The FDA has expanded the approval of obinutuzumab (Gazyva) to reduce the risk of relapse in patients aged 2 years and older with frequently relapsing or steroid-dependent childhood-onset idiopathic nephrotic syndrome who are in complete remission. The decision marks the first new treatment option for this rare autoimmune kidney disease in seven decades, according to the drugmaker Genentech.
The approval is supported by the open-label phase III INShore study, which enrolled 85 participants. A greater proportion of patients treated with obinutuzumab achieved complete remission at week 52 compared with those receiving the standard-of-care oral mycophenolate mofetil (95% vs 73%). The anti-CD20 antibody also produced statistically significant improvements in relapse-free survival, median time to first relapse or death, and the number of relapses from baseline to week 52.
The drug's label includes boxed warnings about hepatitis B virus reactivation and progressive multifocal leukoencephalopathy, along with precautions about infusion-related reactions, serious infections, neutropenia, thrombocytopenia, and embryo-fetal toxicity. The most common adverse events observed in the trial were respiratory tract infections, infusion-related reactions, ear infections, pneumonia, influenza, neutropenia, bronchitis, COVID, conjunctivitis, and urinary tract infection. This is the second approval of obinutuzumab for an autoimmune condition, following its earlier approval in adult lupus nephritis, and it remains under review for other kidney and autoimmune indications.