Novartis has received positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) for the use of Ilaris (canakinumab, ACZ885) in the treatment of patients with acute gouty arthritis who suffer frequent attacks, and whose symptoms cannot or should not be managed with current treatment options.

"Novartis welcomes the decision by the CHMP in support of the approval of Ilaris in the EU," said David Epstein, Division Head of Novartis Pharmaceuticals. He further added, "When approved, lIaris will provide a new treatment option for patients who have endured frequent and crippling gouty arthritis attacks and where existing therapies do not offer relief. We look forward to receiving the final decision from the European Commission in the coming months."

Ilaris is the only available fully human monoclonal antibody that specifically targets IL-1 beta and, when approved, will offer patients suffering gouty arthritis attacks rapid pain relief via a single subcutaneous injection of 150 mg.

Gouty arthritis, commonly referred to as gout, is a serious, chronic and progressive inflammatory disease that generally affects one to four per cent of adults. Gouty arthritis is associated with a high prevalence of comorbidities, such as hypertension, kidney disease, diabetes, dyslipidemia and cardiovascular disease. These conditions can lead to contraindications for existing therapies and complications for disease management.

When approved, Ilaris will be specifically indicated for the 'symptomatic treatment of adult patients with frequent gouty arthritis attacks (at least three attacks in the previous 12 months) in whom non-steroidal anti-inflammatory drugs (NSAIDs) and colchicine are contraindicated, are not tolerated, or do not provide an adequate response, and in whom repeated courses of corticosteroids are not appropriate'. Since urate lowering therapy (ULT) is generally advised in these patients, Ilaris may provide sufficient pain relief to allow initiation or continuation of ULT.

Data from two phase III trials and their extensions, which informed the CHMP's positive opinion for Ilaris in gouty arthritis, showed that patients treated with Ilaris experienced significantly greater pain relief compared to the injectable steroid triamcinolone acetonide (TA). The majority of adverse events (AEs) were mild to moderate, with infections (e.g. upper respiratory tract infections and nasopharyngitis) being the most frequent of them.

The European Commission generally follows the recommendations of the CHMP and usually delivers its final decision within three months of the CHMP recommendation.

Ilaris has been assessed for the treatment of acute gouty arthritis attacks in two multicentre, randomized, double-blind, active-controlled studies in patients with frequent gouty arthritis attacks (>=3 in the previous year) who were unable to use NSAIDs or colchicine (due to contraindication, intolerance or lack of efficacy). The studies were 12 weeks in duration followed by 12 week double-blind initial extensions.

A total of 454 patients were randomized to receive a single dose of Ilaris 150 mg via subcutaneous injection or TA 40 mg via intramuscular injection.

Pain intensity in the overall study population was statistically significantly lower for Ilaris 150 mg compared to TA at 72 hours (-10.7 mm, p<0.0001), with an absolute mean decrease in VAS score of approximately -50 mm. Reduction in pain was observed as early as 6 hours after dosing in both groups. A statistically significant difference between treatments was observed from 24 hours to seven days. Ilaris also reduced the risk of subsequent attacks.

Safety results showed an increased incidence of AEs for Ilaris compared to TA, with 66% vs. 53% of patients reporting any adverse event and 20% vs. 10% of patients reporting an infection adverse event over 24 weeks.

A sub-analysis of these studies included 101 patients unable to use NSAIDs and colchicine, and on stable ULT or unable to use ULT. Pain relief was similar to that shown in the total study population (-10.2 mm for Ilaris 150 mg compared with TA at 72 hours, p=0.0208).

Ilaris is a selective, fully human, monoclonal antibody that inhibits IL-1 beta, which is an important part of the body's immune system defenses. Excessive production of IL-1 beta plays a prominent role in certain inflammatory diseases. Ilaris works by neutralizing IL-1 beta for a sustained period of time, therefore inhibiting inflammation.

Ilaris is approved in more than 60 countries, including in the EU, US, Switzerland and Japan for the treatment of Cryopyrin-Associated Periodic Syndromes (CAPS), a suite of rare, life-long, genetic, autoinflammatory diseases with debilitating symptoms. Ilaris is being investigated in a number of inflammatory conditions, which include, systemic juvenile idiopathic arthritis (SJIA), Tumor Necrosis Factor Receptor-Associated Periodic Syndrome (TRAPS), Familial Mediterranean Fever (FMF) and cardiovascular disease. Not all patients with these diseases would be eligible for treatment with Ilaris, if approved for the applicable disease.

In the US, Novartis continues to work with the Food and Drug Administration (FDA) to determine the next steps for ACZ885 in gouty arthritis, following a Complete Response letter received in August 2011 with a request by the Agency for additional clinical data to evaluate the benefit risk profile in refractory patients.

Gouty arthritis is the most common form of inflammatory arthritis in adults. The chronic and progressive disease is characterized by recurrent attacks in select joints. These attacks occur when the body has a strong inflammatory response to uric acid crystals forming in the affected joint, typically of the toe, foot, ankle, or knee. The intense inflammatory response associated with these attacks may cause severe pain and debilitating symptoms that can last a week or more.

Treatments currently available to manage the pain and inflammation of gouty arthritis attacks, such as NSAIDs, colchicine or corticosteroids, may be inadequate or inappropriate in patients who have certain coexisting medical problems. As a result, there is a significant unmet medical need among individuals with gouty arthritis.

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