FDA has approved tocilizumab (Actemra), given alone or in combination with methotrexate, for the treatment of active systemic juvenile idiopathic arthritis in children 2 years of age and older.
FDA has approved tocilizumab (Actemra), given alone or in combination with methotrexate, for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in children 2 years of age and older.
SJIA, or Still's disease, is a rare, potentially life-threatening disorder in children that causes severe inflammation throughout the body. SJIA is distinguished from other forms of JIA by the prominence of systemic and inflammatory features, including spiking fevers; rash; swelling and inflammation of lymph nodes, liver, and spleen; and high white blood cell and platelet counts. It is estimated that JIA occurs in 1 to 2 children out of 1,000; of these JIA patients, SJIA affects approximately 10%.
“This is a novel drug that provides an FDA-approved option for a disease that’s extremely difficult to treat. Although it’s a rare disease, the impact is tremendous, because it not only may save lives, but also may significantly improve quality of life,” said Robert A. Quercia, MS, RPh, adjunct associate professor, University of Connecticut School of Pharmacy, Storrs, Conn., and Editorial Adviser for Drug Topics' sister publication Formulary.
Tocilizumab is an interleukin-6 receptor blocker approved by FDA on Jan. 8, 2010, for treatment of adults with moderately to severely 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," said Badrul Chowdhury, MD, PhD, director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA's Center for Drug Evaluation and Research.
An international, multicenter, controlled trial demonstrated the safety and effectiveness of tocilizumab, in which 112 patients received either tocilizumab infusions or placebo infusions every 2 weeks. Study participants included 2- to 17-year-old patients with SJIA who had inadequate response to or who were unable to take nonsteroidal anti-inflammatory drugs and corticosteroids.
Eighty-five percent of those receiving tocilizumab 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. In the long-term follow-up period of the trial there were 3 cases of macrophage activation syndrome among SJIA patients receiving tocilizumab.
Tocilizumab carries a Boxed Warning for serious infections. Patients treated with tocilizumab who develop a serious infection should stop tocilizumab 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. The most common side effects in trial participants with SJIA included upper respiratory tract infection, headache, sore throat, and diarrhea.