Novartis Ilaris Achieves Goals in Study of Rare Childhood Arthritis

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Less than one child in 100,000 has systemic juvenile idiopathic arthritis (SJIA), a condition caused by inflammation and characterized by life-long, recurrent painful flares. The cause of the autoimmune disease is unknown, with treatment focusing on reducing symptoms. Most are treated with steroid therapy which has side effects such as slowed growth and delayed puberty. Novartis AG has released the results of a Phase III study which shows their experimental medication known as Ilaris effectively reduces steroid use in almost half of patients with the disease.

The drug, formerly known as ACZ885 and generically as canakinumab, was given to 177 SJIA patients between the ages of 1 and 19 years in a two-part study. In Part I, patients received a subcutaneous dose of Ilaris equaling 4 mg/kg up to 300 mg every 4 weeks. After eight weeks, patients who met certain criteria began tapering their steroid use. In Part II, patients were randomized to either continue receiving the drug or to receive a placebo every 4 weeks until a pre-specified number of flares had occurred.

ACZ885 is a human monoclonal antibody which works by neutralizing the key inflammatory mediator, interleukin-1 beta (IL-1 beta), which plays a role in a number of inflammatory diseases, including SJIA.

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Ilaris helped substantially reduce steroid use (at least 25%) in 45% of patients. SJIA patients on the experimental drug were nearly three times less likely to suffer a new flare versus those on the placebo.
"These data are very welcome because nearly half of ACZ885-treated patients were abl
e to reduce their steroid use during the study, potentially helping decrease the impact that these drugs can have on this young population," said Dr Nico Wulffraat, pediatric immunologist at Wilhelmina Children's Hospital, University Medical Center in The Netherlands. "One of our main goals as physicians, and this applies to all therapies, is to provide patients with treatment options that combine effective control of their disease with a favorable safety profile, making long-term safety data monitoring important."

The most common adverse events, occurring in about 80% of patients, were nasopharyngitis, headache and cough. Fifteen patients had serious events that included infections and flares. One patient in the placebo group died of Macrophage Activation Syndrome (MAS), a known, potentially fatal condition associated with SJIA.

The results of this study were presented at the American College of Rheumatology’s Annual Scientific Meeting, held in Chicago. Another trial was previously presented at the 2011 European Pediatric Rheumatology Congress in Belgium in September.

Novartis hopes to submit for FDA approval for Ilaris in 2012. It is already approved for the treatment of Cryopyrin-Associated periodic Syndromes (CAPS – another inflammatory disorder) in more than 50 countries , including the European Union, Switzerland, Japan, and the US.

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