{"id":63,"date":"2022-11-28T20:54:46","date_gmt":"2022-11-28T20:54:46","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo2\/?page_id=63"},"modified":"2025-12-16T12:11:55","modified_gmt":"2025-12-16T12:11:55","slug":"efficacy-data","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/efficacy-data\/","title":{"rendered":"VOXZOGO Efficacy"},"content":{"rendered":"<div id=\"Linear-growth\" class=\"block wrapped-content bg-band bg-band-secondary block-zero-top\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-66446bef527a8\" class=\"block-wysiwyg\">\n            <h1>The impact of VOXZOGO continues to be studied<sup>1,2<\/sup><\/h1>\n<h3>Additional preliminary data continue to be collected<\/h3>\n<p>VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).<sup>1<\/sup><\/p>\n<p>The following information is provided to inform healthcare providers on the ongoing assessment and experience of patients in the clinical trial program.<\/p>\n<p>These data are not included in the US Prescribing Information and do not establish a clinical benefit or conclusions on efficacy. The analyses are preliminary and exploratory and should be interpreted cautiously.<\/p>\n<h3>Impact on final adult height has not been established and continues to be evaluated as studies are ongoing.<\/h3>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d598dcc33e1\" class=\"block wrapped-content block-zero-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d598dcc3404\" class=\"block-wysiwyg\">\n            <h2>After 3 years, height gain in VOXZOGO-treated and untreated children &lt;2 years old compared with expected gain in average-stature children<sup>3<\/sup><\/h2>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d598dcc3ff8\" class=\"block split-content equal-bias content-reverse content-align-top block-zero-bottom\" data-muted-autoplay=\"false\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t                                                                <div class=\"content-block first-block\">\n                                                                                                <h4 style=\"margin: 0\"><span style=\"color: #c6007e\">Data Limitations<\/span><\/h4>\n<ul>\n<li>VOXZOGO-treated, untreated, and average-stature children are from different studies; compare with caution as study design and methods may differ.<sup>3<\/sup><\/li>\n<li>Effect of VOXZOGO on final adult height not yet established (continually assessing).<sup>3<\/sup><\/li>\n<li>Preliminary results not in US label, are descriptive, reflect small samples, may result from chance.<sup>1,3<\/sup><\/li>\n<\/ul>\n<h4 style=\"margin: 0\"><span style=\"color: #c6007e\">Study Design<\/span><\/h4>\n<ul>\n<li><strong>CANOPY ACH-EXT (Study 208)<\/strong> secondary endpoints include percent height gain in VOXZOGO-treated children after 1, 2, and 3 years of treatment compared to the height gain in matched average-stature children. Similar analysis was conducted with matched untreated children with ACH.<sup>3,4\u2020<\/sup><\/li>\n<li>Cohort: Children with ACH aged 3 months to &lt;2 years at VOXZOGO initiation.<sup>3<\/sup><\/li>\n<li>Untreated children with ACH: From <strong>CLARITY, an external ACH natural history study<\/strong> (N=1341), multiple matched to each treated child at baseline by sex, age, height Z-score, and height.<sup>3,5<\/sup><\/li>\n<li>Average-stature children: Height gain derived from <strong>CDC reference data for average-stature children<\/strong>, age- and sex-matched to respective treated and untreated ACH groups.<sup>3<\/sup><\/li>\n<\/ul>\n                                                                                    <\/div>\n                    \n                                            <div class=\"content-block second-block\">\n                                                                                                                                                                                                                                                                                    \n                                                                                                                \n                                    \n                                    \n                                    <figure>\n                                        <div class=\"image image-responsive\">\n                                                                                            <div class=\"image-desktop\">\n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/Frame-680.png?v=0.161\" alt=\"Bar graph of additional and ongoing VOXZOGO efficacy results from open-label extension studies.\" \/>                                                <\/div>\n                                                <div class=\"image-mobile\">\n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/Frame-680.png?v=0.161\" alt=\"Bar graph of additional and ongoing VOXZOGO efficacy results from open-label extension studies.\" \/>                                                <\/div>\n                                                                                    <\/div>\n                                                                            <\/figure>\n                                                                                    <\/div>\n                                                \t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-6644782b1371f\" class=\"block wrapped-content block-tight-top\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-6644782b13747\" class=\"block-wysiwyg\">\n            <p><small><br \/>\n<strong>ACH,<\/strong> achondroplasia; <strong>CDC<\/strong>, Centers for Disease Control and Prevention; <strong>EXT<\/strong>, extension.<\/small><br \/>\n<small><br \/>\n*Adapted from Savarirayan R, et al. ACMG. Toronto, Canada. 2024. Poster P131.<sup>3<\/sup><br \/>\n<sup>\u2020<\/sup>Percent height gain vs matched average-stature children: After 1 year, ACH untreated (n=287) 66.73% vs ACH treated (n=32) 80.21%; after 2 years, ACH untreated (n=223) 66.02% vs ACH treated (n=25) 73.68%.<sup>3<\/sup><br \/>\n<\/small><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-6866d8e90208d\" class=\"block wrapped-content bg-band bg-band-secondary block-zero-bottom block-remove-bottom-padding\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-6866d8e9020b3\" class=\"block-wysiwyg\">\n            <h2>AGV by age in VOXZOGO-treated children was compared to a separate untreated control group<sup>6<\/sup><\/h2>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68eebb68bf053\" class=\"toggle-image bg-band block bg-band-secondary block-zero-top block-zero-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n            <div class=\"toggle-image-container\">\n                                    <div class=\"toggle-tab tab-one active\">\n                                                    <h2>Mean (\u00b1SD) female AGV by age<sup>6*<\/sup><\/h2>\n                        \n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/female-v5-01-1900x777.png\" alt=\"2 separate line graphs of additional and ongoing VOXZOGO efficacy results from open-label extension studies. The first shows females. The second shows males.\" \/>                        \n                                            <\/div>\n                \n                                    <div class=\"toggle-tab tab-two\">\n                                                    <h2>Mean (\u00b1SD) male AGV by age<sup>6*<\/sup><\/h2>\n                        \n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/male-v5-02-1900x777.png\" alt=\"2 separate line graphs of additional and ongoing VOXZOGO efficacy results from open-label extension studies. The first shows females. The second shows males.\" \/>                        \n                                            <\/div>\n                \n                \n                <div class=\"toggle-buttons\">\n                    <div class=\"slider\"><\/div>\n                    <div class=\"toggle option-one active\">\n                        Females                    <\/div>\n                    <div class=\"toggle option-two\">\n                        Males                    <\/div>\n                <\/div>\n\n                            <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d598dcc43e8\" class=\"block wrapped-content bg-band bg-band-secondary block-zero-top\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d598dcc441f\" class=\"block-wysiwyg\">\n            <h4 style=\"margin: 0\"><span style=\"color: #c6007e\">Data Limitations<\/span><\/h4>\n<ul>\n<li>VOXZOGO-treated and untreated groups are from different studies; compare with caution as study design and methods may differ.<sup>6<\/sup><\/li>\n<li>Effect of VOXZOGO on final adult height not yet established (continually assessing).<sup>6<\/sup><\/li>\n<li>Preliminary results not in the US label, reflect a cross-sectional mix of treatment durations, are descriptive, may result from chance.<sup>1,6<\/sup><\/li>\n<\/ul>\n<h4 style=\"margin: 0\"><span style=\"color: #c6007e\">Study Design<\/span><\/h4>\n<ul>\n<li><strong>CANOPY ACH-EXT (Study 302)<\/strong> primary endpoints include AGV by age and sex in VOXZOGO-treated children.<sup>4,6<\/sup><\/li>\n<li>Cohort: Full analysis set; age 5 to &lt;18 years (N=119).<sup>6<\/sup><\/li>\n<li>Mean (SD) treatment duration: 4.0 (0.8) years (min: 1.7 years, max: 6.2 years).<sup>6<\/sup><\/li>\n<li>AGV derived for height assessments 12 \u00b1 3 months apart and linked to a speci\ufb01c age integer considering the age at the midpoint of the 12-month interval.<sup>6<\/sup><\/li>\n<li><strong>External ACH natural history study (CLARITY):<\/strong> AGV of untreated children with ACH from CLARITY (age- and sex-matched to VOXZOGO-treated children) are plotted.<sup>6<\/sup><\/li>\n<\/ul>\n<p><small><br \/>\n<strong>ACH,<\/strong> achondroplasia; <strong>AGV,<\/strong> annualized growth velocity; <strong>EXT,<\/strong> extension; <strong>SD,<\/strong> standard deviation.<br \/>\n<\/small><small><br \/>\n*Adapted from Savarirayan R, et al. <em>Med<\/em>. 2025;6(5):100566.<sup>6<\/sup><br \/>\n<\/small><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d598dcc445c\" class=\"block wrapped-content block-zero-top\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d598dcc4480\" class=\"block-wysiwyg\">\n            <h2>Body proportionality was measured over 5 years in a subset of VOXZOGO-treated children<sup>6<\/sup><\/h2>\n<p><span style=\"color: #c6007e\"><strong>CANOPY ACH-3 (Study 301):<\/strong><\/span> After 1 year in the pivotal trial, the secondary endpoint of LS mean change from baseline in upper-to-lower body segment ratio was -0.02 in the placebo group (n=61) and -0.03 in the VOXZOGO group (n=58). The difference in LS mean change from baseline was -0.01 (95% CI: -0.05, 0.02; <em>P<\/em>=0.5).<sup>1,4,7<\/sup><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-690a72b90a24e\" class=\"block wrapped-content block-tight-top block-zero-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-690a72b90a26e\" class=\"block-wysiwyg\">\n            <h2 style=\"text-align: center\">Mean (\u00b1SE) upper-to-lower body segment ratio<sup>6*<\/sup><\/h2>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-6866d8e902135\" class=\"block wrapped-content block-zero-top block-zero-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<figure id=\"acf-block-66446bef5298f\">\n    <div class=\"image image-responsive image-align-left\">\n                    <div class=\"image-desktop\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/aFrame-171-1900x818.png\" alt=\"Line graph of additional and ongoing VOXZOGO efficacy results from open-label extension studies.\" \/>\t\t\t<\/div>\n\t\t\t<div class=\"image-mobile\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/aFrame-171-1900x818.png\" alt=\"Line graph of additional and ongoing VOXZOGO efficacy results from open-label extension studies.\" \/>\t\t\t<\/div>\n            <\/div>\n    <\/figure>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d598dcc445c\" class=\"block wrapped-content block-zero-top\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d598dcc4480\" class=\"block-wysiwyg\">\n            <h4 style=\"color: #c6007e;margin: 0\">5-Year Data Limitations<\/h4>\n<ul>\n<li>VOXZOGO-treated and untreated groups are from different trials; compare with caution as study design and methods may differ.<sup>6<\/sup><\/li>\n<li>Effect of VOXZOGO on final adult height and proportionality not yet established (continually assessing).<sup>6<\/sup><\/li>\n<li>Preliminary results not in the US label, are descriptive, reflect small samples, may result from chance.<sup>1,6<\/sup><\/li>\n<\/ul>\n<h4 style=\"color: #c6007e;margin: 0\">Study Design<\/h4>\n<ul>\n<li><strong>CANOPY ACH-EXT (Study 302)<\/strong> exploratory endpoints include body proportionality measured by upper-to-lower body segment ratio in VOXZOGO-treated children.<sup>4,6,8<\/sup><\/li>\n<li>Cohort: Children with assessments at age &lt;11 years (girls) or &lt;12 years (boys); all children were \u22655 years old at VOXZOGO initiation. Assessments beyond these ages are excluded from analysis.<sup>6\u2020<\/sup><\/li>\n<li><strong>CANOPY ACH-OS (Study 901) and CANOPY ACH-3 (Study 301) placebo arm:<\/strong> Upper-to-lower body segment ratio of untreated children with ACH (age-matched to VOXZOGO-treated children) are plotted.<sup>4,6,8<\/sup><\/li>\n<\/ul>\n<p><small><br \/>\n<strong>ACH,<\/strong> achondroplasia; <strong>CI,<\/strong> confidence interval; <strong>EXT,<\/strong> extension; <strong>LS,<\/strong> least squares; <strong>OS,<\/strong> observational= study; <strong>SE,<\/strong> standard error.<\/small><br \/>\n<small><br \/>\n*Adapted from Savarirayan R, et al. <em>Med<\/em>. 2025;6(5):100566.<sup>6<\/sup><br \/>\n<sup>\u2020<\/sup>In average-stature children, average upper-to-lower body segment ratio is 1.7 at birth and decreases to 1.0 at 10 years old. In ACH, the ratio never reaches 1.0 but still declines from birth up to ~age 10 years in girls and 11 years in boys.<sup>6<\/sup><br \/>\n<\/small><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d598dcc445c\" class=\"block wrapped-content bg-band bg-band-alternative\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d598dcc4480\" class=\"block-wysiwyg\">\n            <h2 style=\"text-align: center;margin: 0\">Backed by the longest clinical trial and postmarketing<br \/>\nexperience in achondroplasia.<sup>1,2<\/sup><\/h2>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d45bdb26d69\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d45bdb26da4\" class=\"block-wysiwyg\">\n            <h2>Health-related quality of life measures were evaluated in VOXZOGO-treated children at 1 and 3 years of treatment<sup>9,10<\/sup><\/h2>\n<p><span style=\"color: #c6007e\"><strong>CANOPY ACH-3 (Study 301):<\/strong><\/span> After 1 year, changes from baseline in HRQOL of children in the VOXZOGO and placebo groups were evaluated as exploratory endpoints using the QoLISSY questionnaire (developed for children with short stature), where higher scores represent higher HRQOL.<sup>4,7,9,10*<\/sup><\/p>\n<ul>\n<li><strong>Self-report version<\/strong> (for children aged 8-18 years) assessed 3 core domains (physical, social, emotional), averaged to create the total score, plus 2 additional domains (coping, beliefs)<sup>10<\/sup><\/li>\n<li><strong>Caregiver-report version<\/strong> (for children aged 4-18 years) assessed the same 3 core domains plus 5 additional domains (coping, beliefs, future, effects on parents, treatment); treatment domain not used<sup>10<\/sup><\/li>\n<\/ul>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-6866d8e9022a3\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-6866d8e9022c0\" class=\"block-wysiwyg\">\n            <h3>Self-reported QoLISSY scores: Change from baseline at 1 year of treatment (children 8 to &lt;18 years)<sup>9,10<\/sup><\/h3>\n    <\/div>\n\n<figure id=\"acf-block-66446bef5298f\">\n    <div class=\"image image-responsive image-align-left\">\n                    <div class=\"image-desktop\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/tchart-1900x269.png\" alt=\"Table indicating the domains of the self-reported QoLISSY questionnaire with children on VOXZOGO vs placebo.\" \/>\t\t\t<\/div>\n\t\t\t<div class=\"image-mobile\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/m-tchart-1900x353.png\" alt=\"Table indicating the domains of the self-reported QoLISSY questionnaire with children on VOXZOGO vs placebo.\" \/>\t\t\t<\/div>\n            <\/div>\n    <\/figure>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-6866d8e9022a3\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-6866d8e9022c0\" class=\"block-wysiwyg\">\n            <h3>Caregiver-reported QoLISSY scores: Change from baseline at 1 year of treatment (children 5 to &lt;18 years)<sup>9,10<\/sup><\/h3>\n    <\/div>\n\n<figure id=\"acf-block-66446bef5298f\">\n    <div class=\"image image-responsive image-align-left\">\n                    <div class=\"image-desktop\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/bchart-1900x269.png\" alt=\"Table indicating the domains of the caregiver-reported QoLISSY questionnaire with children on VOXZOGO vs placebo.\" \/>\t\t\t<\/div>\n\t\t\t<div class=\"image-mobile\">\n\t\t\t    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/m-bchart-1900x291.png\" alt=\"Table indicating the domains of the caregiver-reported QoLISSY questionnaire with children on VOXZOGO vs placebo.\" \/>\t\t\t<\/div>\n            <\/div>\n    <\/figure>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68eebb68bf85a\" class=\"toggle-image bg-band\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n            <div class=\"toggle-image-container\">\n                                    <div class=\"toggle-tab tab-one active\">\n                                                    <h2>Self-reported QoLISSY scores: Change from baseline at 3 years of treatment (children aged 8 to &lt;18 years)<sup>10,11*\u2020<\/sup><\/h2>\n                        \n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/qolissy1-1900x866.png\" alt=\"2 separate graphs of additional and ongoing VOXZOGO efficacy results from open-label extension studies. The first is self-reported. The second is caregiver-reported.\" \/>                        \n                                            <\/div>\n                \n                                    <div class=\"toggle-tab tab-two\">\n                                                    <h2>Caregiver-reported QoLISSY scores: Change from baseline at 3 years of treatment (children aged 5 to &lt;18 years)<sup>10,11*\u2020<\/sup><\/h2>\n                        \n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/11\/qolissy2-1900x866.png\" alt=\"2 separate graphs of additional and ongoing VOXZOGO efficacy results from open-label extension studies. The first is self-reported. The second is caregiver-reported.\" \/>                        \n                                            <\/div>\n                \n                \n                <div class=\"toggle-buttons\">\n                    <div class=\"slider\"><\/div>\n                    <div class=\"toggle option-one active\">\n                        Child                    <\/div>\n                    <div class=\"toggle option-two\">\n                        Caregiver                    <\/div>\n                <\/div>\n\n                            <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-664b524494758\" class=\"block wrapped-content block-zero-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-664b52449498d\" class=\"block-wysiwyg\">\n            <h4 style=\"margin: 0\">3-Year Data Limitations<\/h4>\n<ul>\n<li>No placebo control group, limits interpretation of VOXZOGO treatment effects.<sup>10<\/sup><\/li>\n<li>Effect of VOXZOGO on final adult height not yet established (continually assessing).<sup>10<\/sup><\/li>\n<li>Preliminary results not in US label, are descriptive, reflect small subgroup sizes.<sup>1,10<\/sup><\/li>\n<\/ul>\n<h4 style=\"margin: 0\">Study Design<\/h4>\n<ul>\n<li><strong>CANOPY-EXT (Study 302)<\/strong> secondary endpoints include change from baseline in HRQOL of VOXZOGO-treated children, assessed every 6 months by caregivers (QoLISSY caregiver-report version) and by children with ACH (QoLISSY self-report version).<sup>4,10<\/sup><\/li>\n<li>Cohort: VOXZOGO-treated children with ACH aged 5 to &lt;18 years (caregiver-reported) and aged 8 to &lt;18 years \u2028(self-reported).<sup>10<\/sup><\/li>\n<li>Mean (SE) change from baseline of the QoLISSY scores at 3 years of treatment shown for<sup>10<\/sup>:\n<ul>\n<li>All children<\/li>\n<li>Subgroup of children that grew the least (change in ACH height Z-score &lt;1)<\/li>\n<li>Subgroup of children that grew the most (change in ACH height Z-score \u22651)<\/li>\n<\/ul>\n<\/li>\n<li>Untreated ACH referenced Z-scores: Converted from standing height using age- and sex-matched reference data for untreated children with ACH from <strong>CLARITY, an external ACH natural history study<\/strong> (N=1,341).<sup>5,11<\/sup><\/li>\n<\/ul>\n<p><small><br \/>\n<strong>ACH,<\/strong> achondroplasia; <strong>EXT,<\/strong> extension; <strong>HRQOL,<\/strong> health-related quality of life; <strong>QoLISSY,<\/strong> Quality of Life of Short Stature Youth; <strong>SE,<\/strong> standard error.<\/small><br \/>\n<small><br \/>\n*Validation studies using the QoLISSY questionnaire for children with achondroplasia have been conducted.<sup>10,12<br \/>\n\u2020<\/sup>Adapted from Savarirayan R, et al. <em>Genet Med.<\/em> 2024;26(12):101274.<sup>10<\/sup><br \/>\n<\/small><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-664486df0c5d6\" class=\"block wrapped-content bg-band bg-band-secondary block-zero-bottom block-remove-bottom-padding\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d5ba6135e94\" class=\"block-wysiwyg\">\n            <h2>The VOXZOGO clinical trial program continues\u00a0to evaluate multiple endpoints<sup>1,2,7,13-15<\/sup><\/h2>\n<p>VOXZOGO is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).<sup>1<\/sup><\/p>\n<p><strong>As clinical trials are ongoing, the impact on final adult height has not been established and continues to be evaluated along with other pre-specified outcome measures.<sup>2,7,13-15<\/sup><\/strong><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d5ba6137433\" class=\"block split-content equal-bias bg-band bg-band-secondary block-zero-top block-zero-bottom block-remove-top-padding block-remove-bottom-padding\" data-muted-autoplay=\"false\">\n\t<div class=\"wrapper\">\n\t\t<div class=\"inner-wrapper\">\n\t\t                                                                <div class=\"content-block first-block\">\n                                                                                                                                                                                                                                                                                    \n                                                                                                                \n                                    \n                                    \n                                    <figure>\n                                        <div class=\"image image-responsive\">\n                                                                                            <div class=\"image-desktop\">\n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/10\/canopy1.png?v=0.161\" alt=\"\" \/>                                                <\/div>\n                                                <div class=\"image-mobile\">\n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/10\/canopy1.png?v=0.161\" alt=\"\" \/>                                                <\/div>\n                                                                                    <\/div>\n                                                                            <\/figure>\n                                                                                    <\/div>\n                    \n                                            <div class=\"content-block second-block\">\n                                                                                                                                                                                                                                                                                    \n                                                                                                                \n                                    \n                                    \n                                    <figure>\n                                        <div class=\"image image-responsive\">\n                                                                                            <div class=\"image-desktop\">\n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/10\/canopy2.png?v=0.161\" alt=\"\" \/>                                                <\/div>\n                                                <div class=\"image-mobile\">\n                                                    <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2025\/10\/canopy2.png?v=0.161\" alt=\"\" \/>                                                <\/div>\n                                                                                    <\/div>\n                                                                            <\/figure>\n                                                                                    <\/div>\n                                                \t\t<\/div>\n\t<\/div>\n<\/div>\n\n<div id=\"acf-block-68d598dcc445c\" class=\"block wrapped-content bg-band bg-band-secondary block-zero-top block-zero-bottom block-remove-top-padding\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d598dcc4480\" class=\"block-wysiwyg\">\n            <p><small>*Visit clinicaltrials.gov for the complete list of the trials&#8217; secondary outcome measures (NCT03197766, NCT03424018, NCT03583697, NCT03989947).<sup>7,13-15<\/sup><\/small><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d598dcc445c\" class=\"block wrapped-content bg-band bg-band-primary block-zero-top block-zero-bottom\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d598dcc4480\" class=\"block-wysiwyg\">\n            <h2 style=\"margin: 0px;text-align: center\">Learn about your role in ongoing treatment<\/h2>\n<p style=\"text-align: center\">Your patients and their families may need help maintaining their injection routine.<sup>1<\/sup><\/p>\n<p style=\"text-align: center\"><a class=\"button button-arrow\" href=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/administration-adherence\/\">Explore Tips for Adherence<\/a><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-63f4e940a1489\" class=\"block references block-zero-top block-zero-bottom\">\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>VOXZOGO [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.\n<\/span><\/li>\n                                                                                                                                                <li><span>ClinicalTrials.gov. Search: Achondroplasia, BioMarin Pharmaceutical. Accessed September 3, 2025. https:\/\/www.clinicaltrials.gov\/search?cond=Achondroplasia&amp;term=BioMarin%20Pharmaceutical&amp;viewType=Table\n<\/span><\/li>\n                                                                                                                                                <li><span>Savarirayan R, Wilcox WR, Harmatz P, et al. Persistence of growth-promoting effects in infants and toddlers with achondroplasia: results from a Phase 2 extension study with vosoritide. Annual Clinical Genetics Meeting (ACMG). Toronto, Canada. 2024. Poster. P131.\n<\/span><\/li>\n                                                                                                                                                <li><span>Data on file [1]. BioMarin Pharmaceutical Inc; 2025.\n<\/span><\/li>\n                                                                                                                                                <li><span>Hoover-Fong JE, Schulze KJ, Alade AY, et al. Growth in achondroplasia including stature, weight, weight-for-height and head circumference from CLARITY: achondroplasia natural history study-a multi-center retrospective cohort study of achondroplasia in the US. <em>Orphanet J Rare Dis.<\/em> 2021;16(1):522.\n<\/span><\/li>\n                                                                                                                                                <li><span>Savarirayan R, Irving M, Wilcox WR, et al. Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. <em>Med<\/em>. 2025;6(5):100566.\n<\/span><\/li>\n                                                                                                                                                <li><span>ClinicalTrials.gov. Identifier: NCT03197766. Accessed September 3, 2025. https:\/\/www.clinicaltrials.gov\/study\/NCT03197766\n<\/span><\/li>\n                                                                                                                                                <li><span>Savarirayan R, Irving M, Wilcox WR, et al. Sustained growth-promoting effects of vosoritide in children with achondroplasia from an ongoing phase 3 extension study. <em>Med.<\/em> 2025;6(5):100566. Supplementary Appendix.\n<\/span><\/li>\n                                                                                                                                                <li><span>Data on file [2]. BioMarin Pharmaceutical Inc; 2025.\n<\/span><\/li>\n                                                                                                                                                <li><span>Savarirayan R, Irving M, Wilcox WR, et al. Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life. <em>Genet Med.<\/em> 2024;26(12):101274.\n<\/span><\/li>\n                                                                                                                                                <li><span>Savarirayan R, Irving M, Wilcox WR, et al. Persistent growth-promoting effects of vosoritide in children with achondroplasia are accompanied by improvements in physical and social aspects of health-related quality of life. <em>Genet Med.<\/em> 2024;26(12):101274. Supplementary Appendix.\n<\/span><\/li>\n                                                                                                                                                <li><span>Bloemeke J, Sommer R, Witt S, et al. Cross-cultural selection and validation of instruments to assess patient-reported outcomes in children and adolescents with achondroplasia. <em>Qual Life Res<\/em>. 2019;28(9):2553-2563.\n<\/span><\/li>\n                                                                                                                                                <li><span>ClinicalTrials.gov. Identifier: NCT03424018. Accessed September 3, 2025. https:\/\/clinicaltrials.gov\/study\/NCT03424018\n<\/span><\/li>\n                                                                                                                                                <li><span>ClinicalTrials.gov. Identifier: NCT03583697. Accessed September 3, 2025. https:\/\/clinicaltrials.gov\/study\/NCT03583697\n<\/span><\/li>\n                                                                                                                                                <li><span>ClinicalTrials.gov. Identifier: NCT03989947. Accessed September 3, 2025. https:\/\/clinicaltrials.gov\/study\/NCT03989947\n<\/span><\/li>\n                                                            <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":4,"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-63","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>Additional Efficacy Data | VOXZOGO\u00ae (vosoritide) HCP<\/title>\n<meta name=\"description\" content=\"Discover additional efficacy results of VOXZOGO in the open-label extension studies.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link 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