{"id":4478,"date":"2025-09-24T21:01:24","date_gmt":"2025-09-24T21:01:24","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/?page_id=4478"},"modified":"2025-12-16T12:15:23","modified_gmt":"2025-12-16T12:15:23","slug":"pivotal-data","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/pivotal-data\/","title":{"rendered":"VOXZOGO Pivotal Data"},"content":{"rendered":"
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VOXZOGO is FDA approved from birth to increase linear growth in children with achondroplasia and open growth plates1<\/sup><\/h1>\n

Studied in children aged 4 months to <18 years1-4<\/sup><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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ACH,<\/strong> achondroplasia; EXT,<\/strong> extension; OS,<\/strong> observational study.<\/small><\/p>\n

*Duration of \u22656 months if aged \u22653 months at study entry; duration of \u22653 months if aged <3 months at study entry.3\u2028<\/sup><\/small>
\n\u2020<\/sup>Participants completed at least 3 or 6 months of an observational run-in growth study (either in CANOPY ACH-OS [Study 901] or CANOPY ACH-2I [Study 206]) to establish their baseline annualized growth velocity.4,5<\/sup><\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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Data continue to be collected in ongoing clinical trials.8<\/sup><\/span><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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Children with achondroplasia on VOXZOGO grew faster than without treatment1,9,10<\/sup><\/h2>\n

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Canopy ACH-3 (Study 301):<\/strong><\/span> Phase 3 pivotal trial in children aged 5 to 15 years (N=121)2,5<\/sup><\/p>\n

Mean (\u00b1SD) AGV at week 529<\/sup><\/h3>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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Children treated with VOXZOGO had an increase in height Z-score vs placebo that was maintained over baseline1,11<\/sup><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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97% of children on VOXZOGO (58\/60) in the phase 3<\/span>
\npivotal trial remained on treatment during the 1-year study.1<\/sup><\/span><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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Study Design<\/span><\/h4>\n