FDA Approves Benlysta for Children With Active Lupus Nephritis

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The U.S. Food and Drug Administration (FDA) has extended the use of Benlysta (belimumab), given on top of standard therapy, to treat children ages 5 to 17 with active lupus nephritis, a serious form of kidney inflammation caused by lupus.

Benlysta is the first FDA-approved treatment for pediatric lupus nephritis, according to the therapy’s maker GSK, formerly known as GlaxoSmithKline.

“Active lupus nephritis is a potential serious complication in children with lupus, with most cases occurring within the first two years after their initial lupus diagnosis. This approval marks a significant step forward in providing treatment options to these children at risk of incurring kidney damage early on in life,” Stevan Gibson, president and CEO of the Lupus Foundation of America, said in a GSK press release.

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“Our community has much to celebrate with the approval of the first and much-needed treatment for children with lupus nephritis,” Kenneth Farber, president and CEO of the Lupus Research Alliance, said in a separate press release. Farber noted the alliance helped fund early research that led to Benlysta’s development.

Benlysta eased disease activity, severity in trial

Benlysta is an antibody-based therapy designed to bind and inhibit B-lymphocyte stimulator (BLyS), a protein that contributes to the survival of immune B-cells. These cells play a role in the autoimmune attack that drives lupus by making antibodies that target healthy tissue. By blocking BLyS, Benlysta is designed to lower B-cell levels, lessening such autoimmune attacks.

The therapy was approved by the FDA in 2011 to treat adults with active systemic lupus erythematosus (SLE) — the first new therapy approved for lupus in five decades. The FDA extended the approval to cover children, ages 5 and older, with SLE in 2019, and for adults with active lupus nephritis in 2020.

Approval for children with SLE was supported by data from a Phase 2 trial called PLUTO (NCT01649765) that enrolled 93 children, who were randomly assigned to standard treatment plus either Benlysta (10 mg/kg) or a placebo.

About 1 in 5 children had kidney involvement at the trial’s start. Results showed that assessments of disease activity and severity were generally more favorable in the Benlysta group.

Moreover, the proportion of children experiencing at least one severe disease flare was lower in the Benlysta group (17% vs. 35%), corresponding to a 64% lower risk of flare in Benlysta-treated patients compared with placebo.

The therapy was well-tolerated in children, displaying a pharmacological and safety profile similar to that seen in adults with SLE.

In children, Benlysta is administered via hourlong infusions into the bloodstream, given every other week for the first three doses and once a month thereafter. A formulation of the therapy that is administered via subcutaneous (under-the-skin) injections is available, but it is only approved for adults.

Common side effects associated with Benlysta include nausea, diarrhea, fever, inflammation in the airways, insomnia, migraine, pain in the extremities, and depression. Suicidal thoughts have been reported in patients on Benlysta. Because the therapy works by dampening the activity of the immune system, it may increase the risk of serious infections.

“The long-term goal of lupus nephritis management in adults and children is to preserve renal [kidney] function while minimizing treatment-related toxicities and associated morbidity,” said Herson Quinones, vice president of specialty and pipeline U.S. medical affairs at GSK.

“This Benlysta approval highlights GSK’s commitment to bring treatment options to children living with lupus nephritis. This is another example of how GSK continues to get ahead of this burdensome disease by focusing on science and being grounded in over a decade of clinical experience,” added Quinones.