INVESTORS & MEDIA
News Release
Regeneron Receives European Medicines Agency Positive Opinion for Marketing Authorization of Rilonacept for the Treatment of Cryopyrin-Associated Periodic Syndromes (CAPS)
“We are very pleased to receive the EMEA's positive opinion for
rilonacept,” said
CAPS are a group of rare, inherited, auto-inflammatory conditions 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.
Rilonacept is a targeted inhibitor of interleukin-1 (IL-1), the key driver of inflammation in CAPS. In the pivotal clinical development program, 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. Patient-reported symptoms assessment, using a validated daily diary instrument, represents a critical measure of effectiveness in a disease characterized 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 has been developed as a once-weekly injection which can be administered at home by the patient or their care giver 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. Serious, life-threatening infections have been reported in patients taking rilonacept. Treatment should not be initiated in patients with active or chronic infections. Rilonacept should be discontinued if a patient develops a serious infection.
About Cryopyrin-Associated Periodic Syndromes (CAPS)
Recently, medical researchers have identified and described a group of rare, inherited, auto-inflammatory disorders, known as Cryopyrin-Associated Periodic Syndromes or CAPS. Three related conditions make up the broader disease known as CAPS: Familial Cold Auto-inflammatory Syndrome (FCAS), Muckle-Wells Syndrome (MWS), and Neonatal-Onset Multisystem Inflammatory Disease (NOMID). Rilonacept has not been studied in patients with NOMID.
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
About Rilonacept
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. The most commonly reported adverse reactions with rilonacept
were injection-site reaction and upper respiratory tract infection.
In
IL-1 blockade may interfere with immune response to infections. Serious, life-threatening infections have been reported in patients taking ARCALYST. ARCALYST should be discontinued if a patient develops a serious infection. Treatment with ARCALYST should not be initiated in patients with active or chronic infections. Taking ARCALYST with tumor necrosis factor inhibitors is not recommended because this may increase the risk of serious infections. Patients should not receive a live vaccine while taking ARCALYST. It is recommended that patients receive all recommended vaccinations prior to initiation of treatment with ARCALYST. Patients should be monitored for changes in their lipid profiles and provided with medical treatment if warranted. Hypersensitivity reactions associated with ARCALYST® (rilonacept) administration have been rare. Please see the full U.S. Prescribing Information for ARCALYST, available online at www.regeneron.com/ARCALYST-fpi.pdf.
About
Regeneron is a fully integrated biopharmaceutical company that
discovers, develops, and commercializes medicines for the treatment of
serious medical conditions. In addition to ARCALYST® (rilonacept)
Injection for Subcutaneous Use, its first commercialized product in
Forward Looking Statement
This news release discusses historical information and includes
forward-looking statements about Regeneron and its products, development
programs, finances, and business, all of which involve a number of risks
and uncertainties, such as risks associated with preclinical and
clinical development of Regeneron’s drug candidates, determinations by
regulatory and administrative governmental authorities which may delay
or restrict Regeneron’s ability to continue to develop or commercialize
its product and drug candidates, competing drugs that are superior to
Regeneron’s product and drug candidates, uncertainty of market
acceptance of Regeneron’s product and drug candidates, unanticipated
expenses, the availability and cost of capital, the costs of developing,
producing, and selling products, the potential for any collaboration
agreement, including Regeneron’s agreements with the sanofi-aventis
Group and
Source:
Regeneron Pharmaceuticals, Inc.
Peter Dworkin, 914-345-7640
Investor
Relations
peter.dworkin@regeneron.com
or
Laura
Lindsay, 914-345-7800
Media Relations
laura.lindsay@regeneron.com