Novartis’ Ilaris has picked up three breakthrough designations in the US for potential indications spanning a group of auto inflammatory disorders called Periodic Fever Syndromes or Hereditary Periodic Fevers.

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), and Familial Mediterranean Fever (FMF) not adequately controlled with colchicine.

These diseases cause 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 currently no approved medicines available to treat TRAPS or HIDS/MKD and very limited options for patients with FMF. 

“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 Breakthrough Therapy designations were granted based on data from the pivotal Phase III CLUSTER trial, which has also formed the basis for three supplemental Biologic License Applications in the US.

Ilaris was originally approved by US regulators in 2009 to treat two subtypes of a rare autoinflammatory disease called Cryopyrin-Associated Periodic Syndromes: Muckle-Wells syndrome and Familial Cold Autoinflammatory Syndrome, and subsequently for Systemic Juvenile Idiopathic Arthritis in 2013.