RoActemra® (tocilizumab) has been launched as a specific treatment option for pJIA, the form of juvenile arthritis with the worst prognosis.1 This decision heralds a less frequent dosing regimen than currently available treatments for many of the estimated one thousand plus children1 who have failed with methotrexate (MTX) and whose childhood could be affected by pJIA, which can make simple, everyday activities such as walking and playing with friends both painful and challenging.
The treatment, which can be given alone or in combination with MTX every four weeks in children with pJIA,1 showed a significant reduction of flares in a clinical study – when symptoms of the disease temporarily intensify – compared to placebo.2 It is the second approval in a juvenile idiopathic arthritis setting for the treatment, which was approved in 2011 for systemic juvenile idiopathic arthritis (sJIA).3 With a license for sJIA and pJIA, this suggests that the treatment could help alleviate the suffering of over one third of the children in Britain currently living with JIA.1,4
Dr. A. V. Ramanan, Lead Consultant in Paediatric Rheumatology at Bristol Royal Hospital for Children & Royal National Hospital for Rheumatic Diseases noted: “I am excited that RoActemra is now available in pJIA, as clinicians can treat children affected by this debilitating disease with this new treatment option. The fact that it will now be readily available for patients across the UK gives hope to hundreds of children that doing the things that other kids do in their day-to- day lives may soon be a reality. RoActemra provides an extra option for children with difficult to control disease.”
The positive decision was based on data from the phase III CHERISH trial, which demonstrated that children treated with the drug experienced clinically meaningful improvements in the signs and symptoms of pJIA.2 After 40 weeks of treatment, nearly two thirds (65%) of children saw an impressive 70% improvement (JIA ACR70 response) in their condition, compared to placebo.2 Ailsa Bosworth, CEO, National Rheumatoid Arthritis Society (NRAS) comments: “We welcome the positive news for children with pJIA and their families that RoActemra has been licenced for pJIA, offering an additional effective and well tolerated treatment option. JIA is a complex disease to treat and it is therefore important that clinicians and patients have as many treatment options as possible at their disposal to help them optimise clinical outcomes.”
Approximately 50% of children with JIA do not achieve remission despite treatment, often requiring further and expensive rheumatological care as adults.1 Children with pJIA have the worst prognosis, with a remission rate of only 15% over 10 years.1 Approximately 30% – 40% of children with pJIA require early joint replacement.1 pJIA can come on suddenly and symptoms can include on-going joint pain and a slight fever.5
“The licencing of RoActemra for pJIA is fantastic news for the children affected by this incurable condition, as it offers hope for the future and the potential to enjoy their childhood,” said Judi Rhys, CEO, Arthritis Care. “We know, through our work on the Arthritis Care Young People’s Project in supporting families with children with arthritis, that a diagnosis of pJIA can be devastating for a child and their family; it impacts every aspect of a child’s life from schooling, to socialising and everything in between.”
The treatment is indicated for the treatment of active pJIA in patients two years of age and older who have responded inadequately to previous therapy with MTX1 and can be given as monotherapy (in cases of intolerance to MTX or where continued treatment with MTX is inappropriate) or in combination with MTX. It is currently licenced for the treatment of active sJIA in children aged 2-16yrs and in adult rheumatoid arthritis, and is the only licensed biologic treatment to target the interleukin-6 (IL-6) pathway,3 which plays a pivotal role in pJIA pathogenesis.1
Within the CHERISH study, the adverse event profile was comparable with placebo.2 RoActemra has an established safety profile as demonstrated in over 137,167 RA patients having received the medicine.6 The most common adverse reactions reported in clinical studies were upper respiratory tract infection, nasopharyngitis, headache, elevated blood pressure, and increased liver enzymes. The serious adverse reactions reported in clinical studies include serious infections, gastrointestinal perforations and hypersensitivity reactions including anaphylaxis.3
About pJIA
It is estimated that JIA develops in over one thousand children in the UK every year;1 about 10,000 children are affected1 and its cause isn’t fully understood at present but it’s thought to be an autoimmune disease
Polyarticular JIA (pJIA) is the second most common form of JIA,7 representing approximately 23% of all juvenile idiopathic arthritic conditions in the UK1
About 50% of children with JIA do not achieve remission despite treatment and require further rheumatological care as adults1
Children with pJIA have the worst prognosis, with a remission rate of only 15% over 10 years1
Approximately 30% – 40% of children with pJIA require early joint replacement1
Polyarticular JIA affects children between 2 and 17 years of age1
A copy of the Summary of Product Characteristics is available at http://emc.medicines.org.uk
CHERISH trial2
The CHERISH study was a 2-year, 3-part, randomized, double-blind, placebo-controlled withdrawal study of MTX treatment failures in active JIA with polyarticular course:
- Results from Parts 1 and 2 (i.e., until week 40)
- Safety results until November 2011
The CHERISH study met its primary endpoint with a significant reduction of flares compared to placebo. Other conclusions reached included:
- RoActemra is highly effective and results in clinically meaningful improvement of pJIA
- Suggested RoActemra doses are:
- 8 mg/kg every four weeks in patients with pJIA and a body weight ≥30 kg
- 10 mg/kg every four weeks in patients with pJIA and a body weight <30 kg
- The safety profile of RoActemra in patients with pJIA is consistent with that seen in other RoActemra-treated diseases
- Longer-term safety data are forthcoming
About RoActemra
Launched in the UK in 2009 as the first interleukin-6 (IL-6) receptor antagonist, it is licensed for use in combination with MTX for the treatment of moderate to severe active rheumatoid arthritis (RA) in adult patients who have either responded inadequately to, or who were intolerant to, previous therapy with one or more disease-modifying anti-rheumatic drugs (DMARDs) or tumour necrosis factor (TNF) antagonists.3 The treatment has been shown to reduce the rate of progression of joint damage as measured by X-ray and to improve physical function.3
The treatment can be given as a monotherapy to adult patients who cannot tolerate MTX or where continued treatment with MTX is inappropriate, and helps almost four times as many patients achieve remission than those treated with a leading anti-TNF monotherapy.3,8
RoActemra is also indicated for the treatment of active systemic juvenile idiopathic arthritis (sJIA) in patients two years of age and older, who have responded inadequately to previous therapy with NSAIDs and systemic corticosteroids.3 It is also licensed for the treatment of polyarticular juvenile arthritis (pJIA) in children between 2 and 17 years of age.1