The US Food and Drug Administration approved obinutuzumab (Gazyva) to reduce the risk of relapse in adult and pediatric patients aged 2 years and older with frequently relapsing or steroid-dependent, childhood-onset, idiopathic nephrotic syndrome who are in remission, according to a press release from the agency.
Idiopathic nephrotic syndrome is characterized by urinary protein loss that lowers blood albumin levels and may result in swelling. Other potential complications include high cholesterol, infections, and blood clots that may obstruct small blood vessels. The kidney disorder, which has no known cause, most commonly accounts for nephrotic syndrome in pediatric patients and generally develops between ages 2 and 7 years. Estimated annual incidence ranges from 1 to 4 cases per 100,000 pediatric patients, with variation by age, race, and geographic region.
The efficacy and safety of obinutuzumab were assessed in INSHORE, a phase 3 randomized open-label, multicenter controlled trial of 85 patients aged 2 years and older with frequently relapsing or steroid-dependent idiopathic nephrotic syndrome that began in childhood. Enrollment was limited to patients in complete remission, which required normal urine protein levels and no swelling.
Patients were randomly assigned to receive-based intravenous obinutuzumab administered on days 1 and 15 and weeks 24 and 26 or twice-daily oral mycophenolate mofetil.
At week 52, the primary efficacy endpoint was assessed using a first-morning urine protein-creatinine ratio threshold of no more than 0.2 g/g, with patients also required to remain free of relapse between weeks 8 and 52. A statistically significantly greater proportion of patients receiving obinutuzumab reached the endpoint compared with standard-of-care treatment (73%).
The obinutuzumab label includes a boxed warning for hepatitis B virus reactivation and progressive multifocal leukoencephalopathy. Among patients with idiopathic nephrotic syndrome, the most common side effects are infections, infusion-related reactions, and neutropenia.
Obinutuzumab also has previous approvals for certain cancers and for active lupus nephritis in adults receiving standard therapy. For the idiopathic nephrotic syndrome indication, obinutuzumab had Breakthrough Therapy, Orphan Drug, and Priority Review designations.
Source: US Food and Drug Administration
