Novartis Gets FDA Okay for Drug to Treat Rare Inflammatory Disorder

FDA approved Novartis’ Ilaris® for the treatment of children and adults with cryopyrin-associated periodic syndrome (CAPS).

Ilaris thus becomes the first approved treatment, according to the company, for patients as young as four years old suffering from two forms of CAPS: familial cold auto-inflammatory syndrome (FCAS) and Muckle-Wells syndrome (MWS).

CAPS is caused by a single gene mutation that leads to overproduction of interleukin-1 beta (IL-1ß), which causes sustained inflammation and tissue damage. Ilaris is a fully human mAb that blocks IL-1ß, and dosing is once every eight weeks.

“Until now, treatments for CAPS patients have been limited to traditional inflammatory disease medications that work by suppressing the entire immune system and newer therapies that control the disease better but require more frequent injections,” notes Hal Hoffman, M.D., who has served as a consultant for the company and is an associate professor of pediatrics and medicine at University of California, San Diego.

CAPS comprises three disorders of increasing severity: FCAS, MWS, and neonatal-onset multisystem inflammatory disease (NOMID). A clinical study is ongoing to evaluate the potential of Ilaris to treat patients with NOMID. There are currently no approved therapies for the treatment of this disorder.

Clinical trials are also under way with Ilaris in systemic juvenile idiopathic arthritis (SJIA) and more common disorders such as some forms of gout, chronic obstructive pulmonary disorder, and type 2 diabetes. Further development in rheumatoid arthritis is not planned.

Ilaris has orphan drug designation for CAPS in the EU, Switzerland, and Australia, where it is currently under health authority review. Priority review has been granted in Switzerland, Australia, and Canada. Ilaris has been granted orphan drug designation for SJIA in the U.S., EU, and Switzerland, and also has fast-track status for SJIA in the U.S.

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