FDA Approves Gazyva for Pediatric Idiopathic Nephrotic Syndrome
核心洞察
The FDA approved obinutuzumab (Gazyva, Genentech) to reduce relapse risk in adults and children aged 2 years and older with frequently relapsing or steroid-dependent childhood-onset idiopathic nephrotic syndrome (搜索) in remission.
The decision is supported by the phase 3 INSHORE trial, which randomized 85 patients to intravenous obinutuzumab or twice-daily oral mycophenolate mofetil.
Obinutuzumab was given on days 1 and 15 and at weeks 24 and 26, with the primary endpoint assessed at week 52.
The FDA has approved obinutuzumab (Gazyva, Genentech) for the treatment of idiopathic nephrotic syndrome (搜索) in pediatric patients aged 2 years and older, expanding the anti-CD20 (搜索) antibody into a new indication. The injection is now indicated to reduce the risk of relapse in adult and pediatric patients with frequently relapsing or steroid-dependent, childhood-onset, idiopathic nephrotic syndrome who are in remission.
Idiopathic nephrotic syndrome (搜索) is characterized by large amounts of protein leaking into urine from the kidneys, leading to edema around the eyes, abdomen, feet, and legs.
INSHORE Trial Design
The approval is supported by positive efficacy data from the phase 3 INSHORE study, a randomized, controlled, open-label, multicenter trial. INSHORE randomly assigned 85 patients aged 2 years or older with idiopathic nephrotic syndrome (搜索) to treatment with either intravenous obinutuzumab or twice-daily oral mycophenolate mofetil (CellCept), the current standard of care in this setting.
Patients assigned to obinutuzumab received injections on days 1 and 15, as well as at weeks 24 and 26.
Primary Endpoint Results
The primary endpoint was the proportion of patients with a first morning urine protein-creatinine ratio of 0.2 g/g or less at week 52, without a relapse from week 8 to week 52. According to the release, the proportion of patients who achieved this endpoint in the Gazyva cohort was significantly greater than in the standard of care cohort. In the INSHORE trial, more patients treated with Gazyva achieved sustained complete remission at week 52 compared with those receiving mycophenolate mofetil.
Broader Gazyva Development Program
Gazyva is already approved for lupus nephritis (搜索) and several leukemia and lymphoma indications. The nephrotic syndrome approval adds a pediatric-onset kidney disease to the drug's label, extending its use in nephrology beyond lupus nephritis.