FDA expands treatment in nephrotic syndrome - Summary - MDSpire
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FDA expands treatment in nephrotic syndrome

The indication marks an additional use for a therapy previously approved for certain cancers and active lupus nephritis.

  • By

  • Kathryn Wighton

  • September 28, 2026

  • 2 min

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Objective:

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.

Approach:
  • Trial Design: 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.
  • Treatment Comparison: Patients were randomly assigned to receive intravenous obinutuzumab or twice-daily oral mycophenolate mofetil.
  • Efficacy Assessment: 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.
Key Findings:
  • 73% of patients receiving obinutuzumab reached the primary efficacy endpoint compared to a statistically significantly lower percentage of those receiving standard-of-care treatment.
  • Obinutuzumab is associated with risks of hepatitis B virus reactivation and progressive multifocal leukoencephalopathy.
  • Common side effects include infections, infusion-related reactions, and neutropenia.
Interpretation:

Obinutuzumab has received Breakthrough Therapy, Orphan Drug, and Priority Review designations for this indication.

Limitations:
  • Enrollment was limited to patients in complete remission, requiring normal urine protein levels and no swelling.
Conclusion:

Obinutuzumab is now approved for reducing relapse risk in specific pediatric and adult patients with idiopathic nephrotic syndrome.

Sources:

Original Source(s)

FDA expands treatment in nephrotic syndrome

  • by Kathryn Wighton

  • September 28, 2026

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