Positive phase III results for Roche’s Gazyva/Gazyvaro in children and young adults with idiopathic nephrotic syndrome
Roche (OTCQX: RHHBY) reported positive phase III INShore results for Gazyva/Gazyvaro (obinutuzumab) in children and young adults (aged 2–25) with idiopathic nephrotic syndrome.
The study met its primary endpoint: more participants achieved sustained complete remission at week 52 versus mycophenolate mofetil (MMF). Key secondary endpoints met included improved relapse-free survival, longer median time to relapse or death, reduced cumulative corticosteroid dose to week 52, and fewer relapses versus MMF. No new safety signals were identified. Data will be presented at a medical meeting and shared with FDA and EMA.
Roche (OTCQX: RHHBY) ha riportato risultati positivi di fase III INShore per Gazyva/Gazyvaro (obinutuzumab) in bambini e giovani adulti (età 2–25) con sindrome nefrotica idiopatica.
Lo studio ha raggiunto l'endpoint primario: un numero maggiore di partecipanti ha ottenuto una remissione completa sostenuta alla settimana 52 rispetto al mycophenolate mofetil (MMF). Sono stati raggiunti anche i principali endpoint secondari, tra cui un miglioramento della sopravvivenza senza ricadute, un tempo mediano più lungo fino alla ricaduta o alla morte, una redu za della dose cumulativa di corticosteroidi fino alla settimana 52 e meno ricadute rispetto a MMF. Non sono stati identificati nuovi segnali di sicurezza. I dati saranno presentati a un congresso medico e saranno condivisi con la FDA e l'EMA.
Roche (OTCQX: RHHBY) informó resultados positivos de fase III INShore para Gazyva/Gazyvaro (obinutuzumab) en niños y jóvenes adultos (de 2 a 25 años) con síndrome nefrótico idiopático.
El estudio cumplió su objetivo primario: un mayor número de participantes logró una remisión completa sostenida en la semana 52 frente al mofetilo de micofenolato (MMF). Se cumplieron también los principales endpoints secundarios, incluyendo una mejor supervivencia libre de recaídas, un tiempo mediano hasta la recaída o la muerte más largo, una dosis acumulativa de corticosteroides reducida hasta la semana 52 y menos recaídas frente a MMF. No se identificaron nuevas señales de seguridad. Los datos se presentarán en una reunión médica y se compartirán con la FDA y la EMA.
로체(Roche, OTCQX: RHHBY)는 소아 및 청년(2~25세)에서 원인 불명의 부종성 신증후군에 대해 Gazyva/Gazyvaro(obinutuzumab)의 III상 INShore 연구가 긍정적이라고 발표했습니다.
연구는 주요 종료점를 달성했습니다: MMF(mycophenolate mofetil) 대비 52주 차에 지속적 완전 관해를 달성한 참가자 수가 더 많았습니다. 주요 2차 종료점도 충족되었으며, 재발 자유 생존이 개선되고, 재발 또는 사망까지의 중위 시간이 더 길었으며, 52주 차까지 코르티코스테로이드 누적 용량이 감소하고 MMF 대비 재발이 더 적었습니다. 새로운 안전 신호는 확인되지 않았습니다. 데이터는 의학회에서 발표되고 FDA 및 EMA와 공유될 예정입니다.
Roche (OTCQX: RHHBY) a annoncé des résultats positifs de phase III INShore pour Gazyva/Gazyvaro (obinutuzumab) chez des enfants et jeunes adultes (âgés de 2 à 25 ans) atteints du syndrome néphrotique idiopathique.
L’étude a atteint son objectif primaire : un plus grand nombre de participants a atteint une rémission complète soutenue à la semaine 52 par rapport au moférolate de mycophénolate (MMF). Les principaux endpoints secondaires ont également été atteints, notamment une survie sans rechute améliorée, un temps médian jusqu’à la rechute ou au décès plus long, une dose cumulée de corticostéroïdes réduite jusqu’à la semaine 52 et moins de rechutes par rapport à MMF. Aucun nouveau signal de sécurité n’a été identifié. Les données seront présentées lors d’une conférence médicale et communiquées à la FDA et à l’EMA.
Roche (OTCQX: RHHBY) meldete positive Phase-III-INShore-Ergebnisse für Gazyva/Gazyvaro (Obinutuzumab) bei Kindern und jungen Erwachsenen (2–25 Jahre) mit idiopathischem nephrotischem Syndrom.
Die Studie erreichte ihren Primärer Endpunkt: Mehr Teilnehmer erreichten eine nachhaltige vollständige Remission in Woche 52 im Vergleich zu Mycophenolate mofetil (MMF).Wichtige sekundäre Endpunkte wurden ebenfalls erfüllt, darunter eine verbesserte relapse-free survival, eine längere mittlere Zeit bis zum Relapse oder Tod, eine reduzierte kumulative Kortikosteroid-Dosis bis Woche 52 und weniger Relapses im Vergleich zu MMF. Es wurden keine neuen Sicherheitssignale identifiziert. Die Daten werden auf einer medizinischen Tagung präsentiert und mit der FDA und der EMA geteilt.
روش (OTCQX: RHHBY) أبلغت عن نتائج إيجابية في المرحلة الثالثة INShore لـ Gazyva/Gazyvaro (obinutuzumab) لدى الأطفال والشباب (من عمر 2 إلى 25 عامًا) المصابين بمتلازمة نفروتيك غير معروفة السبب.
حققت الدراسة الهدف الأساسي: أصبح عدد المشاركين الذين حققوا تحسناً كاملاً مستمراً عند الأسبوع 52 أكبر مقارنةً بـ MMF (ميكوفينولات ميفل). كما تم تحقيق الأهداف الثانوية الرئيسية، بما في ذلك تحسين البقاء خالٍ من الانتكاسات، وزمن وسيط أطول حتى الانتكاسة أو الوفاة، وانخفاض الجرعة التراكمية للكورتيكوستيرويدات حتى الأسبوع 52، وأقل عدد من الانتكاسات مقارنة بـ MMF. لم تُعرَف أي إشارات أمان جديدة. ستُعرض البيانات في اجتماع طبي وتُشارك مع هيئة الغذاء والدواء الأمريكية (FDA) والوكالة الأوروبية للأدوية (EMA).
罗氏公司 (OTCQX: RHHBY) 报告称，Gazyva/Gazyvaro (obinutuzumab) 在2–25岁儿童及年轻成人的特发性肾病综合征中取得III期INShore研究的积极结果。
该研究达到了其主要终点：52周时实现持续性完全缓解的参与者数量多于MMF（霉酚酸甲酯）组。主要的次要终点也达到预期，包括无复发生存的改善、至复发或死亡的中位时间更长、至52周的累积糖皮质激素用量下降，以及相比MMF的复发更少。未发现新的安全信号。数据将在医学会议上公布，并与FDA和EMA分享。
- 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
- 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 disease1
- INShore is the first global phase III study of a targeted therapy in this chronic kidney disease commonly diagnosed in early childhood
Basel, 28 October 2025 - Roche (SIX: RO, ROG; OTCQX: RHHBY) announced today statistically significant and clinically meaningful results from the phase III INShore study of Gazyva®/Gazyvaro® (obinutuzumab) in children and young adults (aged >= 2-25 years) with idiopathic nephrotic syndrome (INS). The study met its primary endpoint, with more people achieving sustained complete remission at one year (week 52) with Gazyva/Gazyvaro compared with mycophenolate mofetil (MMF). Sustained complete remission was defined by the absence of relapses during the study together with a low amount of protein in the urine (protein to creatine of 0.2 or less) at week 52.1 Certain important key secondary endpoints were also met. No new safety signals were identified and safety was in line with the well-characterised profile of Gazyva/Gazyvaro in adults.
“These results show that Gazyva/Gazyvaro may achieve robust disease control with a reduced need for corticosteroids, which are associated with serious side effects over time,” said Levi Garraway, MD, PhD, Roche’s Chief Medical Officer and Head of Global Product Development. “Idiopathic nephrotic syndrome is a severe and chronic kidney disease usually diagnosed in early childhood, yet meaningful treatment progress has been limited. As a targeted therapy, Gazyva/Gazyvaro has the potential to help address this unmet need and we look forward to sharing the data with health authorities.”
For several decades, treatment for idiopathic nephrotic syndrome has primarily relied on steroids yet relapse rates remain high and long-term use is limited by serious side effects.2-4 Newer approaches that target specific immune cells - such as B cells, thought to be a key driver of disease activity - may help control symptoms more effectively.5,6
Analysis of key secondary endpoints showed statistically significant and clinically meaningful benefits with Gazyva/Gazyvaro with an increase in those with overall relapse-free survival (RFS), median time to relapse or death, reduction in cumulative corticosteroid dose from baseline to week 52, and fewer relapses from baseline to week 52, all compared with MMF. Other key secondary endpoints showed no significant difference with Gazyva/Gazyvaro versus MMF.*
Data will be presented at an upcoming medical meeting and shared with health authorities, including the US Food and Drug Administration and the European Medicines Agency.
INShore data add to a growing body of evidence, including the phase III REGENCY study in lupus nephritis, that shows targeting disease-causing B cells with Gazyva/Gazyvaro may help address disease activity across a spectrum of immune-mediated kidney and kidney-related diseases.7-10 In October 2025, Gazyva/Gazyvaro was approved in the US for the treatment of adults with active lupus nephritis who are receiving standard therapy, based on data from the phase III REGENCY and phase II NOBILITY studies.
In addition to idiopathic nephrotic syndrome, Gazyva/Gazyvaro is being investigated in membranous nephropathy, lupus nephritis, rare immune-mediated kidney diseases and systemic lupus erythematosus an autoimmune disease that can lead to lupus nephritis, as part of our ambition to be leaders in immune-mediated kidney and kidney-related diseases.11-13
About Gazyva/Gazyvaro
Gazyva®/Gazyvaro® (obinutuzumab) is a Type II engineered humanised monoclonal antibody designed to attach to CD20, a protein found on certain types of B cells.14
Gazyva/Gazyvaro is approved for adults with lupus nephritis who are receiving standard therapy in the US. In October 2025, the European Medicines Agency’s Committee for Medicinal Products for Human Use recommended approval in the European Union, with a final decision expected from the European Commission in the near future. Gazyva/Gazyvaro is also approved in 100 countries for various types of haematological cancers.
In the US, Gazyva is part of a collaboration between Genentech and Biogen.
About the INShore study
INShore [NCT05627557] is a phase III open-label, randomised, multicentre study investigating Gazyva®/Gazyvaro® (obinutuzumab) compared with mycophenolate mofetil (MMF) in children and young adults in clinical remission (aged >=2-25 years) with frequently relapsing or steroid-dependent nephrotic syndrome. The study enrolled 85 children or young adults who were randomised 1:1 to receive Gazyva/Gazyvaro at weeks 0,2, 24 and 26, or MMF daily. The primary endpoint is the percentage of participants with sustained complete remission at one year (week 52).
About idiopathic nephrotic syndrome
Idiopathic nephrotic syndrome is a rare kidney-related autoimmune disease, usually diagnosed in early childhood.2 It is characterised by unpredictable relapses that cause fatigue, swelling, weight gain and increased susceptibility to infections and clotting, as well as anxiety, depression and reduced self-esteem, brought on by the fear of relapse and social isolation.2,15 The current mainstay of treatment is steroids, however relapse rates remain high (>
There is an urgent need for new targeted treatment approaches that can sustain remission and reduce the physical and psychosocial burden of the disease.
About Roche in kidney and kidney-related diseases
For more than 20 years, we have combined innovation, scientific expertise and commitment to patients to address unmet needs in kidney diseases. Today, our industry-leading programme includes Gazyva®/Gazyvaro® (obinutuzumab), approved in the US for adults with lupus nephritis, and more than 10 phase II-III clinical studies in immune-mediated kidney and kidney-related diseases with some of the highest unmet needs.
Our aim is to continue delivering meaningful value for those affected, healthcare systems and society, and help address this growing public health burden.
About Roche
Founded in 1896 in Basel, Switzerland, as one of the first industrial manufacturers of branded medicines, Roche has grown into the world’s largest biotechnology company and the global leader in in-vitro diagnostics. The company pursues scientific excellence to discover and develop medicines and diagnostics for improving and saving the lives of people around the world. We are a pioneer in personalised healthcare and want to further transform how healthcare is delivered to have an even greater impact. To provide the best care for each person we partner with many stakeholders and combine our strengths in Diagnostics and Pharma with data insights from the clinical practice.
For over 125 years, sustainability has been an integral part of Roche’s business. As a science-driven company, our greatest contribution to society is developing innovative medicines and diagnostics that help people live healthier lives. Roche is committed to the Science Based Targets initiative and the Sustainable Markets Initiative to achieve net zero by 2045.
Genentech, in the United States, is a wholly owned member of the Roche Group. Roche is the majority shareholder in Chugai Pharmaceutical, Japan.
For more information, please visit www.roche.com.
All trademarks used or mentioned in this release are protected by law.
*Sustained complete remission (week 76), probability of relapse free survival (week 52), proportion of participants experiencing oedema associated relapse (during 52 week treatment period), mean change in CureGN Edema scale (from baseline to week 52), mean change in “General Fatigue” domain of PedsQL-Multidimensional Fatigue Scale total score (from baseline to week 52), mean change in “Physical Functioning” domain of PedsQL (from baseline to week 52).
References
[1] Clinicaltrials.gov. A Study to Evaluate the Efficacy and Safety of Obinutuzumab Versus MMF in Participants With Childhood Onset Idiopathic Nephrotic Syndrome (INShore). [Internet; cited 2025 October 27]. Available from: https://clinicaltrials.gov/study/NCT05627557?term=inshore&rank=1
[2] Vivarelli M, et al. Childhood nephrotic syndrome. Lancet. 2023 Sep 2;402(10404):809-24.
[3] Noone DG, et al. Idiopathic nephrotic syndrome in children. Lancet. 2018 Jul 7;392(10141):61-74.
[4] Canetta PAA ,et al. The Evidence-Based Approach to Adult-Onset Idiopathic Nephrotic Syndrome. Front Pediatr. 2015 Sep 25:3:78.
[5] Colucci M, et al. B-Cell Dysregulation in Idiopathic Nephrotic Syndrome: What We Know and What We Need to Discover. Front Immunol. 2022 Jan 24;13:823204.
[6] Al-Aubodah TA, et al. The extrafollicular B cell response is a hallmark of childhood idiopathic nephrotic syndrome. Nat Commun. 2023 Nov 24;14(1):7682.
[7] Furie RA, et al. B-cell depletion with obinutuzumab for the treatment of proliferative lupus nephritis: a randomised, double-blind, placebo-controlled trial. Ann Rheum Dis. 2022 Jan;81(1):100-07.
[8] Furie RA, et al. Efficacy and safety of obinutuzumab in active lupus nephritis. N Engl J Med. 2025 Feb;392:1471-83.
[9] Arnold J, et al. Efficacy and safety of obinutuzumab in systemic lupus erythematosus patients with secondary non-response to rituximab. Rheumatology (Oxford). 2022 Nov 28;61(12):4905-09.
[10] Cheng X, et al. Efficacy and safety of obinutuzumab in primary membranous nephropathy: a real-world retrospective study. Front Immunol. 2025 Aug 21;16:1650054.
[11] Clinicaltrials.gov. A study evaluating the efficacy and safety of obinutuzumab in participants with primary membranous nephropathy (MAJESTY). [Internet; cited 2025 October 27]. Available from: https://clinicaltrials.gov/study/NCT04629248
[12] Clinicaltrials.gov. A Study To Evaluate The Efficacy And Safety Of Obinutuzumab In Patients With ISN/RPS 2003 Class III Or IV Lupus Nephritis (REGENCY). [Internet; cited 2025 October 22]. Available from: https://clinicaltrials.gov/study/NCT04221477
[13 Clinicaltrials.gov. A study to evaluate the efficacy and safety of obinutuzumab in participants with systemic lupus erythematosus (ALLEGORY). [Internet; cited 2025 October 27]. Available from: https://clinicaltrials.gov/study/NCT04963296.
[14] Herter S, et al. Preclinical activity of the type II CD20 antibody GA101 (obinutuzumab) compared with rituximab and ofatumumab in vitro and in xenograft models. Mol Cancer Ther. 2013 Oct;12(10):2031-42.
[15] Shukla J, et al. Quality of Life of Children with Idiopathic Nephrotic Syndrome. Indian J Nephrol. 2025 Feb 25;35(2):234–42
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FAQ
What did Roche announce about RHHBY and Gazyva/Gazyvaro on October 28, 2025?
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