Ilaris (canakinumab) is a drug approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of active systemic juvenile arthritis (sJA), a subset of juvenile arthritis (JA), in patients aged 2 and older.
The drug, developed by Novartis, is the first interleukin-1 beta (IL-1 beta) inhibitor approved for sJA. It’s also the only treatment approved for sJA that is given as a once-a-month injection under the skin.
How Ilaris works
Ilaris is a selective human monoclonal antibody designed to inhibit a protein known as interleukin-1 beta (IL-1β). IL-1β is an important part of the body’s immune system, but it causes inflammation if produced in excess. By blocking IL-1β, Ilaris helps reduce inflammation and relieve the symptoms of the disease.
Ilaris in clinical trials for sJA
Ilaris’ sJA approval was based on two Phase 3 randomized, controlled trials (NCT00886769 and NCT00889863) in patients aged 2–19 with active sJA. The first trial assessed the safety and effectiveness of Ilaris over four weeks. The second was a two-part study. The first stage dealt with Ilaris’ ability to allow patients to taper off of steroids. The second stage dealt with Ilaris’ longer-term effectiveness.
The results of the two trials showed that Ilaris reduced disease activity, allowed patients to taper off of glucocorticoid therapy, and delayed the time it took for patients’ disease to flare up.
Although the drug was well-tolerated and generally safe, two patients in the trial died. Both were in a placebo group, however. Researchers reported seven macrophage activation syndrome (MAS) events, a potentially fatal complication of rheumatic diseases.
A Phase 3 open-label extension trial (NCT00891046) looked at Ilaris’ long-term safety and effectiveness. It covered some of the patients from the previous trials who had responded well to treatment as well as sJA patients who had never been treated before. The first published results of the trial showed that Ilaris did not have a significant effect on MAS risk in patients with sJA.
Novartis has begun recruiting participants for a new two-part, open-label Phase 3 clinical trial (NCT02296424). The study will evaluate whether reducing patients’ doses of Ilaris would continue to generate a benefit. One group will consist of those from the extension study with an inactive disease for at least 24 continuous weeks. The other will be made up of patients whose diagnosis occurred at least two months prior to enrollment in the extension study.
Recruitment is continuing in a Phase 3 study (NCT02334748) designed to collect safety data — of both serious and non-serious adverse events — from patients who participated in previous clinical trials.
Ilaris was first approved to treat cryopyrin-associated periodic syndromes, or CAPS. Recently, the FDA also approved it for the treatment of tumor necrosis factor receptor-associated periodic syndrome (TRAPS); hyper-immunoglobulin D syndrome (HIDS), also known as mevalonate kinase deficiency (MKD); and familial Mediterranean fever (FMF).
The most common side effects of Ilaris as an sJA treatment are upper respiratory tract infection, pneumonia, runny nose, sore throat, urinary tract infection, nausea and vomiting, diarrhea, stomach pain, and injection site reactions such as redness, swelling, warmth, and itching.
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