First Treatment for Adult-Onset Still Disease Gets FDA Approval

syringe, vials
syringe, vials
The FDA has approved Ilaris for the treatment of active Still disease, including adult-onset Still disease.

The Food and Drug Administration (FDA) has approved Ilaris (canakinumab; Novartis) for the treatment of active Still disease, including adult-onset Still disease. 

Adult-onset Still disease (AOSD) is a rare systemic inflammatory disorder characterized by fever, arthritis, rash and elevated markers for inflammation. The etiology of the disease is unknown, however it is believed to be closely related to systemic juvenile idiopathic arthritis (SJIA).  

Canakinumab is an interleukin-1β (IL-1β) blocker. It works by binding to human IL1β and blocking its interaction with IL-1 receptors, thereby suppressing inflammation in patients with AOSD.

The efficacy of canakinumab in adults with AOSD was based on pharmacokinetic exposure and extrapolation of efficacy data related to canakinumab use in SJIA patients. Efficacy was also assessed in a double-blind, placebo-controlled trial that included patients with AOSD and active joint involvement (n=36). While the study was ended early and the primary end point (proportion of patients with clinically relevant reduction in disease activity at week 12) was not met, the findings showed that treatment did result in improvement of several outcome measures.

With regard to safety, the most common adverse reactions reported with canakinumab in patients with AOSD were infections (nasopharyngitis and upper respiratory tract infections), abdominal pain, and injection site reactions. Additionally, in patients with rheumatic conditions, in particular Still disease, macrophage activation syndrome (MAS) may develop and should be aggressively treated; symptoms of infection or worsening Still disease are known triggers for MAS.

“Prior to today’s approval, patients had no FDA-approved treatments for their disease, which can include symptoms such as painful arthritis, fevers and rash,” said Nikolay Nikolov, MD, acting director of the  Division of Rheumatology and Transplant Medicine in the FDA’s Center for Drug Evaluation and Research. “Today’s approval provides patients with a treatment option.”

Ilaris is already approved for the treatment of SJIA in patients aged 2 years and older. It is also indicated for the treatment of cryopyrin-associated periodic syndromes, tumor necrosis factor receptor associated periodic syndrome, hyperimmunoglobulin D syndrome/mevalonate kinase deficiency, and familial Mediterranean fever. 

Ilaris is supplied in single-dose vials as 150mg lyophilized powder for reconstitution as well as 150mg/mL solution for subcutaneous injection.

For more information visit

This article originally appeared on MPR