Canakinumab (Ilaris) Gets FDA Nod for Three Rare Periodic Fever Syndromes

Megan Brooks

September 26, 2016

The US Food and Drug Administration (FDA) has approved three new indications for the interleukin-1-β inhibitor canakinumab (Ilaris, Novartis).

They are tumor necrosis factor receptor–associated periodic syndrome (TRAPS), hyperimmunoglobulin D syndrome /mevalonate kinase deficiency (HIDS/MKD), and familial Mediterranean fever (FMF).

The three hereditary syndromes are all characterized by periodic attacks of fever and inflammation, along with severe muscle pain. Until now, there have been no approved therapies for TRAPS or HIDS/MKD.

"For the first time, patients with TRAPS and HIDS/MKD, two painful and life-altering diseases, have access to a treatment that may help improve their quality of life," Badrul Chowdhury, MD, PhD, director of the Division of Pulmonary, Allergy and Rheumatology Products in FDA's Center for Drug Evaluation and Research, said in a statement.

TRAPS has an estimated prevalence of 1 per million individuals and is the second most common inherited recurrent fever syndrome after FMF. More than 1000 people worldwide have been diagnosed with TRAPS.

FMF primarily affects populations originating in the Mediterranean region, particularly people of Armenian, Arab, Turkish, or Jewish ancestry. The disorder affects 1 in 200 to 1000 people in these populations. It is less common in other populations.

HIDS/MKD is also extremely rare and is mostly found in Western Europe, especially in the Netherlands and France, but has been described in all ethnic groups.

The FDA approved the three new indications on the basis of clinical studies, including safety, efficacy, and pharmacokinetic data, the agency said. The most common adverse reactions for these indications are injection site reactions and increased susceptibility to infection due to the drug's immune-suppressing effects. Allergic reactions have also been reported. Patients should not receive live vaccines while receiving canakinumab.

Canakinumab is already approved for another rare periodic fever syndrome — cryopyrin-associated periodic syndromes — and for active systemic juvenile idiopathic arthritis.

The FDA advises healthcare professionals to review the prescribing information in the labeling for detailed information about the approved uses.

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