Genentech Reports Positive Phase III Results for Gazyva in Children and Young Adults With Idiopathic Nephrotic Syndrome
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced statistically significant and clinically meaningful findings from its Phase III INShore study evaluating Gazyva® (obinutuzumab) in children and young adults (aged 2–25 years) diagnosed with idiopathic nephrotic syndrome (INS). The late-stage trial met its primary endpoint, demonstrating that a higher proportion of participants treated with Gazyva achieved sustained complete remission at one year (week 52) compared with those receiving mycophenolate mofetil (MMF).
Sustained complete remission in the study was defined by the absence of relapses throughout the treatment period and a protein-to-creatinine ratio of 0.2 or lower at week 52. Gazyva not only met the primary endpoint but also achieved key secondary endpoints, including improvements in relapse-free survival and corticosteroid reduction, without identifying any new safety signals. The safety profile of Gazyva remained consistent with its well-characterized use in adult populations.
These results show that Gazyva may achieve robust disease control with a reduced need for corticosteroids, which are associated with serious side effects over time,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development at Genentech. “Idiopathic nephrotic syndrome is a severe and chronic kidney disease typically diagnosed in early childhood, yet meaningful treatment progress has been limited. As a targeted therapy, Gazyva has the potential to help address this long-standing unmet need, and we look forward to sharing the data with health authorities.
A Major Step Forward in Pediatric Kidney Disease Treatment
Idiopathic nephrotic syndrome is a rare, immune-mediated kidney disorder that most often develops in early childhood. The disease is characterized by significant protein loss through urine, swelling, and increased susceptibility to infections and blood clots. For decades, the standard treatment has relied heavily on corticosteroids, which are effective at controlling initial symptoms but carry the burden of long-term adverse effects, including growth retardation, hypertension, and metabolic complications.
Despite the availability of other immunosuppressive agents, relapse rates remain high, and many patients become dependent on or resistant to steroids over time. This has created a critical need for more effective, targeted approaches that can modify the underlying disease mechanism rather than merely control its symptoms.
Gazyva, a humanized anti-CD20 monoclonal antibody, selectively depletes B cells — a type of immune cell believed to play a central role in driving inflammation and kidney damage in immune-mediated diseases. By precisely targeting B cells, Gazyva may help achieve longer-lasting remission while reducing reliance on non-specific immunosuppressive drugs.
Detailed INShore Study Results
The Phase III INShore study was designed to evaluate the efficacy and safety of Gazyva compared to MMF in pediatric and young adult patients with idiopathic nephrotic syndrome who had previously relapsed or were dependent on corticosteroids. Participants were randomized to receive either Gazyva or MMF, and clinical outcomes were assessed over 52 weeks.
The trial successfully met its primary endpoint, with significantly more participants in the Gazyva arm achieving sustained complete remission at one year compared with MMF. These findings were supported by statistically significant improvements in several key secondary endpoints, including:
- Relapse-Free Survival (RFS): Participants treated with Gazyva experienced a longer duration without relapse or death compared to those on MMF.
- Median Time to Relapse: The median time to disease relapse was substantially extended in the Gazyva group.
- Cumulative Corticosteroid Dose: Gazyva-treated patients required markedly lower cumulative steroid doses from baseline through week 52, suggesting a potential to minimize steroid-related toxicities.
- Number of Relapses: Fewer overall relapses were observed among participants receiving Gazyva versus MMF.
While some secondary endpoints showed no statistically significant difference between treatment arms, the overall clinical profile strongly favored Gazyva, indicating meaningful disease control with a favorable benefit–risk ratio.
Importantly, no new safety signals were detected during the study. The observed safety outcomes were consistent with Gazyva’s established profile in adults treated for other immune-mediated kidney conditions and hematologic malignancies. The most common adverse events were mild to moderate infusion-related reactions, which were manageable with standard supportive care.
Implications and Next Steps
Genentech plans to present the full INShore data at an upcoming medical meeting and submit the results to global health authorities, including the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). These data add to the growing body of evidence supporting Gazyva’s role in treating immune-mediated kidney diseases beyond its existing indications.
The new findings follow the success of Gazyva in the Phase III REGENCY study in lupus nephritis, where the therapy demonstrated significant improvement in kidney outcomes and helped achieve durable disease control when combined with standard therapy. Based on those results, Gazyva received U.S. FDA approval in October 2025 for adults with active lupus nephritis who were receiving standard therapy.
Together, the REGENCY and INShore studies reinforce the potential of B-cell–targeted therapy to transform treatment outcomes across a range of immune-mediated renal disorders. Genentech continues to explore Gazyva’s clinical benefits in other conditions, including membranous nephropathy, systemic lupus erythematosus (SLE), and rare immune-mediated kidney diseases.
The INShore results further expand our understanding of how B-cell depletion therapy can improve kidney health in young patients,” added Garraway. “Our commitment is to develop precision therapies that address the underlying causes of chronic diseases and improve long-term outcomes for patients.
