{"id":2346,"date":"2023-01-19T22:24:08","date_gmt":"2023-01-19T22:24:08","guid":{"rendered":"https:\/\/vimizimhcp-dev-001.azurewebsites.net\/en-us\/vimizim\/?page_id=2346"},"modified":"2023-05-03T11:38:22","modified_gmt":"2023-05-03T11:38:22","slug":"endurance","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/endurance\/","title":{"rendered":"Endurance"},"content":{"rendered":"<div id=\"acf-block-63fc97d21f15d\" class=\"page-header\">         \n            \n        <div class=\"header-image\" style=\"background-image: url(https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2022\/09\/Derric.png?v=0.46)\"><\/div>\n     \n    <div class=\"header-content\">\n                    <h1>VIMIZIM<sup>\u00ae<\/sup> (elosulfase alfa) demonstrated early and sustained improvement in endurance<sup>1-3<\/sup><\/h1>\n            <\/div>\n<\/div>\n\n<div id=\"acf-block-63fc97d21fca6\" class=\"block tabbed-content\">\n\t    <div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t                                                                                        <ul class=\"tabs\">\n                                                                                                                            <li><a href=\"#acf-block-63fc97d21fca6_0\">Pivotal Study Results\n<\/a><\/li>\n                                                                                                                                                            <li><a href=\"#acf-block-63fc97d21fca6_1\">Long-term Extension Study Results\n<\/a><\/li>\n                                                                                    <\/ul>\n                                                                                                                                                <div class=\"box\">\n                                                                                            <div class=\"tab-content\" id=\"acf-block-63fc97d21fca6_0\" style=\"display:none;\">\n                                                                                                                                                                                                                        <h2>In a 24-week clinical trial, people who took VIMIZIM improved their endurance as measured by the 6-minute walk test (6MWT)<sup>1,2<\/sup><\/h2>\n                                                                                                                                                                                                                                                                                                                                <div class=\"block split-content\">\n                                                    <div class=\"wrapper\">\n\t\t                                                <div class=\"inner-wrapper\">\n                                                                                                                            <div class=\"content-block\">\n                                                                                                                                                                                                                        <p><strong>MOR-004: a phase 3, double-blind, randomized, placebo-controlled trial (N=176)<sup>2,3<\/sup><\/strong><br \/>\nTreatment with VIMIZIM<sup>\u00ae<\/sup> (elosulfase alfa) 2 mg\/kg\/wk demonstrated:<\/p>\n<ul>\n<li>Improvement in 6MWT was seen as early as 12 weeks<sup>2<\/sup><\/li>\n<li>22.5-meter improvement in 6MWT over placebo at 24 weeks (P=0.0174)<sup>1,2<\/sup><\/li>\n<\/ul>\n<p><strong><em>Patients in the VIMIZIM once-weekly treatment arm saw a 23.9% mean improvement in 6MWT over baseline<sup>4<\/sup><\/em><\/strong><\/p>\n<p>The 6MWT is a validated measure of endurance in the clinical setting that evaluates the functional reserves of the cardiovascular, pulmonary, or musculoskeletal systems.<sup>5<\/sup><\/p>\n<p>Learn about the overall pivotal phase 3 <a href=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/study-design\/\">study design<\/a><\/p>\n                                                                                                                                                                                                            <\/div>\n                                                            \n                                                                                                                            <div class=\"content-block\">\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                    \n                                                                            \n                                                                            \n                                                                            \n                                                                            <figure>\n                                                                                <div class=\"image\">\n                                                                                                                                                                            <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2023\/01\/6MWT.png?v=0.46\" alt=\"\" \/>                                                                                                                                                                    <\/div>\n                                                                                                                                                            <\/figure>\n                                                                                                                                                                                                            <\/div>\n                                                                                                                    <\/div>\n                                                    <\/div>\n                                                <\/div>\n                                                                                                                                                                                                                                <h2 class=\"h3\">VIMIZIM demonstrated positive results across many efficacy measures at 24 weeks<sup>6<\/sup><\/h2>\n<p>Although statistical significance was only observed in the 6MWT results, improvement was seen in the majority of exploratory efficacy endpoints. The sample size for the study was selected to be sufficient to detect the impact of treatment on only the primary and secondary endpoints. Tertiary endpoints were intended to be exploratory in nature, and this data should be interpreted with caution.<sup>2<\/sup><\/p>\n<p><small><strong>FET<\/strong>, forced expiratory time; <strong>FEV1<\/strong>, forced expiratory volume in 1 second; <strong>FIVC<\/strong>, forced inspiratory vital capacity; <strong>FVC<\/strong>, forced vital capacity; <strong>MPS HAQ<\/strong>, Mucopolysaccharidoses Health Assessment Questionnaire; <strong>MVV<\/strong>, maximum voluntary ventilation; <strong>3MSCT<\/strong>, 3-minute stair-climbing test; <strong>Z<\/strong>-score, how far a measurement deviates from the average.<\/small><\/p>\n                                                                                                                                                                                                                                                                                                                                                                                                                                \n                                                \n                                                \n                                                \n                                                <figure>\n                                                    <div class=\"image\">\n                                                                                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2022\/08\/endurance-1-1900x986.png\" alt=\"endurance 1\" \/>                                                                                                            <\/div>\n                                                                                                    <\/figure>\n                                                                                                                                                                                                                                <h2 class=\"h3\">Adverse reactions<sup>1<\/sup><\/h2>\n<ul>\n<li>In clinical trials, the most common adverse reactions (\u226510%) occurring during infusion included pyrexia, vomiting, headache, nausea, abdominal pain, chills, and fatigue<\/li>\n<li>Serious and severe reactions were associated with VIMIZIM, including hypersensitivity reactions as well as life-threatening allergic reactions (anaphylaxis)\n<ul>\n<li>In the clinical trials, anaphylaxis occurred during VIMIZIM infusions and up to 3 hours after the infusion<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n                                                                                                                                                        <\/div>\n                                                                                                                            <div class=\"tab-content\" id=\"acf-block-63fc97d21fca6_1\" style=\"display:none;\">\n                                                                                                                                                                                                                        <h2>Long-term improvements in endurance with VIMIZIM at 120 weeks<sup>3<\/sup><\/h2>\n<h3>VIMIZIM showed sustained improvements in endurance over 120 weeks<sup>3<\/sup><\/h3>\n                                                                                                                                                                                                                                                                                                                                <div class=\"block split-content\">\n                                                    <div class=\"wrapper\">\n\t\t                                                <div class=\"inner-wrapper\">\n                                                                                                                            <div class=\"content-block\">\n                                                                                                                                                                                                                        <p>VIMIZIM\u00ae (elosulfase alfa)-pooled modified per-protocol (MPP) group achieved sustained improvement in the 6-minute walk test (6MWT) at 120 weeks when compared with a <a href=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/natural-history\/\">natural history<\/a> cohort.<sup>3<\/sup><\/p>\n<p>MOR-005 was an open-label, long-term extension study (N=173) that included a staggered transition to the weekly dosing regimen. In the absence of a placebo group, significance of the sustained improvements could not be evaluated directly.<sup>1,3<\/sup><\/p>\n                                                                                                                                                                                                            <\/div>\n                                                            \n                                                                                                                            <div class=\"content-block\">\n                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                                    \n                                                                            \n                                                                            \n                                                                            \n                                                                            <figure>\n                                                                                <div class=\"image\">\n                                                                                                                                                                            <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/wp-content\/uploads\/sites\/2\/2023\/01\/Long-term.png?v=0.46\" alt=\"\" \/>                                                                                                                                                                    <\/div>\n                                                                                                                                                            <\/figure>\n                                                                                                                                                                                                            <\/div>\n                                                                                                                    <\/div>\n                                                    <\/div>\n                                                <\/div>\n                                                                                                                                                                                                                                <h2 class=\"h3\">Adverse Reactions<sup>1<\/sup><\/h2>\n<ul>\n<li>In clinical trials, the most common adverse reactions (\u226510%) occurring during infusion included pyrexia, vomiting, headache, nausea, abdominal pain, chills, and fatigue<\/li>\n<li>Serious and severe reactions were associated with VIMIZIM, including hypersensitivity reactions as well as life-threatening allergic reactions (anaphylaxis)\n<ul>\n<li>In the clinical trials, anaphylaxis occurred during VIMIZIM infusions and up to 3 hours after the infusion<\/li>\n<\/ul>\n<\/li>\n<\/ul>\n                                                                                                                                                        <\/div>\n                                                                                    <\/div>\n                                    \t\t\t\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-63fc97d21fd09\" class=\"block boxed-content top-border boxed-content-white\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t\t<div class=\"box\">\n\t\t\t\t\t\t\t\t\n<div id=\"acf-block-63fc97d21fd38\" class=\"image-text-block\">\n        <div class=\"content-block\">\n                                                    <p><a class=\"button button-text button-arrow\" href=\"https:\/\/hcp.biomarin.com\/en-us\/vimizim\/pulmonary-function\/\" target=\"_self\">Review the improvements in pulmonary function that patients experienced with VIMIZIM<\/a><\/p>\n                        <\/div>\n<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-63fc97d220fdd\" class=\"block references\">\n    <div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t    \t\t\t    <h4>References:\n<\/h4>\n\t\t\t\t\t\t                <ol>\n                                                                                                                        <li><span>VIMIZIM [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2019. \n<\/span><\/li>\n                                                                                                                                                <li><span>Hendriksz CJ, Burton B, Fleming TR, <em>et al<\/em>. Efficacy and safety of enzyme replacement therapy with BMN 110 (elosulfase alfa) for Morquio A syndrome (mucopolysaccharidosis IVA): a phase 3 randomised placebo-controlled study. <em>J Inherit Metab Dis<\/em>. 2014;37(6):979-990.\n<\/span><\/li>\n                                                                                                                                                <li><span>Hendriksz CJ, Parini R, AlSayed MD, <em>et al<\/em>. Long-term endurance and safety of elosulfase alfa enzyme replacement therapy in patients with Morquio A syndrome. <em>Mol Genet Metab<\/em>. 2016;119(1-2):131-143.\n<\/span><\/li>\n                                                                                                                                                <li><span>Data on file. BioMarin Pharmaceutical, Inc. \n<\/span><\/li>\n                                                                                                                                                <li><span>Harmatz P, Mengel KE, Giugliani R, <em>et al<\/em>. The Morquio A Clinical Assessment Program: baseline results illustrating progressive, multisystemic clinical impairments in Morquio A subjects. <em>Mol Genet Metab<\/em>. 2013;109(1):54-61.\n<\/span><\/li>\n                                                                                                                                                <li><span>Hendriksz CJ, Giugliani R, Harmatz P, <em>et al<\/em>. Multi-domain impact of elosufase alfa in Morquio A syndrome in the pivotal phase III trial.<em> Mol Genet Metab<\/em>. 2015;114(2):178-185.\n<\/span><\/li>\n                                                            <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":2,"featured_media":0,"parent":0,"menu_order":0,"comment_status":"closed","ping_status":"closed","template":"","meta":{"_acf_changed":false,"inline_featured_image":false,"footnotes":""},"class_list":["post-2346","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v27.4 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>VIMIZIM\u00ae (elosulfase alfa) | Study Results<\/title>\n<meta name=\"description\" content=\"VIMIZIM - elosulfase alfa - improves endurance for Morquio A patients as measured by the six-minute walk test. Learn more. 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