Positive phase III results for Roche’s Gazyva/Gazyvaro in children and young adults with idiopathic nephrotic syndrome

Core Insights - Roche announced statistically significant and clinically meaningful results from the phase III INShore study of Gazyva®/Gazyvaro® (obinutuzumab) in children and young adults with idiopathic nephrotic syndrome (INS), achieving its primary endpoint of sustained complete remission at one year [1][8] - The study demonstrated that Gazyva/Gazyvaro may reduce the need for corticosteroids, which are associated with serious side effects, addressing an unmet need in the treatment of INS [2][12] - Key secondary endpoints showed significant benefits, including increased overall relapse-free survival and reduced corticosteroid dosage compared to mycophenolate mofetil (MMF) [3][8] Study Details - The INShore study was a phase III open-label, randomized, multicenter trial involving 85 participants aged 2-25 years, comparing Gazyva/Gazyvaro to MMF [10] - The primary endpoint was the percentage of participants achieving sustained complete remission at week 52, defined by the absence of relapses and low protein levels in urine [10][11] Treatment Context - Idiopathic nephrotic syndrome is a severe chronic kidney disease typically diagnosed in early childhood, with high relapse rates (>70%) and limited treatment options primarily relying on steroids [11][12] - Gazyva/Gazyvaro targets B cells, which are believed to drive disease activity, offering a new approach to managing symptoms effectively [2][4] Regulatory Status - Gazyva/Gazyvaro has been approved in the US for adults with active lupus nephritis and is under review in the European Union, with a decision expected soon [7][8] - The drug is also being investigated for other conditions, including membranous nephropathy and systemic lupus erythematosus, as part of Roche's strategy to lead in immune-mediated kidney diseases [5][6]