CAPS are characterized 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.
CAPS are generally caused by autosomal-dominant mutations (changes) 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 a protein called interleukin-1 (IL-1). As part of the body's infection-fighting defense 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.
The incidence of CAPS has been estimated to be approximately 1 in 1,000,000 people in the European Union.
Rilonacept is a targeted inhibitor of interleukin-1 (IL-1), the key driver of inflammation in Cryopyrin-Associated Periodic Syndromes (CAPS). In the pivotal clinical development program for rilonacept, change in disease activity was measured using a composite patient-reported symptom score composed of a daily evaluation of rash, feelings of fever/chills, joint pain, eye redness/pain, and fatigue. Patients treated with rilonacept experienced an improvement in overall symptom scores as compared with patients treated with placebo. These improvements were sustained over time with continued treatment with rilonacept.
|SOURCE Regeneron Pharmaceuticals, Inc.|
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