Idiopathic nephrotic syndrome (INS), a chronic autoimmune kidney disease most often diagnosed in early childhood, remains without an FDA-approved therapy, leaving pediatric patients dependent on long-term corticosteroids to manage relapsing disease.¹ Chronic steroid exposure carries substantial toxicity in this population, and children with frequently relapsing or steroid-dependent INS remain at risk of progression to end-stage kidney disease.¹ This month, the FDA granted priority review to obinutuzumab (Gazyva; Genentech), a CD20-targeted monoclonal antibody, for the treatment of INS in children and young adults, building on the breakthrough therapy designation the agent received from the FDA in January 2026.²

The filing is supported by data from the phase 3 INShore trial, which evaluated obinutuzumab against mycophenolate mofetil in children and young adults with relapsing or steroid-dependent INS and found that significantly more patients achieved sustained complete remission at 52 weeks with obinutuzumab.² A regulatory decision is expected by September 2026.² To discuss what an approval could mean for pediatric management of this rare disease, Contemporary Pediatrics spoke with Keisha L. Gibson, MD, MPH, a pediatric nephrologist at the University of North Carolina School of Medicine, about the unmet need in INS, the INShore findings, and how a CD20-targeted therapy might reshape the treatment paradigm for children who currently have no approved options.

Q&A

Contemporary Pediatrics: Idiopathic nephrotic syndrome is often managed with repeated courses of steroids. What are the biggest challenges clinicians and families face with current treatment approaches, and where are the greatest unmet needs?

Keisha L. Gibson, MD, MPH: The repeated and prolonged use of steroids is a double-edged sword. Although responding to steroids signals a good long-term prognosis, the journey is incredibly difficult for families. Patients struggle with significant weight gain, changes in appearance, mood, and sleep disturbances, and growth restriction. Furthermore, as pediatricians, we constantly worry about the long-term risks of hypertension, diabetes, and early cardiovascular disease. Our critical priority is finding a treatment that delivers long-term, steroid-free remission with a minimal side-effect profile.

Contemporary Pediatrics: The phase 3 INShore study showed higher rates of sustained complete remission with obinutuzumab compared with mycophenolate mofetil. What findings from the trial do you think are most clinically meaningful for pediatric nephrologists?

Gibson: Pediatric nephrologists want to know that the amount of time our patients with nephrotic syndrome can stay off steroids is at least equal to or better than mycophenolate mofetil and they want to be able to assure families the treatment is safe.

Contemporary Pediatrics: Reducing steroid exposure is a major goal in children with relapsing or steroid-dependent INS. How could a therapy like obinutuzumab change the way clinicians think about long-term disease management?

Gibson: Our community is highly sensitive to the need to think of our patients not just as the young children they are today, but as young people who need our support to transition into adulthood in the healthiest state possible. Right now, managing idiopathic nephrotic syndrome can feel like a series of short-term fires we are constantly putting out with steroids. A targeted therapy like obinutuzumab changes the landscape. By offering longer periods of sustained remission, it allows us to protect these children from the cumulative physical and psychological trauma of chronic steroid exposure. It gives pediatric nephrologists a tool to think beyond the next relapse and allows us to focus on long-term milestones like linear growth, metabolic health, and uninterrupted schooling, ultimately helping these children grow into thriving, healthy adults.

Contemporary Pediatrics: If obinutuzumab receives FDA approval, which patients do you anticipate could benefit most, and how might it fit into current treatment algorithms for INS?

Gibson: Any patient with frequently relapsing or steroid-dependent nephrotic syndrome could benefit from this treatment. Families that find it an impossible task to consistently get their children to take an oral medication every day would find a benefit in an infusion-based therapy like this.

Contemporary Pediatrics: Looking ahead, what impact could the availability of an FDA-approved therapy have on outcomes for children with INS, including disease remission, quality of life, and the risk of progression to more advanced kidney disease?

Gibson: When we look at long-term outcomes for children with steroid-sensitive INS, the good news is that their long-term kidney prognosis is largely excellent. The bitter irony is that the biggest threat to their future health isn't the disease progressing but the cumulative side effects of the therapies we use to treat it. Moreover, much of what we have to offer is off-label (i.e., not FDA-approved). Having an FDA-approved therapy shifts us from the guesswork of off-label dosing to a standardized, rigorously validated treatment path, providing clinicians with predictable safety profiles and families with peace of mind.