FDA approves Novartis' Ilaris for treatment of active systemic juvenile idiopathic arthritis

Novartis announced that the US Food and Drug Administration (FDA) has approved Ilaris^® (canakinumab) for the treatment of active systemic juvenile idiopathic arthritis (SJIA) in patients aged 2 years and older. Ilaris is the first interleukin-1 beta (IL-1 beta) inhibitor approved for SJIA and the only treatment approved specifically for SJIA that is given as a once-monthly subcutaneous injection. SJIA is a rare and disabling form of childhood arthritis characterized by spiking fever, rash and arthritis that can affect children as young as 2 years old and can continue into adulthood.

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. Study 1 showed that 84% of patients treated with one subcutaneous dose of Ilaris achieved the primary endpoint of the adapted pediatric American College of Rheumatology 30 (ACR30), compared to 10% achievement of ACR30 for placebo at Day 15. In the open-label part of Study 2, 92 of 128 patients attempted corticosteroid tapering. Of those 92 patients, 62% were able to substantially reduce their use of corticosteroids, and 46% completely discontinued corticosteroids. In the controlled portion of Study 2, there was a 64% relative reduction in the risk of flare for patients in the Ilaris group as compared to those in the placebo group (hazard ratio of 0.36; 95% CI: 0.17 to 0.75).

"The efficacy of Ilaris, along with its monthly subcutaneous dosing, make it an exciting new option for children who are living with this debilitating disease," said Daniel Lovell , MD, MPH, study investigator and Joseph E. Levinson Professor of Pediatrics at the Cincinnati Children's Hospital Medical Center. "Additionally, the potential to reduce corticosteroid use is particularly beneficial in this patient population given the side effects associated with long-term use of corticosteroids in children."

SJIA affects 5-15 children per 100,000 in the United States, and is the most severe subtype of juvenile idiopathic arthritis. Although the disease can be life-threatening, treatment options are limited. Corticosteroids are often used to treat symptoms and pain despite their association with potentially serious adverse effects, including Cushing syndrome, growth suppression and osteoporosis.

"In the US, this approval marks the second Ilaris indication for patients living with rare, autoinflammatory conditions," said Timothy Wright , MD, Global Head of Development, Novartis Pharmaceuticals. "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."