FDA Approves Belimumab for Pediatric SLE

The Food and Drug Administration (FDA) has approved belimumab, sold under the brand name Benlysta from manufacturer GlaxoSmithKline (GSK), an intravenous (IV) infusion treatment for pediatric systemic lupus erythematosus (SLE) patients. It is the first approved treatment for SLE in young patients.

“The agency expedited the review and approval of this application because Benlysta IV fulfils an unmet need for therapies, specifically in pediatric patients with SLE,” said Janet Woodcock, MD, director of the FDA’s Center for Drug Evaluation and Research, in a press release. “While there is no cure for lupus, treatment can help our youngest patients control their disease with the hope of improving their quality of life and lowering their risk of long-term organ damage and disability.”

Belimumab has been indicated for adult patients with SLE since 2011, the FDA noted. Benlysta is manufactured by GlaxoSmithKline.

According to the Centers for Disease Control and Prevention, people of all ages are at risk for SLE, although it is more common among women aged between 15 and 44 years. While specific statistics vary, women are believed to be at significantly greater risk than men. Still, the Arthritis Foundation estimates that anywhere between 5,000 and 10,000 children in the United States live with SLE.

PLUTO Trial Findings

Belimumab’s efficacy was measured through the randomized controlled PLUTO trial. The study included 93 SLE patients aged between five and 17 years with active disease, as defined by a safety of estrogen in lupus national assessment (SELENA) SLE disease activity index (SLEDAI) score ≥ 6. All patients were required to be on a stable treatment regimen for ≥ 30 days prior to the start of the study. Exclusion criteria included any previous treatment with Benlysta; B-cell targeted therapy treatment in the past year; having received anti-tumor necrosis factor, interleukin-1 receptor antagonist, IVIG, or plasmapheresis within 90 days of the start of the trial; or having received high dose prednisone or equivalent (> 1.5mg/kg/day) or any new immunosuppressive/immunomodulatory agent, anti-malarial agent within 60 days of the start of the trial.

The study compared outcomes for belimumab versus placebo. Belimumab patients received 10 mg/kg IV monthly, and the placebo group received saline.

“The proportion of pediatric patients achieving the composite primary endpoint, the SLE response index (SRI-4), was higher in pediatric patients receiving Benlysta IV plus standard therapy compared to placebo plus standard therapy,” according to the FDA. “Pediatric patients who received Benlysta IV plus standard therapy also had a lower risk of experiencing a severe flare, as well as longer duration of time until a severe flare (160 days versus 82 days).” Safety and pharmacokinetic profiles were not significantly different from those in adult SLE patients.

The most common side effects included nausea, diarrhea, and fever.

The FDA granted belimumab Priority Review, a designation that means the FDA works to take action on an application within six months, as opposed to 10 months for Standard Review applications.