are in complete remission, with the goal of reducing the risk of relapse. It is based on the phase 3 INShore trial. In that trial, 95% of patients treated with obinutuzumab had no relapses after week 8 and were in complete remission at week 52, compared with 73% of those receiving mycophenolate mofetil (MMF).¹

"Gazyva is the first FDA-approved treatment option for idiopathic nephrotic syndrome in 70 years, introducing a substantial advancement for children and families who have long relied on steroids and faced high rates of relapse," Levi Garraway, MD, PhD, chief medical officer and head of Global Product Development at Genentech, said in a statement.¹

INShore Trial Design: Obinutuzumab vs MMF in Steroid Dependent INS

INShore was a randomized, open label phase 3 trial. It included 85 children and young adults aged 2 to 25 years who were in clinical remission and had frequently relapsing or steroid dependent nephrotic syndrome.² Patients were randomized to obinutuzumab or MMF.

Obinutuzumab is a CD20 directed monoclonal antibody that targets B cells. It was previously approved for chronic lymphocytic leukemia, follicular lymphoma, and active lupus nephritis.²

Obinutuzumab Achieves 95.5% Sustained Complete Remission at Week 52

Previously reported INShore data showed sustained complete remission at week 52 in 95.5% of patients treated with obinutuzumab vs 73.2% of those receiving MMF. The adjusted difference was 23.39% (95% CI, 6.85 to 38.90; P = .0031).²

According to Genentech, key secondary endpoints also favored obinutuzumab over MMF:¹

• Overall relapse free survival (RFS)
• Median time to first relapse or death
• Fewer relapses from baseline to week 52

The company reported no new safety signals.

Obinutuzumab Safety in INS: Infections and Infusion Reactions

The most common adverse events in childhood onset INS were:¹

• Upper respiratory tract infection
• Infusion related reactions
• Ear infection
• Pneumonia
• Influenza
• Neutropenia
• Bronchitis
• COVID-19
• Conjunctivitis
• Urinary tract infection

Patients taking obinutuzumab may be at greater risk of severe infections than those taking MMF. Patients with an active infection should not be treated.¹

The prescribing information includes warnings for hepatitis B virus (HBV) reactivation and progressive multifocal leukoencephalopathy (PML). Patients must be screened for HBV before treatment and monitored during and after treatment. Severe neutropenia can develop during or after treatment, and some cases last >1 month.¹

Live vaccines should not be given during treatment. In childhood onset INS, live vaccines should be received ≥28 days before starting obinutuzumab.¹

INS Relapse Rates Exceed 70% With Steroid Based Treatment

INS is the most common form of primary glomerular disease in children. It is marked by unpredictable relapses that cause:¹

• Fatigue, swelling, and weight gain
• Increased susceptibility to infections and clotting
• Anxiety, depression, and reduced self esteem

Steroids remain the mainstay of treatment. However, relapse rates are >70%, and steroid side effects can limit long term use.¹

"For too long, patients living with INS have had to endure unpredictable relapses, prolonged steroid use, and the constant threat of disease progression," Andi Callaway, founder and president of The Nephrotic Syndrome Foundation, said in a statement.¹ "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."

Obinutuzumab Pipeline: Membranous Nephropathy and SLE Decisions Ahead

This is the second FDA approval of obinutuzumab in immune mediated disease, following its approval in adult lupus nephritis in 2025.¹ The FDA granted Breakthrough Therapy Designation for both conditions and granted Priority Review for INS.

Obinutuzumab is also under FDA review for two more indications:¹

• Primary membranous nephropathy, based on the phase 3 MAJESTY trial, with a decision expected by November 2026³
• Systemic lupus erythematosus (SLE), based on the phase 3 ALLEGORY trial, with a decision expected by December 2026⁴

It is also being investigated in pediatric lupus nephritis.¹

References

1. Genentech. FDA approves Gazyva for idiopathic nephrotic syndrome in patients aged 2 and older. Published September 25, 2026. https://www.gene.com/media/statements/ps_092526

2. Obinutuzumab achieves 95.5% remission in INS, may shift steroid-sparing treatment strategies. HCPLive. Published June 25, 2026. https://www.hcplive.com/view/obinutuzumab-95-remission-ins-steroid-sparing-treatment-strategies

3. FDA grants priority review to Genentech's Gazyva for adults with primary membranous nephropathy. Genentech. Published July 14, 2026. https://www.gene.com/media/press-releases/15121/2026-07-14/fda-grants-priority-review-to-genentechs

4. FDA accepts application for Genentech's Gazyva for the treatment of the most common form of lupus. Genentech. Published April 20, 2026. https://www.gene.com/media/press-releases/15107/2026-04-20/fda-accepts-application-for-genentechs-g