PBS listing marks first oral therapy for childhood arthritis

Pharma News: The first oral Janus kinase (JAK) inhibitor, Pfizer’s Xeljanz (tofacitinib), has been listed on the Pharmaceutical Benefits Scheme (PBS) effective December 1st for patients aged 2 years or older with active polyarticular course juvenile idiopathic arthritis (pcJIA) who have responded inadequately to traditional therapy with disease-modifying antirheumatic drugs (DMARDs).

Juvenile idiopathic arthritis affects approximately 6,000 children in Australia, making it one of the most prevalent chronic childhood diseases in the country. Until now, access to PBS-subsidised treatments was limited to biologic systemic therapies, including AbbVie’s Humira (adalimumab), Amgen’s Enbrel etanercept, and Roche’s Actemra (tocilizumab), along with their respective biosimilars.

Dr Jeff Chaitow, Head of Rheumatology at The Children’s Hospital at Westmead, expressed enthusiasm about the PBS listing of Pfizer’s Xeljanz, saying, “The availability of Xeljanz (tofacitinib) on the PBS offers subsidised access to the first and only Janus kinase (JAK) inhibitor approved in Australia for the treatment of pcJIA, in two formulations, a tablet and an oral solution – opening up new possibilities for clinicians to explore additional treatment options.”

He further noted, “Many patients living with juvenile idiopathic arthritis require injections or infusions as part of their treatment, which can present challenges for both the children and their caregivers. This listing represents a significant advancement and offers relief for needle-phobic patients and their families.”

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The approval of Xeljanz is founded on results from a Phase 3 study, showcasing its efficacy and safety in alleviating symptoms and reducing disease flare-ups. The study, involving 225 patients with 5 or more active joints, evaluated two forms of Xeljanz administration – a 5 mg tablet or a 1 mg/mL oral solution – taken twice daily. At the end of the second phase (week 44), patients on Xeljanz demonstrated significantly fewer disease flares compared to those on placebo (29.2% vs 52.9%, P = 0.0031). Additionally, 45% of patients achieved JIA ACR30* as early as week 2. The research also highlighted improved physical function among patients taking Xeljanz compared to the placebo group.

Associate Professor Ruth Colagiuri, founder of Juvenile Arthritis Foundation Australia (JAFA), emphasised the significance of this announcement for the community.

“Eighty percent (80%) of children with juvenile arthritis endure daily pain, leading to limited physical activities, increased risk of mental and physical health issues, social isolation, and disengagement from education,” she explained.

“Juvenile arthritis is one of the most under-recognised, misunderstood, and neglected childhood diseases in Australia. For children and their families affected, managing the condition can be complex, expensive, and take a psychological, social, and economic toll,” added Associate Professor Colagiuri, whose grandchild is living with arthritis.

Anne Harris, Pfizer Australia & New Zealand Managing Director, hailed the PBS listing as a significant progress toward delivering breakthrough medicines to patients and clinicians, showcasing Pfizer’s expertise in JAK science. She expressed gratitude to the Australian Government for acknowledging the importance of providing additional options to individuals living with polyarticular course juvenile idiopathic arthritis and their families.

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*An improvement of 30% from baseline in three of six variables (i.e. physician global assessment of disease activity, parent/guardian’s or patient’s global assessment of overall well-being, functional ability, number of joints with active arthritis, number of joints with limited range of motion and erythrocyte sedimentation rate) and no more than one of the remaining variables worsening by more than 30%.