The European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended extending the indication for intravenous (IV) belimumab (Benlysta, GlaxoSmithKline [Ireland] Limited) to include add-on therapy in children aged 5 years or older with active, autoantibody-positive systemic lupus erythematosus (SLE) with a high degree of disease activity despite standard therapy. If the European Commission approves belimumab, this will be the first time children aged 5 years or older will "have a medicine specifically developed and approved to treat this challenging autoimmune disease," according to a company news release. The US Food and Drug Administration (FDA) approved IV belimumab for children aged 5 years or older with lupus in April. The committee made its decision on the basis of safety, efficacy, and pharmacokinetic data from a postapproval commitment study (PLUTO) that compared belimumab 10 mg/kg IV plus standard therapy to placebo plus standard therapy for 1 year in 93 children (aged 5–11 years, n = 13; aged 12–17 years, n = 80) with active SLE. The researchers defined active SLE as a "Safety of Estrogens in lupus Erythematosus National Assessment-systemic lupus Erythematosus Disease Activity Index (SELENA-SLEDAI) score ≥ 6 and positive autoantibodies at screening." Pediatric lupus is uncommon; therefore, it was not "feasible" to conduct a fully-powered study. The proportion of patients who achieved clinically meaningful improvement in disease activity on the basis of the SLE responder index response rate was numerically higher in those who received belimumab plus standard therapy compared with those in the placebo plus standard therapy group (52.8% vs 43.6%) at week 52. The proportion of patients who experienced more than one adverse event was 79.2% in the belimumab group compared with 82.5% in the placebo group. The proportion of those reporting a serious adverse event was 17% in the belimumab group compared with 35% in the placebo group. There were no new safety signals in the children aged 12 years or older (n = 80). Safety data were limited in those younger than 12 years (n = 13). A positive opinion from CHMP is one of the last steps before the European Commission makes a decision regarding marketing authorization. The summary of product characteristics will include detailed recommendations for use of the product and will be published in the European public assessment report, which will be made available in all official European Union languages after the European Commission grants marketing authorization. Source
