Gazyva/Gazyvaro Shows Positive Phase III Results in Pediatric and Young Adult Idiopathic Nephrotic Syndrome

Roche’s Gazyva/Gazyvaro Shows Promise in Pediatric Kidney Disease, Phase III Data Reveals

Roche (OTCQX: RHHBY) has announced positive topline results from its Phase III INShore study, evaluating Gazyva/Gazyvaro (obinutuzumab) in children and young adults (ages 2-25) with idiopathic nephrotic syndrome (INS). The study, a critical step in addressing a significant unmet need in pediatric nephrology, met its primary endpoint, demonstrating a statistically significant increase in sustained complete remission at week 52 compared to the current standard of care, mycophenolate mofetil (MMF).

This positive outcome signals a potential paradigm shift in the treatment of INS, a chronic kidney disease often diagnosed in early childhood. The current treatment landscape heavily relies on corticosteroids, which, while effective in managing symptoms, are associated with a high rate of relapse and severe long-term side effects. Gazyva/Gazyvaro, a targeted therapy designed to deplete CD20-positive B cells (thought to play a key role in INS pathogenesis), offers a more precise approach to disease management.

Beyond the primary endpoint, the INShore study also achieved several key secondary endpoints. Researchers observed improvements in relapse-free survival, longer median time to relapse or death, a reduction in the cumulative corticosteroid dose required to maintain remission, and fewer relapses overall, all compared to the MMF arm. Notably, the safety profile of Gazyva/Gazyvaro in this pediatric population remained consistent with its well-characterized safety profile in adults, suggesting a manageable risk-benefit ratio.

However, not all secondary endpoints demonstrated significant improvement over MMF, highlighting the complexities of INS and the need for continued research. Specific endpoints, including sustained complete remission at week 76 and certain measures of edema and fatigue, showed no statistically significant difference between the treatment groups.

The INShore trial design, employing an open-label, randomized, multicenter approach, enrolled 85 patients already in clinical remission but experiencing frequent relapses or steroid dependence. This patient population is particularly vulnerable to the long-term consequences of steroid use, making the prospect of a steroid-sparing therapy particularly appealing.

Dr. Levi Garraway, Roche’s Chief Medical Officer and Head of Global Product Development, emphasized the potential of Gazyva/Gazyvaro to achieve robust disease control while minimizing the need for corticosteroids. The company plans to present the full data set at an upcoming medical meeting and will engage with regulatory agencies, including the FDA and EMA, to discuss potential approval pathways.

This positive data builds upon Roche’s growing portfolio in immune-mediated kidney diseases. The company recently secured FDA approval for Gazyva/Gazyvaro in adults with active lupus nephritis, based on data from the REGENCY and NOBILITY studies. With ongoing investigations in membranous nephropathy, lupus nephritis, and other rare immune-mediated kidney diseases, Roche is positioning itself as a key player in addressing the unmet needs of patients with these challenging conditions.

Analysts will be closely watching the forthcoming presentation of the complete INShore data, paying particular attention to the magnitude of benefit across all endpoints and the potential impact on future treatment guidelines. The success of Gazyva/Gazyvaro in INS could pave the way for a new era of targeted therapies in pediatric nephrology, offering the promise of improved outcomes and a better quality of life for young patients and their families.

10/28/2025 – 02:00 AM

Gazyva/Gazyvaro versus mycophenolate mofetil shows significantly more children and young adults achieved sustained complete remission at week 52.

If approved, Gazyva/Gazyvaro could help children and young adults sustain remission, potentially with a reduced need for steroids to manage their disease.

INShore is the first global phase III study of a targeted therapy in this chronic kidney disease commonly diagnosed in early childhood.

Primary endpoint met: sustained complete remission at week 52 versus MMF

Relapse-free survival increased versus MMF

Median time to relapse or death improved versus MMF

Cumulative corticosteroid dose reduced from baseline to week 52 versus MMF

No new safety signals; safety consistent with adult profile

Regulatory pathway: data to be shared with FDA and EMA

Some key secondary endpoints showed no significant difference versus MMF

What did Roche announce about RHHBY and Gazyva/Gazyvaro on October 28, 2025?
Roche announced positive phase III INShore results showing more children and young adults achieved sustained complete remission at week 52 with Gazyva/Gazyvaro versus MMF.

How did Gazyva/Gazyvaro affect steroid use in the INShore study for RHHBY?
INShore reported a reduction in cumulative corticosteroid dose from baseline to week 52 with Gazyva/Gazyvaro versus MMF.

Did the INShore study for RHHBY raise new safety concerns about Gazyva/Gazyvaro?
No new safety signals were identified; safety was reported as in line with the well‑characterised adult profile.

What are the next regulatory steps for RHHBY after the INShore results?
Roche said it will present INShore data at a medical meeting and share results with FDA and EMA for regulatory review.

Which patient age group was included in the INShore trial for RHHBY’s Gazyva/Gazyvaro?
The trial enrolled children and young adults aged 2–25 years with idiopathic nephrotic syndrome.

Did all secondary endpoints succeed in the INShore study for RHHBY?
No; while several secondary endpoints showed benefit, the release notes that other key secondary endpoints showed no significant difference versus MMF.