Rilonacept for cryopyrin associated periodic syndromes
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1 Rilonacept for cryopyrin associated periodic syndromes August 2009 This technology summary is based on information available at the time of research and a limited literature search. It is not intended to be a definitive statement on the safety, efficacy or effectiveness of the health technology covered and should not be used for commercial purposes. The Research Programme is part of the National Institute for Health Research
2 Rilonacept for cryopyrin associated periodic syndromes Target group Cryopyrin associated periodic syndromes (CAPS) in people aged 12 and over with severe symptoms of: Muckle-Wells Syndrome (MWS). Familial cold autoinflammatory syndrome (FCAS) also known as familial cold uriticaria (FCU). Background CAPS is a family of three autoinflammatory syndromes MWS, FCAS and chronic infantile neurological, cutaneous, and articular syndrome (CINCA). These syndromes have been linked to mutations in the gene (CIAS-1) encoding cryopyrin (NALP3), an immune system regulatory protein. Symptoms include varying severity of fever, fatigue, skin rashes, conjunctivitis, painful joints and muscles, headaches and aseptic meningitis, often in response to exposure to cool ambient temperatures. Skeletal and neurological abnormalities during growth and development are quite common, and include distortion of the face, deafness and sometimes blindness. Features of the disease generally become evident shortly after birth, and those relating to acute inflammation persist in a fluctuating manner for the remainder of the patient s life. About 25% of patients develop systemic amyloid associated (AA) amyloidosis as a result of the chronic inflammation, typically in early adult life and resulting in renal failure, dialysis dependence and death within 5-10 years. Technology description. Rilonacept (Arcalyst, IL-1 Trap) is a dimeric fusion protein made up of the extracellular domains of both of the interleukin-1 (IL-1) receptor components: IL-1 receptor type 1 and IL-1 receptor accessory protein linked to a portion of human immunoglobulin G. Rilonacept binds to IL-1 blocking its interaction with cell surface receptors. It is administered by subcutaneous injection (SC) at 160mg per week. The company state that rilonacept should not be administered to people with an active or chronic infection, and serious life-threatening infections have been reported with its use. Taking rilonacept with tumour necrosis factor inhibitors is not recommended because this may increase the risk of serious infections. Rilonacept is also in phase III clinical trials for gout. Innovation and/or advantages There is currently no licensed treatment for CAPS in Europe. Rilonacept will be one of the first licensed treatments for this indication. Developer Regeneron Pharmaceuticals Inc. Availability, launch or marketing dates, and licensing plans: In phase III clinical trials. NHS or Government priority area: No NHS or government priority area was identified. 2
3 Relevant guidance No relevant guideline was identified. Clinical need and burden of disease There are an estimated 500 people with CAPS in the EU 1. There are believed to be no more than 60 cases in the UK. Existing comparators and treatments There is no standard therapy for CAPS within the EU. Severe flares are managed symptomatically with anti-inflammatory products. Daily anakinra (Kineret, unlicensed) an IL-1 receptor antagonist, has been reported to be effective in anecdotal case reports. Efficacy and safety Trial NCT ; FCAS and MWS; rilonacept vs placebo; phase III (stage 1). NCT ; FCAS and MWS; rilonacept vs placebo; phase III (stage 2). NCT extension; FCAS and MWS. Sponsor Regeneron. Regeneron. Regeneron. Status Published 2. Published 2. Abstract 3. Location USA. USA. USA. Design Participants and schedule Randomised, double blind, controlled, stratified by disease activity. n=47; adults; FCAS (n=44) or MWS (n=3). Randomised to loading dose rilonacept 320mg then rilonacept 160mg per week or placebo for 6 wks. Single blind extension, then randomised, controlled, withdrawal trial. n=46 completing NCT (stage 1); all participants received rilonacept 160mg per week for 9 weeks, then randomised to rilonacept 160mg per week or placebo for 9 weeks. Open-label extension. n=56; completing NCT and 12 new patients. Received rilonacept 160mg per week. Follow-up 6 weeks treatment period. 18 weeks treatment period. 24 weeks additional treatment period. Primary outcomes Patient assessed disease activity (key symptoms). Patient assessed disease activity (key symptoms). Patient assessed disease activity Secondary outcomes Key results Response: 30, 50 or 70% response in key symptoms, multi- and single symptom disease flare days, physicians assessment of disease activity, limitations in daily activities, C-reactive protein and serum amyloid A (SAA), safety. Mean change in key symptom score -2.6 with rilonacept and -0.3 with placebo (p=0.0001). Rilonacept reduced the number of multi-symptom (rilonacept -8.5, placebo -1.2) and singlesymptom (rilonacept -12.1, placebo -1.2) flare days (p<0.0001), and significantly improved the physician s Response: 30, 50 or 70% response in key symptoms, multi- and single symptom disease flare days, physicians assessment of disease activity, daily activities, C-reactive protein and serum amyloid A (SAA), safety. 9 week extension: patients on rilonacept from stage 1 maintained benefits; patients on placebo in stage 1 had rapid improvement in symptoms. Withdrawal study: rilonacept maintained low key symptom scores vs. placebo (+0.1 vs. +1.0, p=0.0002). Also significantly better at (key symptoms). Disease flare days, C-reactive protein and serum amyloid A (SAA), safety. The efficacy of rilonacept was sustained over the additional 24 week period. 3
4 Adverse effects (AEs) global assessment of health (p<0.0001) and all other secondary outcomes. 74% of the rilonacept and 54% of placebo group experienced treatment emergent AEs. The most common AEs with rilonacept was injection site reaction (48%) and upper respiratory tract infection (26%). 1 discontinuation due to preexisting hepatitis C virus infection. maintaining low multi- and single symptom flare days, and physician s global assessment of health. The most common AEs were injection site reaction (rilonacept 36%, placebo 13%). Other common AEs with rilonacept included headache, arthralgia and diarrhoea. Estimated cost and cost impact The cost of Rilonacept has yet to be determined. Potential or intended impact speculative Patients Reduced morbidity Quicker, earlier or more accurate diagnosis or identification of disease Services Increased use: weekly SC administration Decreased use e.g. shorter length of stay, reduced referrals Costs Increased unit cost compared to alternative New costs: References Reduced mortality or increased length of survival Service reorganisation required Increased costs: more patients coming for treatment Savings: Potential if efficacy reduces other NHS costs Improved quality of life for patients and/or carers None identified Staff or training required None identified Increased costs: capital investment needed 1 European Medicines Agency (EMEA). Pre-authorisation evaluation of medicines for human use. Committee for orphan medicinal products. Public summary of positive opinion for orphan designation of rilonacept for the treatment of cryopyrin-associated periodic syndromes (familial cold urticaria syndrome (FCUS), Muckle - Wells syndrome (MWS), and neonatal onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurological cutaneous articular syndrome CINCA). 29 th July Accessed 6 th July Hoffman HM, Throne ML, Amar NJ, et al. Efficacy and safety of rilonacept (Interleukin-1 Trap) in patients with cryopyrin associated periodic syndromes. Arthritis & Rheumatism 2008;58: Hoffman H, Wolfe F, Kavanaugh A et al. Sustained efficacy of rilonacept (IL-1 Trap) in patients with cryopyrin-associated periodic syndromes (CAPS). American Collage of Rheumatology 2007 Annual Scientific Meeting. Abstract
5 The National Institute for Health Research Research Programme is funded by the Department of Health. The views expressed in this publication are those of the author and not necessarily those of the NHS, the NIHR or the Department of Health The, Department of Public Health and Epidemiology University of Birmingham, Edgbaston, Birmingham, B15 2TT, England Tel: +44 (0) Fax +44 (0)
Familial Cold Autoinflammatory Syndrome
Familial Cold Autoinflammatory Syndrome The National Organization for Rare Disorders (NORD) web site, its databases, and the contents thereof are copyrighted by NORD. No part of the NORD web site, databases,
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