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

FDA News Release

• For Immediate Release: June 16, 2020

The U.S. Food and Drug Administration today approved Ilaris (canakinumab) injection for the treatment of Active Still’s disease, including Adult-Onset Still’s Disease (AOSD). Ilaris was previously approved for Systemic Juvenile Idiopathic Arthritis (SJIA) in patients aged 2 years and older.

“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, M.D., 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.”

AOSD is a rare and serious autoinflammatory disease of unknown origin. Autoinflammatory diseases are 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.

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, under which the FDA’s goal is to take action on an application within six months where the agency determines that the drug, if approved, would significantly improve the safety or effectiveness of treating, diagnosing or preventing a serious condition.

The approval of Ilaris was granted to Novartis Pharmaceuticals Corp.

The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.