The US Food and Drug Administration (FDA) has approved belimumab (Benlysta, GlaxoSmithKline) intravenous (IV) infusion for use in children as young as age 5 years with systemic lupus erythematosus (SLE).
Belimumab is a B-lymphocyte stimulator protein inhibitor that is thought to decrease the amount of abnormal B cells. It is hypothesized that an abnormal level of B cells is a mechanism of action in lupus.
Belimumab was first approved in the US in March 2011 for adults. Today's approval extends its use to children aged 5 years and older with active, autoantibody-positive, SLE who are receiving standard therapy.
Belimumab is currently the only medicine specifically approved in the US for both adults and children with SLE. It's also the first time the FDA has approved a treatment for pediatric patients with SLE.
"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 a news 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," said Woodcock.
"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," Hal Barron, MD, chief scientific officer and president, R&D, GlaxoSmithKline, said in a company news release.
"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. 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," added Kenneth M. Farber, president and CEO, Lupus Research Alliance, in the company release.
Approval of belimumab for children was based on data from the PLUTO study, which were first presented at the 2018 American College of Rheumatology (ACR) last October.
The study evaluated the efficacy, safety, and pharmacokinetics of 10 mg/kg IV belimumab plus standard therapy compared with placebo plus standard therapy for 1-year in 93 children ages 5 to 17 years old with active SLE.
The proportion of children achieving the composite primary endpoint, the SLE response index (SRI-4), was higher in those receiving belimumab plus standard therapy compared with those receiving placebo plus standard therapy.
Pediatric patients who received belimumab 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 vs 82 days).
The drug's safety and pharmacokinetic profiles in pediatric patients were consistent with those in adults with SLE.
Belimumab includes a warning for mortality, serious infections, hypersensitivity, and depression, based on data from the clinical studies in adults with SLE. The drug should not be administered with live vaccines. The manufacturer is required to provide a medication guide to inform patients of the risks associated with belimumab.
The most common side effects in patients included nausea, diarrhea, and fever. Patients also commonly experienced infusion reactions, so healthcare professionals are advised to pretreat patients with an antihistamine.
Stevan W. Gibson, president and CEO of the Lupus Foundation of America, also welcomed the approval. "Lupus is a potential life-threatening disease that can be more aggressive and severe in children than it is in adults. For the first time, children with lupus will now have a lupus-specific treatment option for their disease. As a research community, we all share in the excitement of this historic milestone as it underscores our dedication to bringing new treatments to people living with lupus," he said in the GlaxoSmithKline release.
Cite this: FDA OKs First Treatment for Pediatric Lupus - Medscape - Apr 26, 2019.