FDA Approves First Treatment for Adult Onset Still’s Disease, a Severe and Rare Disease

Rare Daily Staff

The U.S. Food and Drug Administration approved expanded use of Novartis’ Ilaris injection for the treatment of active Still’s disease, including adult-onset Still’s disease, a rare and serious autoinflammatory disease.

Ilaris (canakinumab) was previously approved for systemic juvenile idiopathic arthritis in patients aged 2 years and older.

Adult-onset Still’s (AOSD) disease is caused by abnormalities of the immune system, which trigger an inflammatory response that can damage the body’s own tissues. Characteristics of AOSD have considerable overlap with Systemic Juvenile Idiopathic Arthritis (SJIA), which includes fever, arthritis, rash, and elevated markers for inflammation. The overlapping features of AOSD and SJIA suggest this is a disease continuum rather than two separate diseases.

The role of interleukin-1 (IL-1), a type of cytokine important in regulating the body’s immune system, is well-established in AOSD and SJIA. Ilaris works by blocking the effects of IL-1 and suppressing inflammation in patients with this autoinflammatory disorder. The safety and efficacy of Ilaris for the treatment of patients with AOSD was established using comparable pharmacokinetic exposure and extrapolation of established efficacy of canakinumab in patients with SJIA, as well as the safety of canakinumab in patients with AOSD and other diseases.

“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, 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.”

Common side effects reported by patients treated with Ilaris are infections (colds and upper respiratory tract infections), abdominal pain and injection-site reactions. The prescribing information for Ilaris includes a warning for potential increased risk of serious infections due to IL-1 blockade. Macrophage activation syndrome (MAS) is a known, life-threatening disorder that may develop in patients with rheumatic conditions, in particular Still’s disease, and should be aggressively treated. Treatment with immunosuppressants may increase the risk of malignancies. Patients are advised not to receive live vaccinations during treatment.

Ilaris was granted Priority Review designation.