Pharmaceutical Business review

FDA approves expanded use of Novartis’ Ilaris to treat three rare periodic fever syndromes

The approvals for the new indications, tumor necrosis factor-receptor associated periodic syndrome (TRAPS), hyperimmunoglobulin D syndrome (HIDS)/mevalonate kinase deficiency (MKD) and familial Mediterranean fever (FMF), were based on clinical studies, including safety, efficacy and pharmacokinetic data.

At 16 weeks, patients prescribed Ilaris had rapid and sustained disease control versus patients who received placebo.  

Because of the positive data from the phase III trial , the FDA granted breakthrough therapy status and priority reviews to Ilaris for each of the three Periodic Fever Syndrome conditions.

FDA center for drug evaluation and research director of the division of pulmonary, allergy and rheumatology products Badrul Chowdhury said: “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.”

Novartis Pharmaceuticals CEO Paul Hudson said: "Gaining three simultaneous FDA approvals of Ilaris is a momentous turning point for people who suffer with these severe, debilitating diseases.

"There is a significant unmet need in these conditions, particularly in children, and Novartis is proud to provide a new treatment option to those who so desperately need it."

Ilaris is a selective, high-affinity, human monoclonal antibody that inhibits Interleukin-1 (IL-1) beta, which is a key part of the body's immune system defenses.

It blocks the action of IL-1 beta for a sustained period of time, therefore inhibiting inflammation that is caused by its excess-production.


Image: Novartis headquarters in Basel. Photo: courtesy of –Andrew- from Flickr.