Tocilizumab (Actemra) is approved for the treatment of young people with active systemic juvenile idiopathic arthritis, a rare and severe form of inflammatory arthritis, according to a statement from the Food and Drug Administration.
The interleukin-6 receptor blocker was approved in January of 2010 for adults with moderate to severe rheumatoid arthritis that does not respond to other therapies.
The new juvenile indication, announced April 15, is for children, aged 2 years and older, with active systemic juvenile idiopathic arthritis (SJIA), or Still’s disease, a potentially life-threatening disorder that causes swelling and inflammation of the lymph nodes, liver, and spleen, among other effects.
Juvenile idiopathic arthritis is estimated to affect 1-2 children per 1,000, and SJIA, the most severe form of JIA, is seen in about 10% of those cases, according to the FDA. Tocilizumab is the first medication specifically approved by the agency for this patient group. It can be used as monotherapy or in combination with methotrexate.
The agency based the approval on results from a randomized, placebo-controlled trial involving 112 patients, aged 2-17 years, with active SJIA for at least 6 months. The participants either could not tolerate or did not respond well to their current therapies (nonsteroidal disease-modifying drugs and/or systemic corticosteroids). They were randomly assigned to receive tocilizumab intravenous infusions (8 mg/kg or 12 mg/kg depending on weight) or placebo every 2 weeks.
After 12 weeks, 85% of children in the treatment group responded to treatment compared with 24% of children in the control group. Treatment response was defined as at least a 30% improvement according to the American College of Rheumatology’s JIA efficacy variables, and the absence of fever in the preceding 7 days.
The medication carries an FDA boxed warning for serious infections. Patients treated with tocilizumab who develop serious infections should discontinue treatment until the infection is controlled.
Changes in certain laboratory test results such as liver tests, blood counts, and cholesterol are not uncommon with tocilizumab and should be monitored with regular blood tests, according to an FDA statement.
Three cases of macrophage activation syndrome occurred among the patients receiving tocilizumab.
The most common side effects included upper respiratory tract infection, headache, sore throat, and diarrhea.