A monoclonal antibody targeting interleukin-1 beta, has been approved for the treatment of systemic juvenile idiopathic arthritis (JIA), the manufacturer announced.
Following the demonstration of efficacy in two pivotal trials, the Food and Drug Administration granted an indication for canakinumab (Ilaris) as a once-monthly subcutaneous injection for patients 2 years and older with systemic JIA.
"In the U.S., this approval marks the second Ilaris indication for patients living with rare, autoinflammatory conditions," said Timothy Wright, MD, of Novartis Pharmaceuticals, in a press release.
Canakinumab was previously approved for the treatment of cryoprin-associated periodic syndrome.
Systemic JIA is characterized by arthritis, rash, and recurrent spiking fevers. Conventional treatment has relied on corticosteroids, but long-term use can lead to growth suppression, osteoporosis, and Cushing syndrome in these children.
The observation that interleukin-1 beta plays a central role in the pathogenesis of systemic JIA prompted efforts to target this cytokine therapeutically.
The first study was a 4-week, double-blind trial in which 84 patients ages 2 to 19 were randomized to a single injection of canakinumab (4 mg/kg) or placebo. The primary outcome measure was a 30% decrease in American College of Rheumatology (ACR) symptoms and resolution of fever by week 2.
A total of 84% of patients receiving the active treatment achieved this ACR30 response, compared with only 10% of patients given placebo (P<0.001).
In a second trial, 177 patients received 4 mg/kg subcutaneously once monthly, and 92 attempted corticosteroid discontinuation.
Among those who attempted to withdraw from steroid treatment, 62% were able to reduce their use of the drugs and 46% were able to discontinue altogether.
The second study also included a randomized phase following the open-label treatment, in which patients continued on canakinumab or were given placebo until flare occurred.
Risk of flare in the active treatment group was reduced by 64% (HR 0.36, 95% CI 0.17 to 0.75), the company reported.