April 15, 2011 — The US Food and Drug Administration (FDA) has approved tocilizumab (Actemra, Genentech Inc), given alone or in combination with methotrexate, for treatment of children age 2 years and older with active systemic juvenile idiopathic arthritis (SJIA).
Actemra is an interleukin-6 receptor blocker approved by the FDA in January 2010 for treatment of adults with moderate to severe active rheumatoid arthritis who have had an inadequate response to other approved therapies.
"This new indication of Actemra provides the first approved therapy for children with this rare disease," Badrul Chowdhury, MD, PhD, director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA's Center for Drug Evaluation and Research, noted in an FDA statement.
SJIA, or Still's disease, is a potentially life-threatening disorder in children that is distinguished from other forms of juvenile idiopathic arthritis (JIA) by the prominence of systemic and inflammatory features, the FDA noted in a statement. These include spiking fevers, rash, swelling, and inflammation of lymph nodes, liver, and spleen, as well as high white blood cell and platelet counts. JIA occurs in an estimated 1 to 2 children per 1000, and SJIA affects about 10% of all patients with JIA.
Pivotal Trial in Children
An international, multicenter controlled trial demonstrated the safety and effectiveness of Actemra. In the study, 112 patients with SJIA, age 2 to 17 years, received Actemra infusions or placebo infusions every 2 weeks. All of them had had an inadequate response to, or who were unable to take, nonsteroidal anti-inflammatory drugs and corticosteroids.
Eighty-five percent of those receiving Actemra responded to treatment, compared with 24% of patients receiving placebo. Response was defined as at least 30% improvement in the American College of Rheumatology's JIA efficacy variables, along with absence of fever in the preceding 7 days.
Three cases of macrophage activation syndrome among patients with SJIA receiving Actemra occurred. This potentially fatal complication of childhood systemic inflammatory disorders is thought to be caused by excessive activation and proliferation of certain immune cells, the FDA notes.
Actemra carries a "Black Box" warning for serious infections. It states that patients treated with Actemra who develop a serious infection should stop Actemra treatment until the infection is controlled.
The FDA also notes that changes in liver enzymes, blood counts, and cholesterol are not uncommon with Actemra and should be monitored with regular blood tests.
The most common side effects in trial participants with SJIA included upper respiratory tract infection, headache, sore throat, and diarrhea.
Actemra is marketed by San Francisco-based Genentech Inc, a subsidiary of the Roche Group.
Medscape Medical News © 2011 WebMD, LLC
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Cite this: Tocilizumab Approved for Active Systemic JIA - Medscape - Apr 15, 2011.