FDA approves at-home belimumab autoinjector for children with lupus nephritis

FDA approves at-home belimumab autoinjector for children with lupus nephritis |© Contemporary Pediatrics

The US Food and Drug Administration (FDA) has approved a 200 mg/mL autoinjector formulation of belimumab (Benlysta; GSK) for children aged 5 years and older with active lupus nephritis (LN), offering a new at-home administration option for patients receiving standard therapy. The approval, announced by GSK, provides pediatric patients and caregivers with the first subcutaneous, at-home biologic treatment for LN.¹

Belimumab, a B-lymphocyte stimulator (BLyS)-specific monoclonal antibody, was originally approved for adult systemic lupus erythematosus (SLE) in 2011. The indication was expanded to pediatric SLE in 2019, adult LN in 2020, and pediatric LN in 2022. The 200 mg/mL autoinjector formulation was approved for pediatric SLE earlier in 2024.¹

“For children and parents of children with lupus nephritis, this approval represents a choice in their care,” said Court Horncastle, senior vice president and head of US Specialty at GSK. “Providing this at-home treatment option with the efficacy and safety of Benlysta is a testament to our ongoing commitment to the lupus community.”

Lupus nephritis, a complication of systemic lupus erythematosus, occurs when the immune system attacks the kidneys, leading to inflammation and potentially irreversible damage. It affects approximately 30% to 50% of children with lupus, usually within 1 to 2 years of diagnosis.¹

“In children, lupus tends to be more aggressive and severe than it is in adults,” said Louise Vetter, president and CEO of the Lupus Foundation of America. “The symptoms can be more intense, and the disease can have long-term effects on a child's growth and quality of life. Having the Benlysta autoinjector provides a much-needed option that can help reduce the burden of frequent clinic visits for treatment and add greater flexibility for children and their families when considering continuity of care and routines of daily life.”

The FDA’s decision to approve the autoinjector for at-home use was based on its established safety profile and the growing need to reduce the treatment burden on pediatric patients and families. Children currently receiving intravenous belimumab may be eligible to transition to the autoinjector following provider consultation.²

Belimumab works by inhibiting BLyS, a protein critical for B-cell survival. By blocking BLyS, it reduces the activity of autoreactive B cells and antibody production, key drivers of lupus-related inflammation.

The new autoinjector formulation allows for subcutaneous injection at home, either administered by a health care provider or a trained caregiver. It is available immediately for eligible pediatric patients.

Lupus nephritis remains a leading cause of end-stage kidney disease in children with SLE. Clinical markers include proteinuria, elevated serum creatinine, and the presence of blood cells in the urine. Long-term outcomes remain poor for many patients, despite improvements in treatment.

Belimumab is not recommended for patients with severe active central nervous system lupus. Key safety concerns include serious infections, hypersensitivity reactions, progressive multifocal leukoencephalopathy (PML), depression and suicidality, and the risk of malignancy. Live vaccines should not be given within 30 days of treatment, and concurrent use with other biologic therapies, such as rituximab, is not advised due to increased risk of serious adverse events.¹

Adverse reactions reported in clinical trials with pediatric and adult patients include nausea, fever, depression, injection site reactions, and infections such as bronchitis and nasopharyngitis.¹

References:

1. GSK. FDA approves Benlysta (belimumab) Autoinjector for children with active lupus nephritis. Businesswire. June 24, 2025. Accessed June 24, 2025. https://businesswire.com/news/home/20250623981547/en/FDA-approves-Benlysta-belimumab-Autoinjector-for-children-with-active-lupus-nephritis
2. Stohl W, Kwok A. Belimumab for the treatment of pediatric patients with lupus nephritis. Published online February 7, 2023:1-9. doi:https://doi.org/10.1080/14712598.2023.2178297