London UK, Philadelphia USA April 26, 2019 — GSK today announced that 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.
Dr Hal Barron, Chief Scientific Officer and President, R&D, GSK commented, “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.”
Stevan W Gibson, President and CEO, Lupus Foundation of America commented, “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.”
Kenneth M Farber, President and CEO, Lupus Research Alliance, commented, “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.”
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 paediatric 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 52.1
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.
About the study
‘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.
The study results were first presented at the 2018 American College of Rheumatology (ACR). Abstract 2867.
About systemic lupus erythematosus (SLE)
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.2
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.3,4 It is It has been calculated that there are between 5,000 and 10,000 children with SLE in the United States.5
About Benlysta (belimumab)
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.
Limitations of Use: The efficacy of Benlysta has not been evaluated in patients with severe active lupus nephritis or severe active central nervous system lupus. Benlysta has not been studied in combination with other biologics or intravenous cyclophosphamide. Use of Benlysta is not recommended in these situations.
Benlysta is not licensed in the European Union or other countries for use in children.
GSK’s commitment to immunology
GSK is focused on the research and development of medicines for immune-mediated diseases, such as lupus and rheumatoid arthritis, that are responsible for a significant health burden to patients and society. Our world-leading scientists are focusing research on the biology of the immune system with the aim to develop immunological-based medicines that have the potential to alter the course of inflammatory disease. As the only company with a biological treatment approved for adult and paediatric lupus, GSK is leading the way to help patients and their families manage this chronic, inflammatory autoimmune disease. Our aim is to develop transformational medicines that can alter the course of inflammatory disease to help people live their best day, every day.
Cautionary statement regarding forward-looking statements
GSK cautions investors that any forward-looking statements or projections made by GSK, including those made in this announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Such factors include, but are not limited to, those described under Item 3.D 'Principal risks and uncertainties' in the company's Annual Report on Form 20-F for 2018.
Posted: April 2019
- GSK Receives FDA Approval for a New Self-Injectable Formulation of Benlysta (belimumab) for Systemic Lupus Erythematosus – July 21, 2017
- FDA Approves Benlysta to Treat Lupus – March 10, 2011
- GlaxoSmithKline and Human Genome Sciences announce FDA extension of Benlysta PDUFA target date to 10th March 2011 – December 6, 2010
- Human Genome Sciences and GlaxoSmithKline Announce Vote of FDA Advisory Committee to Recommend Approval of Benlysta for Systemic Lupus Erythematosus – November 17, 2010
- Human Genome Sciences and GlaxoSmithKline Announce FDA Priority Review Designation for Benlysta (belimumab) as a Potential Treatment for Systemic Lupus Erythematosus – August 19, 2010
- Human Genome Sciences and Lonza Enter Commercial Manufacturing Agreement for Benlysta, a Potential New Treatment for Systemic Lupus Erythematosus – July 13, 2010
- Human Genome Sciences Announces Submission of Biologics License Application to FDA for Benlysta (belimumab) – June 10, 2010
Benlysta (belimumab) FDA Approval History
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