The US Food and Drug Administration (FDA) has granted three Breakthrough Therapy Designations for Novartis' Ilaris (canakinumab) to treat three 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, which may be accompanied by joint pain and swelling, muscle pain and skin rashes, with complications that can be life-threatening. Most patients present with symptoms in infancy or childhood. The three conditions for which Ilaris is being reviewed are Tumor Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS) and Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD), as well as Familial Mediterranean Fever (FMF) not adequately controlled with colchicine.

"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."

The FDA considers a treatment a Breakthrough Therapy if it is intended to treat a serious or life-threatening condition and preliminary evidence indicates it may be better than existing treatments. 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 be 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.

Ilaris was approved by the FDA in 2009 to treat two subtypes of a rare autoinflammatory disease called Cryopyrin-Associated Periodic Syndromes (CAPS): Muckle-Wells syndrome (MWS) and Familial Cold Autoinflammatory Syndrome (FCAS), in patients aged four and older. In 2013, the FDA approved Ilaris for a rare, autoinflammatory form of juvenile idiopathic arthritis called Systemic Juvenile Idiopathic Arthritis (SJIA) in patients aged two and older.

Periodic fever syndromes are a group of autoinflammatory diseases that cause serious recurrent fevers that do not have an infectious cause. Most patients present with symptoms in infancy or childhood, but in some patients the condition only becomes apparent or diagnosed in adulthood.

There are no approved medicines available to treat TRAPS or HIDS/MKD and very limited options for patients with FMF. Current treatments, such as oral anti-inflammatory drugs, for example corticosteroids, only help manage the symptoms. While medicines such as non-steroidal anti-inflammatory drugs (NSAIDs), have some effect at reducing symptoms, they do not prevent or change the course of an episode of fever.

Of the three rare conditions for which Ilaris is being reviewed by the FDA, FMF is the most common and mainly affects people of Eastern Mediterranean ancestry, affecting 1 in 250 to 1 in 1,000 individuals in these populations. It is far less common in other ethnic groups, but it can affect anyone. TRAPS and HIDS/MKD are less common, with only about 1-2 people per million affected.

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.

Ilaris is approved for the treatment of SJIA in the US and for the symptomatic treatment of refractory acute gouty arthritis in the EU. Ilaris is also approved in more than 70 countries, including in the EU, Switzerland, Canada, and Japan for the treatment of CAPS, rare, lifelong, genetic disorders with debilitating symptoms. In the US, Ilaris is approved for two subtypes of CAPS: MWS and FCAS. The approved indications may vary depending upon the individual country.