Roche has announced that the European Medicines Agency ( EMA ) has approved RoActemra ( Tocilizumab ) to treat children with polyarticular juvenile idiopathic arthritis ( PJIA ), a rare, chronic and debilitating form of childhood arthritis.
The medicine can be used to treat patients two years of age and older who have not responded adequately to treatment with Methotrexate ( MTX ), a disease modifying anti-rheumatic drug.
RoActemra can be used alone or in combination with Methotrexate.
PJIA, or juvenile idiopathic polyarthritis, is a form of juvenile idiopathic arthritis ( JIA ), also known as juvenile rheumatoid arthritis. Juvenile idiopathic arthritis affects approximately 100 in every 100,000 children, with PJIA accounting for around 30% of cases.
PJIA is characterised by inflammation in five or more joints within the first six months of the disease and most commonly affects the small joints in the body, such as those in the hands and feet.
This is the second pediatric indication for RoActemra and follows its 2011 approval for systemic juvenile idiopathic arthritis ( sJIA ), another rare form of juvenile idiopathic arthritis.
The approval is based on phase III CHERISH study data that showed patients treated with RoActemra experienced clinically meaningful improvement in signs and symptoms of polyarticular juvenile idiopathic arthritis.
In addition, safety data collected to date for RoActemra in PJIA patients is consistent with that observed in previous studies in Tocilizumab-treated patients.
CHERISH is a 104-week, phase III study in patients aged 2-17 years with active polyarticular juvenile idiopathic arthritis for greater than or equal to 6 months who have failed treatment with Methotrexate.
Treatment with RoActemra was efficacious, with sustained clinically meaningful improvement in signs and symptoms of PJIA, using a monthly regimen at doses of 8 mg/kg if body weight greater than or equal to 30 kg and 10 mg/kg if body weight less than 30 kg.
The study met the primary endpoint with Tocilizumab-treated patients experiencing significantly fewer disease flares compared to placebo-treated patients ( 25.6% versus 48.1%, respectively ).
In the CHERISH study, infections were the most common adverse events ( AEs ) and serious adverse events ( SAEs ) over 40-weeks. Laboratory abnormalities known to occur with RoActemra were also observed in this study, including decreases in white blood cell counts and platelet counts, and elevation in ALT and AST liver enzyme levels.
RoACTEMRA, known as ACTEMRA outside of Europe, is the first humanised interleukin-6 ( IL-6 ) receptor antagonist approved for the treatment of adult patients with moderately to severely active rheumatoid arthritis ( RA ) who have had an inadequate response to one or more disease-modifying anti-rheumatic drugs ( DMARDs ).
The extensive RoACTEMRA clinical development programme included five phase III clinical studies and enrolled more than 4,000 people with rheumatoid arthritis in 41 countries.
In addition, the phase IV ADACTA study showed that monotherapy with Tocilizumab was superior to monotherapy with Adalimumab in reducing signs and symptoms of rheumatoid arthritis in MTX-intolerant patients or patients for whom MTX treatment was considered ineffective or inappropriate. ( Xagena )
Source: Roche, 2013