FDA approves Benlysta for children, teens with lupus

Joan M. Von Feldt

The FDA has approved intravenous belimumab for the treatment of children aged 5 years and older with systemic lupus erythematosus, according to an FDA press release.

This decision extends the previous indication for intravenous belimumab (Benlysta, GlaxoSmithKline), first approved in March 2011, which had originally been limited to adult patients with lupus.

“As an adult rheumatologist who cared for so many young adults with severe damage from their disease and the corticosteroid treatment of their lupus during their teenage years, approval of Benlysta for pediatric lupus is wonderful news for kids with lupus, and their families,” Joan M. Von Feldt, MD, MSEd, an immunology medical expert for GlaxoSmithKline, told Healio Rheumatology. “With approval of Benlysta for pediatric lupus, pediatric rheumatologists now have a treatment option for lupus that can help effectively treat lupus and mitigate the severe consequences of lupus that carry into adulthood.”

The FDA approval allows the drug to be administered to patients aged 5 years and older with active, auto-antibody-positive SLE who are also receiving standard therapy. According to GlaxoSmithKline, the approval from the FDA makes belimumab the only drug currently approved in the United States for both adults and children with SLE. Although intravenous belimumab is not currently approved for use in children outside of the U.S., GlaxoSmithKline noted that it has begun the regulatory process to gain approval elsewhere.

“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,” Janet Woodcock, MD, director of the FDA’s Center for Drug Evaluation and Research, said in the 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.”

The FDA based its approval on results of the PLUTO study, a multicenter, randomized, double-blind trial comparing the efficacy, safety and pharmacokinetics of intravenous belimumab plus standard therapy against a placebo among 93 pediatric patients with active SLE. The researchers assessed disease activity, as measured by the SLE responder index, through week 52.

According to study results presented at the 2018 American College of Rheumatology annual meeting, 52.8% of patients treated with belimumab plus standard therapy achieved a clinically meaningful improvement in disease activity, compared with 43.6% among those who received placebo plus standard therapy. In addition, 17% of patients in the belimumab group experienced a serious adverse event, compared with 35% in the placebo group. The proportions of patients who experienced any adverse event was 79.2% in the belimumab group and 82.5% in the placebo group.

According to the FDA, the most common adverse events that led to treatment discontinuation included acute pancreatitis, hepatitis A, hypertransaminasemia, lupus nephritis, post-herpetic neuralgia and retinal vasculitis.

“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,” Kenneth M. Farber, president and chief executive officer of the Lupus Research Alliance, said in a press release. “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.”