The FDA has approved Roche's obinutuzumab (Gazyva) for patients aged 2 and older with frequently relapsing or steroid-dependent idiopathic nephrotic syndrome (INS), marking the first new treatment for the rare autoimmune kidney disease in 70 years. In the phase III INShore trial, 95% of patients on Gazyva achieved complete remission at week 52, significantly outperforming the standard-of-care mycophenolate mofetil. This approval cements Gazyva's commercial pivot from oncology into autoimmune diseases.
FDA approval of obinutuzumab
- ▪The FDA approved obinutuzumab (Gazyva) on September 25, 2026, to reduce the risk of relapse in patients aged 2 and older with frequently relapsing or steroid-dependent childhood-onset idiopathic nephrotic syndrome in complete remission.
- ▪The FDA approval of obinutuzumab for idiopathic nephrotic syndrome represents the first new treatment option approved for the rare kidney disease in 70 years.
INShore study efficacy results
- ▪The phase III INShore study met secondary endpoints, showing statistically significant improvements in overall relapse-free survival, median time to first relapse or death, and fewer relapses from baseline to week 52.
- ▪In the phase III INShore study, 95% of patients treated with obinutuzumab achieved complete remission at week 52, compared to 73% of patients treated with twice-daily oral mycophenolate mofetil.
Obinutuzumab safety and warnings
- ▪Obinutuzumab prescribing information warns against serious risks including infusion-related reactions, hypersensitivity reactions, tumor lysis syndrome, serious infections, neutropenia, thrombocytopenia, disseminated intravascular coagulation, and embryo-fetal toxicity.
- ▪The prescribing label for obinutuzumab carries a boxed warning regarding the risk of hepatitis B virus reactivation and progressive multifocal leukoencephalopathy.
- ▪Patients receiving obinutuzumab are advised to avoid live virus vaccines until B-cell recovery has occurred.
- ▪The most common adverse events observed with obinutuzumab in the INShore trial include upper respiratory tract infection, infusion-related reactions, ear infection, pneumonia, influenza, neutropenia, bronchitis, COVID, conjunctivitis, and urinary tract infection.
About idiopathic nephrotic syndrome
- ▪Idiopathic nephrotic syndrome is a rare, kidney-related autoimmune disease where large amounts of protein leak into the urine, causing hypoalbuminemia, fatigue, swelling, weight gain, and increased susceptibility to infections.
- ▪Idiopathic nephrotic syndrome is frequently diagnosed in childhood, and its unpredictable relapses can cause anxiety, depression, and reduced self-esteem in young patients.
Other indications of obinutuzumab
- ▪The approval for idiopathic nephrotic syndrome marks the second autoimmune indication for obinutuzumab, following its approval for adult lupus nephritis last October.
- ▪Obinutuzumab was originally approved in 2013 for a leukemia indication and is also indicated to treat follicular lymphoma.
- ▪Obinutuzumab is currently under FDA review for systemic lupus erythematosus and primary membranous nephropathy, and is being investigated for pediatric lupus nephritis.
Debatable claims
- ▪An 85-participant open-label trial is sufficient to justify FDA approval for rare disease treatments
- ▪Obinutuzumab should replace mycophenolate mofetil as the standard treatment for relapsing pediatric kidney disease
- ▪Repurposing older oncology drugs for autoimmune diseases is an effective way to address rare conditions
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