FDA Breakthrough status for Novartis drug in Periodic Fever Syndromes

The US regulator has said it hopes to give Novartis’ Ilaris (canakinumab) a fast review in three types of Periodic Fever Syndromes, after granting it Breakthrough Therapy status in these new uses.

If approved, Novartis said Ilaris will likely be the first approved treatment for Tumour Necrosis Factor-Receptor Associated Periodic Syndrome (TRAPS) and Hyperimmunoglobulin D Syndrome (HIDS)/Mevalonate Kinase Deficiency (MKD), as well as Familial Mediterranean Fever not adequately controlled with colchicine.

Should the FDA approve Ilaris on the basis of the Phase 3 CLUSTER trial, this could be a boost for the drug, which generated sales of around $62 million in Q1 this year, as it is already approved in a rare autoinflammatory disease.

Novartis has several other drugs in its immunology/dermatology portfolio, including recently launched psoriasis drug Cosentyx (secukinumab), which amassed sales of $176m in Q1.

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 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. The designation means the FDA will give the drug a faster review if data appears to be favourable.

The Breakthrough Therapy Designations were granted based on the pivotal Phase 3 CLUSTER trial. Based on this study Novartis submitted three filings 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.

Ilaris 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. Excessive production of IL-1 beta plays a prominent role in certain inflammatory diseases.

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