Roche Holding AG's rheumatoid arthritis drug Actemra received approval from the U.S. Food and Drug Administration (FDA) to treat a rare type of arthritis in children.
The U.S. health regulator cleared Actemra (tocilizumab), given alone or in combination with methotrexate, for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in children ages 2 years 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 juvenile idiopathic arthritis (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. The prevalence of JIA is an estimated 1 to 2 per 1,000 children, and SJIA affects about 10 percent of all JIA patients.
This new indication of Actemra provides the first approved therapy for children with this rare disease, said Badrul Chowdhury, director of the Division of Pulmonary, Allergy, and Rheumatology Products in the FDA’s Center for Drug Evaluation and Research.
Actemra was approved last year to treat adults with moderately to severely active rheumatoid arthritis who have had an inadequate response to other approved therapies.
According to the FDA, an international, multicenter controlled trial demonstrated the safety and effectiveness of Actemra. Study participants included patients with SJIA aged 2 to 17 years old who had 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 percent of patients receiving placebo, the FDA said.