Positive Clinical Data for Gazyva
Roche has announced new clinical data showing that its CD20-targeting antibody, Gazyva (obinutuzumab), achieved statistically significant and clinically meaningful improvements in patients with idiopathic nephrotic syndrome (INS). The findings follow Gazyva’s recent FDA approval for lupus nephritis, marking its first non-oncology indication.
INShore Trial Success
According to top-line results from the company’s late-stage INShore trial, Gazyva outperformed the immunosuppressant mycophenolate mofetil (MMF) in helping children and young adults with INS achieve sustained remission after one year of treatment. The trial enrolled 85 participants aged 2 to 25 years, and its primary endpoint was met, Roche’s Genentech unit said in a press release.
While detailed results were not disclosed, Roche stated that full findings would be presented at an upcoming medical meeting. The company also plans to share the data with regulators worldwide, including the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
Background on Gazyva and INS
Gazyva was first approved in 2013 for the treatment of chronic lymphocytic leukemia. Until October 20, all four of its approved uses were in oncology. Roche said Gazyva, by targeting the CD20 protein, could address a range of immune-mediated kidney and kidney-related conditions driven by disease-causing B cells.
Idiopathic nephrotic syndrome, commonly diagnosed in early childhood, is characterized by unpredictable relapses that can cause fatigue, swelling, weight gain, and a higher risk of infection and clotting. Roche added that these relapses may also lead to psychological effects such as anxiety and social withdrawal. Current treatment mainly relies on corticosteroids, but long-term use can lead to serious side effects, and many patients continue to experience relapses.
Detailed Study Outcomes
In the INShore study, Roche defined complete remission as the absence of relapses during the 52-week trial period, combined with low levels of protein in the urine. Gazyva met this criterion and showed additional benefits over MMF across several secondary endpoints, including improved relapse-free survival, longer median time to relapse or death, reduced cumulative corticosteroid exposure, and fewer relapses from baseline to week 52.
However, the company acknowledged that on certain other measures, Gazyva did not demonstrate a statistically significant difference compared to MMF. These endpoints included sustained complete remission, relapse-free survival probability, the proportion of patients with edema-related relapse, and average changes in reported fatigue.
Safety and Future Outlook
No new safety concerns were identified in the study, and Roche reported that Gazyva’s safety profile remained consistent with previous adult studies.
“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., Roche’s chief medical officer and head of global product development. He added that idiopathic nephrotic syndrome is a severe and chronic kidney disease usually diagnosed in early childhood, yet meaningful treatment progress has been limited, and that Gazyva has the potential to help address this unmet need.
Beyond INS and lupus nephritis, Roche is investigating Gazyva’s potential in other immune-mediated kidney conditions, including membranous nephropathy and systemic lupus erythematosus. In 2024, Gazyva generated 910 million Swiss francs (approximately $1.15 billion) in global sales, marking a 16% increase compared with 2023.
