The expanded indication allows the use of intravenous belimumab (Benlysta) in children as young as 5 years of age.
The US Food and Drug Administration (FDA) has expanded the indication for intravenous belimumab (Benlysta) to include the treatment of lupus for children as young as 5 years of age.
The indication is specifically for patients at least 5 years old with active, autoantibody-positive systemic lupus erythematosus who are receiving standard therapy. Today’s approval was conducted under Priority Review.
“Children with lupus have had limited options available to help treat their condition. This accelerated decision means children in the US now have an innovative, FDA-approved medicine available to help manage the impact of living with this challenging autoimmune disease,” said Hal Barron, MD, Chief Scientific Officer and President, R&D, GSK.
Belimumab was previously approved in the US for the treatment of lupus in adults in March 2011. It’s is the only medicine currently approved in the US for both children and adults with lupus.
The FDA’s decision to approve the supplement Biologics License Application for belimumab was supported by results from the PLUTO (Pediatric Lupus Trial of Belimumab Plus Background Standard Therapy) study, which evaluated the efficacy, safety, and pharmacokinetics of intravenous belimumab in children.
The randomized, double-blind PLUTO study included 13 participants aged 5-11 years, and 80 participants aged 12-17 years. The study compared 10 mg/kg intravenous belimumab compared to placebo, each in addition to standard therapy, over the course of 1 year.
At week 52, the number of children who reached a clinically meaningful improvement (assessed by the systemic lupus erythematosus responder index response rate) was 52.8% for those in the belimumab group compared to 43.6% for those in the placebo group.
A serious adverse event occurred in 17.0% of patients receiving belimumab versus 35.0% for placebo, and more than one adverse event occurred in 79.2% versus 82.5%, respectively. The adverse events that led to participant discontinuation were lupus nephritis, hepatitis A, hypertransaminasemia, acute pancreatitis, post herpetic neuralgia, retinal vasculitis, and pancreatitis.
“The go-ahead from the FDA for belimumab to be used to treat children with lupus is terrific news for a community desperate for more treatment options,” said Kenneth M Farber, President and CEO, Lupus Research Alliance. “As the only biologic approved for the disease, belimumab has been helping many adults with lupus, and now physicians will have another, much-needed tool for treating their pediatric patients.”