Novartis’ Ilaris provides hope for Periodic Fever Syndrome patients

Novartis announced today that the US Food and Drug Administration (FDA) has granted three breakthrough therapy designations for Ilaris® (canakinumab) to treat rare types of Periodic Fever Syndromes, also known as Hereditary Periodic Fevers. This means Novartis will work closely with the FDA to expedite the regulatory review of Ilaris for these conditions.

Periodic Fever Syndromes are a group of autoinflammatory diseases that cause disabling and recurrent fevers, with complications that can be life-threatening. The three conditions for which Ilaris is being reviewed are Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS), Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD), and Familial Mediterranean Fever (FMF) – not adequately controlled with colchicine. 

 Novartis offer hope

“This is an important day for patients, including many children, who are affected by these serious and debilitating syndromes that have no or limited treatment options,” said David Epstein, Division Head, Novartis Pharmaceuticals. “Ilaris is a promising medicine under review for these conditions, marking our commitment to making a significant difference to the lives of people with rare diseases.”

Novartis move toward unique treatment

There are presently no approved medicines available to treat TRAPS or HIDS/MKD and very limited options for patients with FMF. Current treatments only help manage the symptoms and do not prevent or change the course of an episode of fever.
If approved, Ilaris will likely be the first medicine to gain approval from drug regulators for the treatment of TRAPS and HIDS/MKD and it will provide an alternative to the only FDA-approved treatment for FMF: colchicine. 

The Breakthrough Therapy Designations were granted based on the pivotal Phase III CLUSTER trial. Based on this study Novartis submitted three supplemental Biologic License Applications in the US to register Ilaris for use in these indications.

How Ilaris works

Ilaris (canakinumab) is a selective, high-affinity, and human monoclonal antibody that inhibits Interleukin-1 (IL-1) beta, which is an important part of the body’s immune system defenses. Excessive production of IL-1 beta plays a prominent role in certain inflammatory diseases. Ilaris works by blocking the action of IL-1 beta for a sustained period of time, therefore, inhibiting inflammation that is caused by its over-production.