What did the FDA just approve for children with nephrotic syndrome?

Gazyva reduced relapses and steroid exposure in a phase 3 study of children already in complete remission. It is a treatment option, not a cure.

The FDA approved Gazyva for childhood-onset idiopathic nephrotic syndrome. Genentech describes it as the first FDA-approved treatment option for this condition in 70 years. The INShore phase 3 trial enrolled 85 patients who were already in complete remission: one group received Gazyva by IV infusion, while the other took mycophenolate mofetil twice daily.

At week 52, 95% of the Gazyva group remained in complete remission without a relapse after week 8, compared with 73% in the mycophenolate group; there were four relapses versus 23, and cumulative steroid exposure was lower. The honest takeaway is fewer relapses and potentially less long-term steroid burden in the childhood-onset population studied—not a cure. Families should ask a pediatric nephrologist whether the option fits and should never stop or change medicine on their own.