{"id":6395,"date":"2026-08-04T06:56:00","date_gmt":"2026-08-04T06:56:00","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/?page_id=6395"},"modified":"2026-08-25T14:47:09","modified_gmt":"2026-08-25T14:47:09","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\">\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 style=\"text-align: left\">Additional preliminary data continue to be collected<\/h3>\n<p style=\"text-align: left\">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 style=\"text-align: left\">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 style=\"text-align: left\"><strong>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.<\/strong><\/p>\n<h3 style=\"text-align: left\">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-6a6b2867b812f\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-6a6b2867b815e\" class=\"block-wysiwyg\">\n            <h2>VOXZOGO was studied in children aged 4 months to &lt;5 years<sup>3<\/sup><\/h2>\n    <\/div>\n\n<figure id=\"acf-block-6a6b2867b858f\">\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\/2026\/08\/Frame-171-2.svg\" alt=\"image\" \/>\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\/2026\/07\/Frame-680.svg\" alt=\"image\" \/>\t\t\t<\/div>\n            <\/div>\n    <\/figure>\n\n<div id=\"acf-block-6a6b2867b85e1\" class=\"block-wysiwyg\">\n            <h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Study Design (Study 206)<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li>Cohort: Children with ACH aged 4.4 to 59.8 months old at treatment initiation with VOXZOGO (n=32 randomized; n=11 sentinels) or placebo (n=32 randomized)<sup>3<\/sup>\n<ul class=\"hcp-biomarin-list-pd\">\n<li>Completed an observational run-in growth study (Study 901 or Study 206) to establish baseline AGV<sup>3<\/sup>*<\/li>\n<\/ul>\n<\/li>\n<li>No formal sample-size calculations were made to power the study, predefined statistical analyses are descriptive (no control for type 1 error); results should be interpreted with caution<sup>3<\/sup><\/li>\n<\/ul>\n<h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Co-primary Endpoints (Study 206)<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\" style=\"text-align: left\">\n<li><strong>Safety and tolerability of VOXZOGO<\/strong><sup>3,7<\/sup><\/li>\n<li><strong>Change from baseline in height Z-score at 52 weeks:<\/strong> After 1 year, difference in LSM height Z-score change from baseline with VOXZOGO (n=32) relative to placebo (n=32) was 0.25 (95% CI: -0.02 to 0.53)<sup>3,7\u2020\u2021<\/sup><\/li>\n<\/ul>\n<p><small><strong>ACH,<\/strong> achondroplasia; <strong>AGV,<\/strong> annualized growth velocity; <strong>CDC,<\/strong> Centers for Disease Control and Prevention; <strong>CI,<\/strong> confidence interval; <strong>EXT,<\/strong> extension; <strong>LSM,<\/strong> least squares mean; <strong>OS,<\/strong> observational study.<\/small><\/p>\n<p><small>*Duration of \u22656 months if aged \u22653 months at study entry; duration of \u22653 months if aged &lt;3 months at study entry.<sup>4<\/sup><br \/>\n<sup>\u2020<\/sup>Average-stature referenced height Z-scores converted from standing height using age- and sex-matched CDC reference data for average-stature children; if &lt;24 months at baseline, body length took precedence over standing height.<sup>3,8<\/sup><br \/>\n<sup>\u2021<\/sup>Difference in LSM change from baseline: VOXZOGO &#8211; placebo. LSM (95% CI) and LSM differences estimated from ANCOVA (analysis of covariance) model, including treatment arm, sex, randomization age strata, and baseline age, height Z-score, and AGV.<sup>3,8<\/sup><\/small><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-68d598dcc445ca\" 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\">Supported by the longest ongoing clinical trial and postmarketing experience in achondroplasia\u2014explore the latest preliminary data.<sup>1,2<\/sup><\/h2>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-6a6c4e5be6807\" 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-6a6c4e5be6807_0\">AGV by Age\n<\/a><\/li>\n                                                                                                                                                            <li><a href=\"#acf-block-6a6c4e5be6807_1\">Height\n<\/a><\/li>\n                                                                                                                                                            <li><a href=\"#acf-block-6a6c4e5be6807_2\">Arm Span\n<\/a><\/li>\n                                                                                                                                                            <li><a href=\"#acf-block-6a6c4e5be6807_3\">Proportionality\n<\/a><\/li>\n                                                                                                                                                            <li><a href=\"#acf-block-6a6c4e5be6807_4\">Spine\n<\/a><\/li>\n                                                                                                                                                            <li><a href=\"#acf-block-6a6c4e5be6807_5\">Lower Leg\n<\/a><\/li>\n                                                                                    <\/ul>\n                                                                                                                                                <div class=\"box\">\n                                                                                            <div class=\"tab-content\" id=\"acf-block-6a6c4e5be6807_0\" style=\"display:none;\">\n                                                                                                                                                                                                                        <h2 style=\"text-align: left\">AGV by age in VOXZOGO-treated children was compared to separate untreated control groups<sup>9<\/sup>*<\/h2>\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\/2026\/08\/Female-1.svg\" alt=\"Line graph showing annual growth velocity (AGV) for females by age in achondroplasia with growth after puberty onset. Treated patients showed consistently higher AGV than untreated patients across ages\" \/>                                                            <\/div>\n                                                            <div class=\"image-mobile\">\n                                                                <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2026\/08\/Female-2.svg\" alt=\"Line graph showing annual growth velocity (AGV) for females by age in achondroplasia with growth after puberty onset. Treated patients showed consistently higher AGV than untreated patients across ages\" \/>                                                            <\/div>\n                                                                                                            <\/div>\n                                                                                                    <\/figure>\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\/2026\/08\/male.svg\" alt=\"Line graph showing annual growth velocity (AGV) for males by age in achondroplasia with growth after puberty onset. Treated patients showing consistently higher AGV than untreated patients across ages\" \/>                                                            <\/div>\n                                                            <div class=\"image-mobile\">\n                                                                <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2026\/08\/male-1.svg\" alt=\"Line graph showing annual growth velocity (AGV) for males by age in achondroplasia with growth after puberty onset. Treated patients showing consistently higher AGV than untreated patients across ages\" \/>                                                            <\/div>\n                                                                                                            <\/div>\n                                                                                                    <\/figure>\n                                                                                                                                                                                                                                <h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Data Limitations<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li><strong>Results are exploratory, are not in the US label, and should be interpreted with caution<\/strong><sup>1,9<\/sup><\/li>\n<li>VOXZOGO-treated and untreated groups are from different studies; compare with caution as study design and methods may differ<sup>9<\/sup><\/li>\n<li>Effect of VOXZOGO on final adult height not yet established (continually assessing); preliminary results reflect a cross-sectional mix of treatment durations, are descriptive, may result from chance<sup>9<\/sup><\/li>\n<\/ul>\n<h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Study Design<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li><strong>CANOPY ACH-EXT (Study 208 and Study 302)<\/strong> secondary and primary endpoints include AGV by age and sex in VOXZOGO-treated children<sup>5,9,12<\/sup><\/li>\n<li>Cohorts: Study 208, age &lt;2 years (N=33), mean treatment duration (SD) for ages 0 to &lt;0.5 years and 0.5 to &lt;2 years: 4.5 (0.8) years and 4.8 (1.5) years; Study 302, age 5 to &lt;18 years (N=119), mean treatment duration (SD): 4.0 (0.8) years<sup>9,12<\/sup><\/li>\n<li>AGV derived for height assessments 12 \u00b1 3.5 months apart and linked to a speci\ufb01c age integer considering the age at the midpoint of the 12-month interval<sup>10-12\u2021<\/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>9,12<\/sup><\/li>\n<\/ul>\n                                                                                                                                                                                                                                <p><small><strong>ACH,<\/strong> achondroplasia; <strong>AGV,<\/strong> annualized growth velocity; <strong>EXT,<\/strong> extension; <strong>SD,<\/strong> standard deviation.<\/small><\/p>\n<p><small>*AGV by age was assessed in Study 208 and Study 302, with an external ACH natural history study (CLARITY) used as separate untreated control groups.<sup>9,12<\/sup><br \/>\n<sup>\u2020<\/sup>In Study 302, the mean age of puberty onset was 10.7 years in females and 12.1 years in males; 26 females and 33 males were assessed for puberty onset as defined by the increase of any Tanner stage from 1 to 2 (ie, females: earliest increase in pubic hair or breast budding; males: earliest increase in pubic hair or testicular volume).<sup>13<\/sup><br \/>\n<sup>\u2021<\/sup>For ages &lt;3 years, AGV derived for height assessments 6 \u00b1 1.5 months apart.<sup>10<\/sup><\/small><\/p>\n                                                                                                                                                        <\/div>\n                                                                                                                            <div class=\"tab-content\" id=\"acf-block-6a6c4e5be6807_1\" style=\"display:none;\">\n                                                                                                                                                                                                                        <h2 style=\"text-align: left\">Height Z-score was measured over 7 years in VOXZOGO-treated children relative to untreated children<sup>14<\/sup><\/h2>\n                                                                                                                                                                                                                                <h3 style=\"text-align: center\">Change from baseline in height Z-Score (ACH referenced)<sup>14<\/sup>*<\/h3>\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\/2026\/08\/Mean-Upper-to-Lower-Body-Segment-ratio@4x-1.svg\" alt=\"Change from baseline in height Z-score was measured over 7 years in VOXZOGO-treated children relative to untreated children with achondroplasia\" \/>                                                            <\/div>\n                                                            <div class=\"image-mobile\">\n                                                                <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2026\/08\/Mean-Upper-to-Lower-Body-Segment-ratio@4x-1-1.svg\" alt=\"Change from baseline in height Z-score was measured over 7 years in VOXZOGO-treated children relative to untreated children with achondroplasia\" \/>                                                            <\/div>\n                                                                                                            <\/div>\n                                                                                                    <\/figure>\n                                                                                                                                                                                                                                <h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Data Limitations<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li><strong> Results are exploratory, are not in the US label, and should be interpreted with caution<\/strong><sup>1,14<\/sup><\/li>\n<li>VOXZOGO-treated and untreated groups are from different studies; compare with caution as study design and methods may differ<sup>14<\/sup><\/li>\n<li>Effect of VOXZOGO on final adult height not yet established (continually assessing); preliminary results are descriptive, may result from chance<sup>14<\/sup><\/li>\n<\/ul>\n<h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Study Design<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li><strong>CANOPY ACH-EXT (Study 208 &amp; Study 302)<\/strong> primary endpoints include height Z-scores in VOXZOGO-treated children, assessed every 6 months<sup>5,6,14,15<\/sup><\/li>\n<li>Cohorts: Study 208, age &lt;5 years at VOXZOGO initiation (N=73), mean treatment duration (SD): 5.0 (1.5) years; Study 302, age 5 to &lt;18 years at VOXZOGO initiation (N=119), mean treatment duration (SD): 5.2 (1.4) years<sup>14<\/sup><\/li>\n<li>Untreated ACH referenced height Z-scores: Derived from height\/body length using age- and sex-matched reference data of untreated children with ACH from an <strong>external ACH natural history study (CLARITY)<\/strong><sup>14,16<\/sup><\/li>\n<\/ul>\n                                                                                                                                                                                                                                <p><small><strong>ACH,<\/strong> achondroplasia; <strong>EXT,<\/strong> extension; <strong>SD,<\/strong> standard deviation; <strong>SE,<\/strong> standard error.<\/small><\/p>\n<p><small>*Adapted from Savarirayan R, et al. PES. San Francisco, CA, USA. 2026. Poster 32.<sup>14<\/sup><\/small><\/p>\n                                                                                                                                                        <\/div>\n                                                                                                                            <div class=\"tab-content\" id=\"acf-block-6a6c4e5be6807_2\" style=\"display:none;\">\n                                                                                                                                                                                                                        <h2 style=\"text-align: left\">Arm span Z-score was measured over 7 years in VOXZOGO-treated children relative to untreated children<sup>14<\/sup><\/h2>\n                                                                                                                                                                                                                                <h3 style=\"text-align: center\">Change from baseline in arm span Z-score (ACH referenced)<sup>14<\/sup>*<\/h3>\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\/2026\/08\/Mean-Upper-to-Lower-Body-Segment-ratio@4x-1-17.svg\" alt=\"Change from baseline in arm span Z-score was measured over 7 years in VOXZOGO-treated children relative to untreated children with achondroplasia\" \/>                                                            <\/div>\n                                                            <div class=\"image-mobile\">\n                                                                <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2026\/08\/Mean-Upper-to-Lower-Body-Segment-ratio@4x-1-16.svg\" alt=\"Change from baseline in arm span Z-score was measured over 7 years in VOXZOGO-treated children relative to untreated children with achondroplasia\" \/>                                                            <\/div>\n                                                                                                            <\/div>\n                                                                                                    <\/figure>\n                                                                                                                                                                                                                                <h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Data Limitations<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li><strong>Results are exploratory, are not in the US label, and should be interpreted with caution<\/strong><sup>1,14<\/sup><\/li>\n<li>VOXZOGO-treated and untreated children are from different studies; compare with caution as study design and methods may differ<sup>14<\/sup><\/li>\n<li>Effect of VOXZOGO on final adult height not yet established (continually assessing); preliminary results are descriptive, may result from chance<sup>14<\/sup><\/li>\n<\/ul>\n<h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Study Design<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li><strong>CANOPY ACH-EXT (Study 208 &amp; Study 302)<\/strong> secondary endpoints include body proportionality measures (arm span, arm span-to-height ratio) in VOXZOGO-treated children, assessed every year<sup>5,6,14,17<\/sup><\/li>\n<li>Cohorts: Study 208, age &lt;5 years at VOXZOGO initiation (N=73), mean treatment duration (SD): 5.0 (1.5) years; Study 302, age 5 to &lt;18 years at VOXZOGO initiation (N=119), mean treatment duration (SD): 5.2 (1.4) years<sup>14<\/sup><\/li>\n<li>Untreated ACH referenced arm span Z-scores: Derived from age- and sex-matched reference data of untreated children with ACH from an <strong>external ACH natural history study<\/strong><sup>14,18<\/sup><\/li>\n<\/ul>\n                                                                                                                                                                                                                                <p><small><strong>ACH,<\/strong> achondroplasia; <strong>EXT,<\/strong> extension; <strong>SD,<\/strong> standard deviation; <strong>SE,<\/strong> standard error.<\/small><\/p>\n<p><small>*Adapted from Savarirayan R, et al. PES. San Francisco, CA, USA. 2026. Poster 32.<sup>14<\/sup><\/small><\/p>\n                                                                                                                                                        <\/div>\n                                                                                                                            <div class=\"tab-content\" id=\"acf-block-6a6c4e5be6807_3\" style=\"display:none;\">\n                                                                                                                                                                                                                                                                                                                                                                                                                                                        <h2 style=\"text-align: left\">Upper-to-lower body segment ratio was measured over 5 years in a subset of VOXZOGO-treated children<sup>12<\/sup><\/h2>\n<p style=\"text-align: left\"><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>1,5,19<\/sup><\/p>\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\/2026\/08\/Frame-172.svg\" alt=\"Line graph showing upper-to-lower body segment ratio over time in achondroplasia, with VOXOGO-treated patients vs untreated patients\" \/>                                                            <\/div>\n                                                            <div class=\"image-mobile\">\n                                                                <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2026\/08\/Frame-172-1.svg\" alt=\"Line graph showing upper-to-lower body segment ratio over time in achondroplasia, with VOXOGO-treated patients vs untreated patientsLine graph showing upper-to-lower body segment ratio over time in achondroplasia, with VOXOGO-treated patients vs untreated patients\" \/>                                                            <\/div>\n                                                                                                            <\/div>\n                                                                                                    <\/figure>\n                                                                                                                                                                                                                                <h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">5-Year Data Limitations<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li><strong>Results are exploratory, are not in the US label, and should be interpreted with caution<\/strong><sup>1,12<\/sup><\/li>\n<li>VOXZOGO-treated and untreated groups are from different trials; compare with caution as study design and methods may differ<sup>12<\/sup><\/li>\n<li>Effect of VOXZOGO on final adult height and proportionality not yet established (continually assessing); preliminary results are descriptive, reflect small samples, may result from chance<sup>12<\/sup><\/li>\n<\/ul>\n<h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Study Design<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\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>5,12,15<\/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>12,15\u2020<\/sup><\/li>\n<li><strong>CANOPY ACH-OS (Study 901)<\/strong> and <strong>CANOPY ACH-3 (Study 301)<\/strong> placebo arm: Upper-to-lower body segment ratio of untreated children with ACH (age-matched to VOXZOGO-treated children) are plotted<sup>5,12,15<\/sup><\/li>\n<\/ul>\n                                                                                                                                                                                                                                <p><small><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><\/p>\n<p><small>*Adapted from Savarirayan R, et al. <em>Med.<\/em> 2025;6(5):100566.<sup>12<\/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>12<\/sup><\/small><\/p>\n                                                                                                                                                        <\/div>\n                                                                                                                            <div class=\"tab-content\" id=\"acf-block-6a6c4e5be6807_4\" style=\"display:none;\">\n                                                                                                                                                                                                                        <h2 style=\"text-align: left\">At 1 year, change from baseline in spinal canal measures were evaluated in VOXZOGO-treated children &lt;5 years old vs placebo<sup>20<\/sup><\/h2>\n                                                                                                                                                                                                                                <h3 style=\"text-align: center\">Difference in change from baseline in interpedicular distance and spinal canal sagittal width at all lumbar vertebrae with VOXZOGO vs placebo<sup>20<\/sup>*<\/h3>\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\/2026\/08\/Mean-Upper-to-Lower-Body-Segment-ratio@4x-1-6.svg\" alt=\"Difference in change from baseline in interpedicular distance and spinal canal sagittal width at all lumbar vertebrae with VOXZOGO-treated children under 5 years old vs placebo\" \/>                                                            <\/div>\n                                                            <div class=\"image-mobile\">\n                                                                <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2026\/08\/Mean-Upper-to-Lower-Body-Segment-ratio@4x-1-7.svg\" alt=\"Difference in change from baseline in interpedicular distance and spinal canal sagittal width at all lumbar vertebrae with VOXZOGO-treated children under 5 years old vs placebo\" \/>                                                            <\/div>\n                                                                                                            <\/div>\n                                                                                                    <\/figure>\n                                                                                                                                                                                                                                <h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Data Limitations<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li><strong>Results are exploratory, are not in the US label, and should be interpreted with caution<\/strong><sup>1,20<\/sup><\/li>\n<li>Effect of VOXZOGO on final adult height not yet established (continually assessing); preliminary results are descriptive, reflect small samples, may result from chance<sup>20<\/sup><\/li>\n<\/ul>\n<h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Study Design<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li><strong>CANOPY ACH-2I (Study 206)<\/strong> secondary endpoints include change from baseline in lumbar spine measures (IPD, spinal canal sagittal width) at 52 weeks in children treated with VOXZOGO vs placebo<sup>5,7,20<\/sup><\/li>\n<li>Cohort: Individuals with ACH aged 4 months to &lt;5 years at initiation of VOXZOGO (n=40) or placebo (n=27)<sup>20<\/sup><\/li>\n<li>Spinal canal morphology: Assessed with A\/P and lateral X-rays at baseline and 52 weeks with standardized image acquisition; IPD and spinal canal sagittal width for L1-5 were defined by independent radiologists<sup>20<\/sup><\/li>\n<\/ul>\n                                                                                                                                                                                                                                <p><small><strong>A,<\/strong> anterior (front); <strong>ACH<\/strong>, achondroplasia; <strong>AGV,<\/strong> annualized growth velocity; <strong>CI,<\/strong> confidence interval; <strong>IPD,<\/strong> interpedicular distance; <strong>LSM,<\/strong> least squares mean; <strong>L1-5,<\/strong> lumbar vertebra 1-5; <strong>P,<\/strong> posterior (back).<\/small><\/p>\n<p><small>*Difference in LSM change from baseline was calculated as VOXZOGO minus placebo. LSM differences (95% CI) between treatment groups estimated from ANCOVA (analysis of covariance) model, which included treatment arm, sex, randomization age strata, and baseline age, height Z-score, AGV, and morphology measures.<sup>20<\/sup><\/small><\/p>\n                                                                                                                                                        <\/div>\n                                                                                                                            <div class=\"tab-content\" id=\"acf-block-6a6c4e5be6807_5\" style=\"display:none;\">\n                                                                                                                                                                                                                        <h2 style=\"text-align: center\">At 1 year, change from baseline in lower leg morphology measures were evaluated in VOXZOGO-treated children vs placebo<sup>21<\/sup><\/h2>\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\/2026\/08\/Frame-927.svg\" alt=\"At 1 year, change from baseline in lower leg morphology measures were evaluated in VOXZOGO-treated children vs placebo\" \/>                                                            <\/div>\n                                                            <div class=\"image-mobile\">\n                                                                <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2026\/08\/Frame-902-2.svg\" alt=\"At 1 year, change from baseline in lower leg morphology measures were evaluated in VOXZOGO-treated children vs placebo\" \/>                                                            <\/div>\n                                                                                                            <\/div>\n                                                                                                    <\/figure>\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\/2026\/08\/Frame-901.svg\" alt=\"At 1 year, change from baseline in lower leg morphology measures were evaluated in VOXZOGO-treated children vs placebo\" \/>                                                            <\/div>\n                                                            <div class=\"image-mobile\">\n                                                                <img decoding=\"async\" class=\"\" src=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2026\/08\/Frame-880.svg\" alt=\"At 1 year, change from baseline in lower leg morphology measures were evaluated in VOXZOGO-treated children vs placebo\" \/>                                                            <\/div>\n                                                                                                            <\/div>\n                                                                                                    <\/figure>\n                                                                                                                                                                                                                                <h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Data Limitations<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li><strong>Results are exploratory, are not in the US label, and should be interpreted with caution<\/strong><sup>1,21<\/sup><\/li>\n<li>Effect of VOXZOGO on final adult height not yet established (continually assessing); preliminary results are descriptive, reflect small samples, may result from chance<sup>21<\/sup><\/li>\n<\/ul>\n<h4 style=\"margin: 0px;text-align: left\"><span style=\"color: #c6007e\">Study Design<\/span><\/h4>\n<ul class=\"hcp-biomarin-list-pd\">\n<li><strong>CANOPY ACH-2I (Study 206) <\/strong>and<strong> CANOPY ACH-3 (Study 301)<\/strong> secondary endpoints include change from baseline in lower leg bone morphology measures (tibial bowing angle, F\/T ratio) at 52 weeks in children treated with VOXZOGO vs placebo<sup>4,7,21<\/sup><\/li>\n<li>Cohort:\n<ul>\n<li><strong>Study 206:<\/strong> Children with ACH aged 0 to &lt;5 years at initiation of VOXZOGO (n=40) or placebo (n=27)<sup>21<\/sup><\/li>\n<li><strong>Study 301:<\/strong> Children with ACH aged \u22655 to &lt;15 years at initiation of VOXZOGO (n=57) or placebo (n=59)<sup>21<\/sup><\/li>\n<\/ul>\n<\/li>\n<li>Overall tibial bowing angle and F\/T ratio (left and right legs combined): Assessed with A\/P lower limb X-rays at baseline and 52 weeks with standardized image acquisition and were defined by independent radiologists<sup>21<\/sup><\/li>\n<\/ul>\n                                                                                                                                                                                                                                <p><small><strong>ACH,<\/strong> achondroplasia; <strong>A\/P,<\/strong> anterior\/posterior; <strong>CI,<\/strong> confidence interval; <strong>F\/T,<\/strong> fibula\/tibia; <strong>LSM,<\/strong> least squares mean.\u00a0<\/small><\/p>\n<p><small>*Difference in LSM change from baseline was calculated as VOXZOGO &#8211; placebo. LSM differences (95% CI) between treatment groups estimated from repeated measures ANCOVA (analysis of covariance) model, which included treatment, sex, Tanner stage (Study 301 only), age stratum (Study 206 only), and baseline age, annualized growth velocity, height Z-score, and bone morphology measure.<sup>21<\/sup><\/small><\/p>\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-664486df0c5d6\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d5ba6135e94\" class=\"block-wysiwyg\">\n            <h2 style=\"text-align: left\">The VOXZOGO clinical trial program continues\u00a0to evaluate multiple endpoints<sup>1,2,6,7,17,19<\/sup><\/h2>\n<p style=\"text-align: left\">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 style=\"text-align: left\"><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.<\/strong><sup>2,6,7,17,19<\/sup><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-69dda7cd271e4\" class=\"block wrapped-content\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<figure id=\"acf-block-69dda7cd2741e\">\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\/2026\/08\/Clinical_trial_table_Desktop.svg\" alt=\"CANOPY ACH-2I (Study 206)\/CANOPY ACH-EXT (Study 208) Secondary outcome measures include: [body proportionality,] functional and health-related quality of life measures, spinal alignment, and skull morphology\" \/>\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\/2026\/08\/Clinical_trial_table_Mobile.svg\" alt=\"CANOPY ACH-2I (Study 206)\/CANOPY ACH-EXT (Study 208) Secondary outcome measures include: [body proportionality,] functional and health-related quality of life measures, spinal alignment, and skull morphology\" \/>\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\">\n    <div class=\"wrapper\">\n        <div class=\"inner-wrapper\">\n                \n<div id=\"acf-block-68d598dcc4480\" class=\"block-wysiwyg\">\n            <p><small><strong>ACH,<\/strong> achondroplasia; <strong>EXT,<\/strong> extension; <strong>HRQOL,<\/strong> health-related quality of life; <strong>ITQOL,<\/strong> Infant Toddler Quality of Life; <strong>QoLISSY,<\/strong> Quality of Life in Short Stature Youth; <strong>ULBR,<\/strong> upper-to-lower body segment ratio; <strong>WeeFIM-II,<\/strong> Pediatric Functional Independence Measure, 2nd version.<\/small><\/p>\n<p><small>*Visit clinicaltrials.gov for the complete list of the trials&#8217; secondary outcome measures (NCT03197766, NCT03424018, NCT03583697, NCT03989947).<sup>6,7,17,19<\/sup><\/small><\/p>\n    <\/div>\n        <\/div>\n    <\/div>\n<\/div>\n\n<div id=\"acf-block-6a71bec5705c5\" class=\"hero hero-split\">\n\t<div class=\"split-hero-text-area\">\n\t\t<div class=\"split-hero-text-content\">\n\t\t    \t\t\t                                        <h2 style=\"text-align: center;color: #ffffff\">&#8220;I&#8217;m glad I was able to stay on VOXZOGO as long as I did.&#8221;<\/h2>\n<p style=\"text-align: center\">\u2013 Vivan, 18 years old and completed treatment with VOXZOGO<\/p>\n                        \t\t<\/div>\n\t<\/div>\n\t<div class=\"split-hero-image\" style=\"background-image: url(https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-content\/uploads\/sites\/4\/2026\/08\/Mobile-Video-Thumbnail-1.png?v=0.171);\">\n\t\t\t<\/div>\n<\/div>\n\n<div id=\"tips\" class=\"block wrapped-content bg-band bg-band-primary 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=\"text-align: center\">An established safety profile<sup>1,12,22<\/sup><\/h2>\n<p style=\"text-align: center\">VOXZOGO has been carefully evaluated in infants and children with achondroplasia.<sup>1,12,22<\/sup><\/p>\n<p style=\"text-align: center\"><a class=\"button button-arrow\" href=\"\/en-us\/voxzogo\/administration-adherence\/\">Learn About Safety<\/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&nbsp;\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 March 17, 2026. https:\/\/clinicaltrials.gov\/search?cond=Achondroplasia&#038;term=BioMarin%20Pharmaceutical&#038;viewType=Table\n<\/span><\/li>\n                                                                                                                                                <li><span>Savarirayan R, Wilcox WR, Harmatz P, et al. Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial. <em>Lancet Child Adolesc Health<\/em>. 2024;8(1):40-50.\n<\/span><\/li>\n                                                                                                                                                <li><span>Savarirayan R, Tofts L, Irving M, et al. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. <em>Lancet<\/em>. 2020;396(10252):684-692. Supplementary Appendix.\n<\/span><\/li>\n                                                                                                                                                <li><span>Data on file [1]. BioMarin Pharmaceutical Inc; 2025.\n<\/span><\/li>\n                                                                                                                                                <li><span>ClinicalTrials.gov. Identifier: NCT03989947. Accessed March 17, 2026. https:\/\/clinicaltrials.gov\/study\/NCT03989947\n<\/span><\/li>\n                                                                                                                                                <li><span>ClinicalTrials.gov. Identifier: NCT03583697. Accessed March 17, 2026. https:\/\/clinicaltrials.gov\/study\/NCT03583697\n<\/span><\/li>\n                                                                                                                                                <li><span>Savarirayan R, Wilcox WR, Harmatz P, et al. Vosoritide therapy in children with achondroplasia aged 3-59 months: a multinational, randomised, double-blind, placebo-controlled, phase 2 trial. <em>Lancet Child Adolesc Health<\/em>. 2024;8(1):40-50. Supplementary Appendix.\n<\/span><\/li>\n                                                                                                                                                <li><span>Savarirayan R, Bacino C, Carroll RS, et al. Early start, maximum impact: long-term trial data supporting cumulative clinical benefit in children who initiated vosoritide &lt;2 years old. ACMG Annual Clinical Genetics Meeting. Baltimore, MD, USA. 2026. Poster P174.\n\n<\/span><\/li>\n                                                                                                                                                <li><span>Data on file [2]. BioMarin Pharmaceutical Inc; 2026.\n<\/span><\/li>\n                                                                                                                                                <li><span>Data on file [3]. BioMarin Pharmaceutical Inc; 2026.\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>Hoover-Fong JE, Labed AH, Lyon N, et al. Continued growth after puberty in participants with achondroplasia treated with vosoritide in a phase 3 long-term extension trial. American Society for Bone and Mineral Research (ASBMR) Annual Meeting. Seattle, WA, USA. 2025. Poster Sat-555.\n<\/span><\/li>\n                                                                                                                                                <li><span>Savarirayan R, Bacino CA, Hoover-Fong JE, et al. Effect of long-term vosoritide treatment on growth in children with achondroplasia in open-label, multicenter clinical trials. PES. San Francisco, CA, USA. 2026. Poster 32.\n<\/span><\/li>\n                                                                                                                                                <li><span>Data on file [4]. BioMarin Pharmaceutical Inc; 2022.\n<\/span><\/li>\n                                                                                                                                                <li><span>Hoover-Fong JE, Alade AY, Hashmi SS, et al. Achondroplasia Natural History Study (CLARITY): a multicenter retrospective cohort study of achondroplasia in the United States. <em>Genet Med<\/em>. 2021;23(8):1498-1505.\n<\/span><\/li>\n                                                                                                                                                <li><span>ClinicalTrials.gov. Identifier: NCT03424018. Accessed March 17, 2026. https:\/\/clinicaltrials.gov\/study\/NCT03424018\n<\/span><\/li>\n                                                                                                                                                <li><span><span style=\"font-size: 14px\">Merker A, Neumeyer L, Hertel NT, et al. Development of body proportions in achondroplasia: sitting height, leg length, arm span, and foot length. <em>Am J Med Genet A<\/em>. 2018;176(9):1819-1829.<\/span>\n<\/span><\/li>\n                                                                                                                                                <li><span>ClinicalTrials.gov. Identifier: NCT03197766. Accessed March 17, 2026. https:\/\/clinicaltrials.gov\/study\/NCT03197766\n<\/span><\/li>\n                                                                                                                                                <li><span>Irving M, Savarirayan R, Hoover-Fong JE, et al. Effect of vosoritide on spine morphology in children\u00a0with achondroplasia: 1-year results from a randomized phase 2 study. <em>J Endocr Soc<\/em>. 2026;10(3):bvag008.\n<\/span><\/li>\n                                                                                                                                                <li><span><span style=\"font-size: 14px\">White KK, Irving M, Mukherjee S, et al. Effect of vosoritide on genu varum in children with achondroplasia after 1 year in randomized placebo-controlled trials. <em>J Endocr Soc<\/em>. 2026;10(3):bvag024.<\/span>\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                                                            <\/ol>\n\t\t\t\t\t<\/div>\n\t<\/div>\n<\/div>","protected":false},"excerpt":{"rendered":"","protected":false},"author":18,"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-6395","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"<!-- This site is optimized with the Yoast SEO plugin v28.2 - https:\/\/yoast.com\/product\/yoast-seo-wordpress\/ -->\n<title>Ongoing Treatment Efficacy | VOXZOGO\u00ae Additional Efficacy Data<\/title>\n<meta name=\"description\" content=\"Explore additional treatment outcomes for children with achondroplasia taking VOXZOGO\u00ae (vosoritide), including data on growth velocity by age, spinal morphology, and more.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, 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