Ilaris Gains FDA Approval for CAPS

Novartis received approval from the FDA for Ilaris (canakinumab) for the treatment of children and adults with cryopyrin-associated periodic syndrome (CAPS), which includes a number of rare but life-long auto-inflammatory disorders with debilitating symptoms and limited treatment options. The drug was granted priority review based on its potential to meet an important clinical need for CAPS patients.

Ilaris is the first approved treatment for patients as young as four years old suffering from two forms of CAPS: familial cold auto-inflammatory syndrome (FCAS) and Muckle-Wells syndrome. 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 including long-term consequences such as deafness, bone and joint deformities, central nervous system damage leading to visual loss, and amyloidosis resulting in renal failure and early death.

Ilaris, previously known as ACZ885, is a fully human monoclonal antibody that rapidly and selectively blocks IL-1ß. The dosing schedule for Ilaris is once every eight weeks, which is less frequent than the current approved therapy.

Clinical trials for Ilaris are also underway in systemic juvenile idiopathic arthritis (SJIA), gout, chronic obstructive pulmonary disorder (COPD) and type 2 diabetes.