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Rilonacept Approved in the EU for Cryopyrin-Associated Periodic Syndromes

TARRYTOWN, NY -- October 27, 2009 -- The interleukin-1 (IL-1) blocker rilonacept (Arcalyst) is now approved in the European Union for the treatment of cryopyrin-associated periodic syndromes (CAPS) with severe symptoms, including familial cold auto-inflammatory syndrome (FCAS) and Muckle-Wells syndrome (MWS), in adults and children over age 12 years.

CAPS include a group of rare, inherited, auto-inflammatory conditions characterised by life-long, recurrent symptoms of rash, fever/chills, joint pain, eye redness/pain, and fatigue. Intermittent, disruptive exacerbations or flares can be triggered at any time by exposure to cooling temperatures, stress, exercise, or other unknown stimuli.

Rilonacept is a targeted inhibitor of IL-1, the key driver of inflammation in CAPS. In the clinical development programme, patients treated with rilonacept reported a greater improvement in overall symptom scores than patients treated with placebo. These improvements were sustained over time with continued rilonacept treatment. Patients reported their symptoms using a validated daily diary instrument. These assessments represent a critical measure of effectiveness in a disease characterised by frequent, unpredictable symptom flares of variable severity and duration. Unlike other agents used in the treatment of CAPS, rilonacept is supported by patient-reported symptoms data using a validated assessment instrument.

Rilonacept is a once-weekly injection which can be administered at home by the patient or their caregiver following appropriate training. The most commonly reported adverse reactions with rilonacept were injection-site reaction and upper respiratory tract infection. IL-1 blockade may interfere with immune response to infections and treatment should not be initiated in patients with active or chronic infections. Serious, life-threatening infections have been reported in patients taking rilonacept; rilonacept should be discontinued if a patient develops a serious infection.

The authorisation for approval of rilonacept in the European Union under exceptional circumstances is permissible for products for which a company can demonstrate that comprehensive data cannot be provided, for example, because of the rarity of the condition. Each year, Regeneron will need to provide the European Medicines Agency (EMEA) with any new information that may become available for review.

Rilonacept has not been studied in patients with neonatal-onset multisystem inflammatory disease (NOMID), 1 of the 3 conditions that make up CAPS.

Approximately 1 in 1,000,000 people in the European Union have CAPS. CAPS are generally caused by autosomal-dominant mutations in the NLRP-3 (previously known as CIAS1) gene and resultant alterations in the protein cryopyrin, which it encodes. Cryopyrin, active in circulating, infection-fighting, white blood cells, controls the production of IL-1. As part of the body's infection-fighting defence system, IL-1 circulates throughout the body and can trigger inflammatory reactions when it binds to inflammatory cells. Researchers have found that alterations in the cryopyrin protein lead to over-production of IL-1, resulting in an inflammatory response and the symptoms of CAPS. Most, but not all, patients with CAPS have the NLRP-3 gene mutation.

SOURCE: Regeneron Pharmaceuticals, Inc.

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