The FDA has approved Ilaris for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in children aged between 2 and 19 years.
Ilaris is currently used to treat adults and children as young as four years suffering from cryopyrin-associated periodic syndrome, in over 60 countries. Cryopyrin-associated periodic syndrome is a rare lifelong auto-inflammatory genetic disorder that raises the production of the inflammatory cytokine interleukin-1 beta, in the body.
While only about 7,000 people worldwide have the cryopyrin-associated periodic syndrome, SJIA affects five to 15 children per 100,000 in the United States, and is the most severe subtype of juvenile idiopathic arthritis. Patients afflicted with the disease manifest spiking fever, rash and arthritis and debilitating symptoms, including joint destruction and growth retardation, with serious developmental and psychological consequences. The treatment options are limited: prolonged use of corticosteroids to treat symptoms and pain is fraught with potentially serious adverse effects, including Cushing syndrome, growth suppression and osteoporosis.
"In the U.S., this approval marks the second Ilaris indication for patients living with rare, auto-inflammatory conditions," says Timothy Wright, global head of development for Novartis Pharmaceuticals in a news report online.
"This approval was based on two Phase III trials in SJIA patients, aged 2-19, showing significant improvement in the majority of Ilaris-treated patients," according to the Novartis press release. Ilaris is a fully human, monoclonal antibody that blocks the protein called interleukin-1beta (IL-1 beta). IL-1 beta is an important part of the body's immune system defenses. Ilaris neutralizes IL-1 beta and thereby inhibits inflammation. The results from the first trial showed that by the end of Day 15, 84 percent of patients treated with one subcutaneous dose of Ilaris (4 mg/kg, up to 300 mg) achieved the desired goal of at least a 30 percent improvement in systemic and arthritic symptoms versus baseline, compared to 10 percent in those who were untreated. In the second trial, Ilaris treatment led to substantial reduction of corticosteroid usage among 62 percent of the 92 patients, and 46 percent completely discontinued corticosteroids. It also reduced the risk of flare for patients in a controlled category using the Ilaris therapy.
Indeed, the company is elated that the therapeutic goals of Ilaris were successfully achieved.
"We are committed to studying Ilaris in other IL-1 beta mediated inflammatory diseases, including several rare diseases for which treatment options do not currently exist," says Wright. Ilaris is currently being tested for a number of rare autoinflammatory conditions, including Tumor Necrosis Factor Receptor-Associated Periodic Syndrome (TRAPS), colchicine-resistant Familial Mediterranean Fever (FMF) and Hyper IgD Syndrome (HIDS). Given the results with SJIA, the manufacturer is confident that the drug will show benefit even for the other conditions.
