Novartis’ Ilaris wins three FDA breakthrough designations to treat periodic fever syndromes

The three conditions for which Ilaris is being reviewed include tumor necrosis factor-receptor associated periodic syndrome (TRAPS) and Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD), and Familial Mediterranean Fever (FMF) not adequately controlled with colchicine.

The diseases result in disabling and recurrent fevers, possibly accompanied by joint pain and swelling, muscle pain and skin rashes, with complications that can be life-threatening.

There are very limited options for patients with FMF and no approved medicines available to treat TRAPS or HIDS/MKD.

Novartis said the designations support potential expedited review of Ilaris to help address the unmet need of patients with the three rare conditions.

The regulator’s decision was based on data from the pivotal phase III Cluster trial, which has also formed the basis for three supplemental biologic license applications.

Ilaris inhibits Interleukin-1 (IL-1) beta, which is an important part of the body’s immune system defenses.

Over production of IL-1 beta plays a key role in several inflammatory diseases. Ilaris blocks the action of IL-1 beta for a sustained period of time, resulting the inhibition of inflammation that is caused by its excessive production.

The FDA approved Ilaris in 2009 to treat two subtypes of a rare autoinflammatory disease called Cryopyrin-Associated Periodic Syndromes: Muckle-Wells syndrome and Familial Cold Autoinflammatory Syndrome, in patients aged four and older.

In 2013, Ilaris secured the FDA approval for a rare, autoinflammatory form of juvenile idiopathic arthritis called Systemic Juvenile Idiopathic Arthritis in patients aged two and older.

Ilaris is approved for the symptomatic treatment of refractory acute gouty arthritis in the European Union.

Image: Novartis Logo, Basel Headquarters. Photo: courtesy of Novartis AG.