GSK announced the US Food and Drug Administration (FDA) has approved, under priority review, the use of the intravenous (IV) formulation of Benlysta (belimumab), a B-lymphocyte stimulator (BLyS)-specific inhibitor, in children with lupus from as young as five years of age.
"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." Dr. Hal Barron, Chief Scientific Officer and President, R&D, GSK said.
"Lupus is a potential life-threatening disease that can be more aggressive and severe in children than it is in adults,” Stevan W Gibson, President and CEO, Lupus Foundation of America said. “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."
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The approval extends the current indication in the US for the IV formulation of Benlysta in adults, to patients aged 5 years and older with active, autoantibody-positive, systemic lupus erythematosus (SLE) who are receiving standard therapy. Benlysta was approved in the US in March 2011 for adults, and is currently the only medicine specifically approved in the US for both adults and children with SLE. The IV formulation of Benlysta is currently not approved for use in children anywhere else in the world although regulatory submissions are ongoing in other parts of the world.
The supplemental Biologics License Application (sBLA), which received FDA priority review, to support today's approval was based on data from a post-approval commitment study (the 'PLUTO' study), assessing the efficacy, safety and pharmacokinetics of 10 mg/kg intravenous belimumab plus standard therapy compared with placebo plus standard therapy for one-year in children aged 5 – 11 years (n=13,) and 12 – 17 years (n=80) with SLE. As pediatric lupus is an uncommon disease, a fully powered study was not feasible.
The proportion of children achieving a clinically meaningful improvement in disease activity, as assessed by the SLE responder index (SRI) response rate, was numerically higher in patients receiving belimumab plus standard therapy (52.8%) compared with placebo plus standard therapy (43.6%) at Week 52.
The proportion of patients experiencing more than one adverse event (AE) and a serious AE was 79.2% and 17.0% for the belimumab group compared to 82.5% and 35.0% for the placebo group, respectively. AEs that led to discontinuation were lupus nephritis, hepatitis A, hypertransaminasemia, acute pancreatitis, post herpetic neuralgia, retinal vasculitis and pancreatitis. Refer to 'Important Safety Information for belimumab' below for further information on the safety of belimumab.
'PLUTO' (Pediatric Lupus Trial of Belimumab Plus Background Standard Therapy) is a multicentre, randomised, double-blind study (BEL114055) in 93 children with active SLE. The study comprises three phases: Part A (randomised double-blind 52-week treatment phase), Part B (long-term open-label safety phase) and Part C (long-term safety follow-up phase). The long-term follow-up phases of the study (Parts B and C) are planned for a total of up to 10 years.
SLE is a chronic, incurable, autoimmune disease associated with a range of symptoms that can fluctuate over time including painful or swollen joints, extreme fatigue, unexplained fever, skin rashes and organ damage. SLE is the most common form of lupus, affecting approximately 70 percent of an estimated 5 million people with lupus worldwide.
Compared with adult SLE, children with SLE often have more active disease both at the time of diagnosis and over time. SLE in children is associated with more rapid accrual of damage, and has a higher degree of morbidity compared with SLE in adult populations.
Benlysta, a BLyS-specific inhibitor, is a human monoclonal antibody that binds to soluble BLyS. Benlysta does not bind B cells directly. By binding BLyS, Benlysta inhibits the survival of B cells, including autoreactive B cells, and reduces the differentiation of B cells into immunoglobulin-producing plasma cells.
Benlysta is currently the only medicine specifically developed and approved for SLE. It is registered as both an IV and SC formulation in the US. In the US, the IV formulation of Benlysta is approved for use in adults and children, and the subcutaneous formulation of Benlysta is only approved for use in adults. The indication of the IV formulation of Benlysta is for the treatment of patients aged 5 years and older with active, autoantibody-positive, systemic lupus erythematosus who are receiving standard therapy.