Written By: Sheetal Barbade, BPharm
Reviewed By: Pharmacally Editorial Team
Roche has announced positive Phase III results from the global INShore study evaluating its targeted therapy Gazyva (obinutuzumab) in children and young adults aged 2 to 25 years with idiopathic nephrotic syndrome (INS), a chronic kidney disease predominantly diagnosed in early childhood. The study demonstrated that Gazyva achieved a statistically significant and clinically meaningful improvement in sustained complete remission (SCR) at one year (week 52) compared to the standard immunosuppressant comparator, mycophenolate mofetil (MMF). Sustained complete remission was defined as the absence of relapses during the study accompanied by a low protein-to-creatinine ratio of 0.2 or less in urine at week 52.
Clinical Evidence
The INShore Trial was a global, randomized, double-blind, controlled Phase III study evaluating the efficacy and safety of Gazyva (obinutuzumab) in children and young adults aged 2 to 25 years diagnosed with idiopathic nephrotic syndrome (INS). The trial enrolled 85 participants with active disease who had a history of relapses despite standard treatment, primarily corticosteroids, and immunosuppressive therapy. Participants were randomised 1:1 to receive Gazyva at weeks 0, 2, 24 and 26, or MMF daily. The primary endpoint was the proportion of patients achieving sustained complete remission (SCR) at week 52, which was defined as the absence of relapses during the study period along with a urine protein-to-creatinine ratio of 0.2 or less at week 52. Key secondary endpoints included relapse-free survival (RFS), median time to relapse or death, cumulative corticosteroid dose reduction, and the number of relapses during the trial period.
Results and Safety Profile
The INShore study met its primary endpoint, demonstrating that a significantly higher proportion of children and young adults treated with Gazyva achieved sustained remission at week 52 compared to those on mycophenolate mofetil (MMF). Secondary endpoints also showed substantial benefits for Gazyva, including increased relapse-free survival, longer median time to relapse, a reduction in corticosteroid use, and fewer relapses overall. Importantly, safety data indicated that
Gazyva’s safety profile was consistent with its known profile in adults, with no new safety signals or adverse effects identified during the trial. The tolerability observed in this younger population supports Gazyva’s potential as a safe and effective targeted therapy for idiopathic nephrotic syndrome in children and young adults, reducing the reliance on steroids and their associated long-term side effects.
Key Opinions
Roche’s Chief Medical Officer, Dr. Levi Garraway, highlighted that Gazyva offers strong disease control while reducing the need for corticosteroids, which carry serious long-term side effects. He noted idiopathic nephrotic syndrome is a severe, chronic kidney disease primarily diagnosed in early childhood with limited treatment options. As a targeted therapy, Gazyva has the potential to address this significant unmet medical need, and Roche is preparing to share these promising data with health authorities for regulatory review.
About Gazyva (Obinutuzumab)
Gazyva (obinutuzumab) is a glycoengineered, humanized type II anti-CD20 monoclonal antibody designed to selectively target and deplete CD20-positive B lymphocytes, which play a central role in driving autoimmune inflammation and tissue damage. Originally approved for hematologic cancers such as chronic lymphocytic leukemia and follicular lymphoma, Gazyva’s therapeutic applications have now expanded to autoimmune kidney diseases like idiopathic nephrotic syndrome (INS). By effectively eliminating pathogenic B cells responsible for immune complex formation and renal inflammation, Gazyva disrupts the harmful cycle of immune-mediated kidney injury. This targeted mechanism helps preserve kidney function while maintaining overall immune system balance, offering a promising new treatment approach for patients with this severe and chronic kidney condition.
About Idiopathic Nephrotic Syndrome
Idiopathic nephrotic syndrome (INS) is a kidney disorder characterized by damage to the glomeruli the tiny filtering units in the kidneys that causes excessive protein loss in the urine. The term “idiopathic” indicates that the cause of the condition is unknown. INS primarily affects children, especially between 2 and 7 years of age, and is the most common cause of nephrotic syndrome in this group. The damage to the kidney filtering barrier leads to protein leakage, resulting in low protein levels in the blood (hypoalbuminemia), swelling (edema), particularly in the face, legs, and around the eyes, and elevated blood lipid levels (hyperlipidemia).
References
Positive phase III results for Roche’s Gazyva/Gazyvaro in children and young adults with idiopathic nephrotic syndrome, Roche, 28 October 2025, https://www.roche.com/media/releases/med-cor-2025-10-28
A Study to Evaluate the Efficacy and Safety of Obinutuzumab Versus MMF in Participants With Childhood Onset Idiopathic Nephrotic Syndrome (INShore), ClinicalTrials.gov ID NCT05627557, https://clinicaltrials.gov/study/NCT05627557
Dossier C, et al, Obinutuzumab in Frequently Relapsing and Steroid-Dependent Nephrotic Syndrome in Children, Clin J Am Soc Nephrol, 2023 Dec 1;18(12):1555-1562, doi: 10.2215/CJN.0000000000000288. Epub 2023 Sep 6. PMID: 37678236; PMCID: PMC10723910.
FDA Approves Gazyva for Lupus Nephritis, Extending Roche and Biogen’s CD20 Therapy Beyond Cancer, 22 Oct 2025, Pharmacally, https://pharmacally.com/fda-approves-gazyva-for-lupus-nephritis-extending-roche-and-biogens-cd20-therapy-beyond-cancer/
GAZYVA® (obinutuzumab) injection, for intravenous use, Highlights Of Prescribing Information, https://www.accessdata.fda.gov/drugsatfda_docs/label/2022/125486s029lbl.pdf