Ilaris recommended for EU approval to treat group of auto-inflammatory diseases

Novartis medicine Ilaris (canakinumab) has been recommended for approval in the European Union to treat patients with cryopyrin-associated periodic syndrome (CAPS), a potentially fatal auto-inflammatory disease. When approved, Ilaris will be the only treatment in the EU indicated for CAPS patients aged four years and older.

Ilaris targets a condition that is triggered by a specific genetic mutation. In CAPS patients, this mutation drives the overproduction of interleukin 1-beta (IL-1ß), which causes the widespread sustained inflammation and tissue damage associated with the disease.

Because Ilaris normalises the production of IL-1ß, it is also being studied in other diseases in which IL-1ß plays a pivotal role such as systemic juvenile idiopathic arthritis (SJIA), gout, chronic obstructive pulmonary disorder (COPD), and Type 2 diabetes.

"By concentrating initially on a rare syndrome with a well-defined disease process such as CAPS, we have been able to demonstrate a clear therapeutic advantage with Ilaris," said Trevor Mundel, head of global development at Novartis Pharma. "Our focus now is to establish whether this could also provide a new approach to the treatment of other diseases involving a similar underlying process."

The recommendation for approval of Ilaris comes shortly after approvals in the US and Switzerland where Ilaris was granted a priority review based on its potential to fulfil an important unmet need for CAPS patients.

The EU submission was supported by data showing that Ilaris, a monoclonal antibody formerly known as ACZ885, produced rapid and sustained remission of symptoms in up to 97% of CAPS patients, with most responding from the first injection.

CAPS is believed to occur in around 6,500 patients worldwide and 2,500 in the EU. However, due to lack of diagnosis or misdiagnosis, fewer than 1,000 cases have been officially reported worldwide.

The Ilaris filing was based on a clinical trial programme involving more than 100 CAPS patients. The study is a three-part, one-year Phase III study involving 35 patients aged nine to 74 with varying degrees of disease severity.