{"id":65,"date":"2022-11-28T20:55:16","date_gmt":"2022-11-28T20:55:16","guid":{"rendered":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo2\/?page_id=65"},"modified":"2026-06-02T21:30:49","modified_gmt":"2026-06-02T21:30:49","slug":"safety-data","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/safety-data\/","title":{"rendered":"VOXZOGO Safety"},"content":{"rendered":"
\n
\n
\n \n
\n

The safety profile of VOXZOGO has been carefully evaluated1-3<\/sup><\/h1>\n

Established safety profile in children 5 to 15 years1,2<\/sup><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
\n\t
\n\t\t
\n\t\t
\n

Over 1 year in the phase 3 pivotal trial (Study 301),<\/strong> adverse reactions that occurred in \u22655% of patients aged 5 to 15 years treated with VOXZOGO and at a percentage greater than placebo are provided in the table.1<\/sup><\/p>\n

In the long-term open-label extension study (Study 302),<\/strong> adverse reactions reported for up to 6 years of treatment are consistent with those seen in the 1-year pivotal trial.2<\/sup><\/p>\n <\/div>\n \n

\n \n \n \n \n
\n
\n
\n \"\" <\/div>\n
\n \"\" <\/div>\n <\/div>\n <\/figure>\n <\/div>\n \t\t<\/div>\n\t<\/div>\n<\/div>\n\n
\n
\n
\n \n
\n
\n
\n\t\t\t \"\"\t\t\t<\/div>\n\t\t\t
\n\t\t\t \"\"\t\t\t<\/div>\n <\/div>\n <\/figure>\n <\/div>\n <\/div>\n<\/div>\n\n
\n
\n
\n \n
\n

*Includes adverse reactions occurring more frequently in the VOXZOGO arm and with a risk difference of \u22655% (ie, difference of >2 subjects) between treatment arms.1
\n<\/sup>\u2020<\/sup>Injection site reactions occurring more frequently in VOXZOGO-treated patients than placebo.1
\n<\/sup>\u2021<\/sup>Includes the preferred terms: gastroenteritis and gastroenteritis, viral.1
\n<\/sup>\u00a7<\/sup>Includes the preferred terms: dizziness, presyncope, procedural dizziness, and vertigo.1
\n<\/sup>||<\/sup>Includes the preferred terms: fatigue, lethargy, and malaise.1<\/sup><\/small><\/p>\n

 <\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

\n
\n
\n \n
\n

Consistent safety profile in children aged <5 years1,3<\/sup><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
\n\t
\n\t\t
\n\t\t
\n

Over 1 year in the phase 2 trial (Study 206),<\/strong> the most common adverse reactions (>10%) reported in patients <5 years were injection site reactions (86%) and rash (28%).1<\/sup><\/p>\n

In the long-term open-label extension study (Study 208),<\/strong> adverse reactions reported for up to 4 years of treatment are consistent with those seen in the 1-year trial (see table).3<\/sup><\/p>\n <\/div>\n \n

\n \n \n \n \n
\n
\n
\n \"Table <\/div>\n
\n \"Table <\/div>\n <\/div>\n <\/figure>\n <\/div>\n \t\t<\/div>\n\t<\/div>\n<\/div>\n\n
\n
\n
\n \n
\n
\n
\n\t\t\t \"\"\t\t\t<\/div>\n\t\t\t
\n\t\t\t \"\"\t\t\t<\/div>\n <\/div>\n <\/figure>\n <\/div>\n <\/div>\n<\/div>\n\n
\n
\n
\n \n
\n

ADL,<\/strong> activities of daily living; AE,<\/strong> adverse event; CTCAE,<\/strong> Common Terminology Criteria for Adverse Events; SAE,<\/strong> serious adverse event.<\/small>
\n
\n*Data combined from children aged <2 years and 2 to <5 years at the start of treatment in clinical trials.3<\/sup>
\n\u2020<\/sup>CTCAE (v5.0) grades refers to the severity of the AE: Grade 1 = mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated; Grade 2 = moderate; minimal, local, or non-invasive intervention indicated; limiting age-appropriate instrumental ADL; Grade 3 = severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL; Grade 4 = life-threatening consequences; urgent intervention indicated; Grade 5 = death related to AE.4<\/sup>
\n\u2021<\/sup>No CTCAE Grade \u22653 hypersensitivity or anaphylaxis reported.3<\/sup>
\n\u00a7<\/sup>No CTCAE Grade \u22652 injection site reactions were reported.3<\/sup>
\n<\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

\n
\n
\n \n
\n

Consistent safety profile in children aged <2 years3<\/sup><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
\n\t
\n\t\t
\n\t\t
\n

Over 4 years in the in the long-term open-label extension study (Study 208),<\/strong> adverse reactions reported in children <2 years old at treatment initiation (see table) were consistent with those seen in children aged 2 to <5 years old.3<\/sup><\/p>\n <\/div>\n \n

\n \n \n \n \n
\n
\n
\n \"Table <\/div>\n
\n \"Table <\/div>\n <\/div>\n <\/figure>\n <\/div>\n \t\t<\/div>\n\t<\/div>\n<\/div>\n\n
\n
\n
\n \n
\n

ADL,<\/strong> activities of daily living; AE,<\/strong> adverse event; CTCAE,<\/strong> Common Terminology Criteria for Adverse Events; SAE,<\/strong> serious adverse event.<\/small><\/p>\n

*CTCAE (v5.0) grades refers to the severity of the AE: Grade 1 = mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated; Grade 2 = moderate; minimal, local, or non-invasive intervention indicated; limiting age-appropriate instrumental ADL; Grade 3 = severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self-care ADL; Grade 4 = life-threatening consequences; urgent intervention indicated; Grade 5 = death related to AE.4<\/sup>
\n\u2020<\/sup>No CTCAE Grade \u22653 hypersensitivity or anaphylaxis reported.3<\/sup>
\n\u2021<\/sup>No CTCAE Grade \u22652 injection site reactions were reported.3<\/sup><\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

\n
\n
\n \n
\n

With over 10 years of clinical trial experience in achondroplasia, VOXZOGO has been prescribed to 5,000+ infants and children with achondroplasia worldwide.1,5,6<\/sup><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
\n
\n
\n \n
\n

Select adverse reactions from the phase 3 pivotal trial1<\/sup><\/h2>\n

Injection site reactions (ISRs) of children aged 5 to 15 years1<\/sup><\/span><\/h3>\n <\/div>\n\n
\n
\n
\n\t\t\t \"\"\t\t\t<\/div>\n\t\t\t
\n\t\t\t \"\"\t\t\t<\/div>\n <\/div>\n

*One patient discontinued treatment due to pain, and one discontinued treatment due to anxiety with injections.1<\/sup><\/small><\/p>\n<\/figcaption>\n <\/figure>\n <\/div>\n <\/div>\n<\/div>\n\n

\n
\n
\n \n
\n

Transient decreases in blood pressure of children aged 5 to 15 years1,7\u2020<\/span><\/sup><\/span><\/h3>\n <\/div>\n\n
\n
\n
\n\t\t\t \"\"\t\t\t<\/div>\n\t\t\t
\n\t\t\t \"\"\t\t\t<\/div>\n <\/div>\n

VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients; to reduce the risk of a decrease in blood pressure and associated symptoms (dizziness, feeling tired, or nausea), patients should be well fed and hydrated in the hour before receiving VOXZOGO.1<\/sup><\/p>\n

\u2020<\/sup>Decreased blood pressure includes blood pressure decrease and hypotension; changes were identified mainly during periods of frequent vital sign monitoring at clinical visits after dosing over 1 year of treatment.1<\/sup>
\n\u2021<\/sup>3% (2\/60) of patients on VOXZOGO each had 1 symptomatic decreased blood pressure event with vomiting and\/or dizziness vs 0% of patients on placebo.1<\/sup>
\n\u00a7<\/sup>Post-dose decreases in systolic blood pressure <70 mm Hg (+2x age): 23% (14\/60) patients on VOXZOGO vs 25% (15\/61) patients on placebo; post-dose decreases in diastolic blood pressure <40 mm Hg: 17% (10\/60) patients on VOXZOGO vs 10% (6\/61) patients on placebo.7<\/sup>
\n<\/small><\/p>\n<\/figcaption>\n <\/figure>\n <\/div>\n <\/div>\n<\/div>\n\n

\n
\n\t\t
\n\t\t\t
\n\t\t\t\t
<\/div>\n\t\t\t\t
\n\t\t\t\t \t\t\t\t\t
\n
\n\t\t\t\t\t \t\t\t\t\t \t\t\t\t\t\t

Hear from a pediatric endocrinologist\n<\/h2>\n\t\t\t\t\t\t \t\t\t\t\t\t \t\t\t\t\t\t

Amrit Bhangoo, MD, explains why he prescribes VOXZOGO as early as birth.1<\/sup><\/p>\n\t\t\t\t\t\t \t\t\t\t\t <\/div>\n\t\t\t\t\t

\n

Watch Now<\/a><\/p>\n <\/div>\n\t\t\t\t\t <\/div>\n\t\t\t\t<\/div>\n\t\t\t<\/div>\n\t\t<\/div>\n\t<\/div>\n<\/div>\n\n

\n
\n\t\t
\n\t\t \t\t\t

References:\n<\/h4>\n\t\t\t\t\t\t
    \n
  1. VOXZOGO [package insert]. Novato, CA: BioMarin Pharmaceutical Inc; 2024.\n<\/span><\/li>\n
  2. 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. Med<\/em>. 2025;6(5):100566.\n<\/span><\/li>\n
  3. 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
  4. US Department of Health and Human Services. Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0. Published November 27, 2017. Accessed March 17, 2026. https:\/\/dctd.cancer.gov\/research\/ctep-trials\/for-sites\/adverse-events\/ctcae-v5-5×7.pdf\n<\/span><\/li>\n
  5. ClinicalTrials.gov. Search: Achondroplasia, BioMarin Pharmaceutical. Accessed March 17, 2026. https:\/\/www.clinicaltrials.gov\/search?cond=Achondroplasia&term=BioMarin%20Pharmaceutical&viewType=Table\n<\/span><\/li>\n
  6. Data on file [1]. BioMarin Pharmaceutical Inc; 2025.\n<\/span><\/li>\n
  7. 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. Lancet<\/em>. 2020;396(10252):684-692.\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-65","page","type-page","status-publish","hentry"],"acf":[],"yoast_head":"\nSafety Profile | VOXZOGO\u00ae (vosoritide) HCP<\/title>\n<meta name=\"description\" content=\"VOXZOGO safety in infants and children has been evaluated in multiple clinical trials. Learn more about the safety profile of this therapy.\" \/>\n<meta name=\"robots\" content=\"index, follow, max-snippet:-1, max-image-preview:large, max-video-preview:-1\" \/>\n<link rel=\"canonical\" href=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/safety-data\/\" \/>\n<meta property=\"og:locale\" content=\"en_US\" \/>\n<meta property=\"og:type\" content=\"article\" \/>\n<meta property=\"og:title\" content=\"Safety Profile | VOXZOGO\u00ae (vosoritide) HCP\" \/>\n<meta property=\"og:description\" content=\"VOXZOGO safety in infants and children has been evaluated in multiple clinical trials. Learn more about the safety profile of this therapy.\" \/>\n<meta property=\"og:url\" content=\"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/safety-data\/\" \/>\n<meta property=\"og:site_name\" content=\"BMRN Voxzogo HCP EN-US\" \/>\n<meta property=\"article:modified_time\" content=\"2026-06-02T21:30:49+00:00\" \/>\n<meta name=\"twitter:card\" content=\"summary_large_image\" \/>\n<script type=\"application\/ld+json\" class=\"yoast-schema-graph\">{\"@context\":\"https:\\\/\\\/schema.org\",\"@graph\":[{\"@type\":\"WebPage\",\"@id\":\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/voxzogo\\\/safety-data\\\/\",\"url\":\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/voxzogo\\\/safety-data\\\/\",\"name\":\"Safety Profile | VOXZOGO\u00ae (vosoritide) HCP\",\"isPartOf\":{\"@id\":\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/voxzogo\\\/#website\"},\"datePublished\":\"2022-11-28T20:55:16+00:00\",\"dateModified\":\"2026-06-02T21:30:49+00:00\",\"description\":\"VOXZOGO safety in infants and children has been evaluated in multiple clinical trials. Learn more about the safety profile of this therapy.\",\"breadcrumb\":{\"@id\":\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/voxzogo\\\/safety-data\\\/#breadcrumb\"},\"inLanguage\":\"en-US\",\"potentialAction\":[{\"@type\":\"ReadAction\",\"target\":[\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/voxzogo\\\/safety-data\\\/\"]}]},{\"@type\":\"BreadcrumbList\",\"@id\":\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/voxzogo\\\/safety-data\\\/#breadcrumb\",\"itemListElement\":[{\"@type\":\"ListItem\",\"position\":1,\"name\":\"Home\",\"item\":\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/voxzogo\\\/\"},{\"@type\":\"ListItem\",\"position\":2,\"name\":\"VOXZOGO Safety\"}]},{\"@type\":\"WebSite\",\"@id\":\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/voxzogo\\\/#website\",\"url\":\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/voxzogo\\\/\",\"name\":\"BMRN Voxzogo HCP EN-US\",\"description\":\"\",\"potentialAction\":[{\"@type\":\"SearchAction\",\"target\":{\"@type\":\"EntryPoint\",\"urlTemplate\":\"https:\\\/\\\/hcp.biomarin.com\\\/en-us\\\/voxzogo\\\/?s={search_term_string}\"},\"query-input\":{\"@type\":\"PropertyValueSpecification\",\"valueRequired\":true,\"valueName\":\"search_term_string\"}}],\"inLanguage\":\"en-US\"}]}<\/script>\n<!-- \/ Yoast SEO plugin. -->","yoast_head_json":{"title":"Safety Profile | VOXZOGO\u00ae (vosoritide) HCP","description":"VOXZOGO safety in infants and children has been evaluated in multiple clinical trials. Learn more about the safety profile of this therapy.","robots":{"index":"index","follow":"follow","max-snippet":"max-snippet:-1","max-image-preview":"max-image-preview:large","max-video-preview":"max-video-preview:-1"},"canonical":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/safety-data\/","og_locale":"en_US","og_type":"article","og_title":"Safety Profile | VOXZOGO\u00ae (vosoritide) HCP","og_description":"VOXZOGO safety in infants and children has been evaluated in multiple clinical trials. Learn more about the safety profile of this therapy.","og_url":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/safety-data\/","og_site_name":"BMRN Voxzogo HCP EN-US","article_modified_time":"2026-06-02T21:30:49+00:00","twitter_card":"summary_large_image","schema":{"@context":"https:\/\/schema.org","@graph":[{"@type":"WebPage","@id":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/safety-data\/","url":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/safety-data\/","name":"Safety Profile | VOXZOGO\u00ae (vosoritide) HCP","isPartOf":{"@id":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/#website"},"datePublished":"2022-11-28T20:55:16+00:00","dateModified":"2026-06-02T21:30:49+00:00","description":"VOXZOGO safety in infants and children has been evaluated in multiple clinical trials. Learn more about the safety profile of this therapy.","breadcrumb":{"@id":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/safety-data\/#breadcrumb"},"inLanguage":"en-US","potentialAction":[{"@type":"ReadAction","target":["https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/safety-data\/"]}]},{"@type":"BreadcrumbList","@id":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/safety-data\/#breadcrumb","itemListElement":[{"@type":"ListItem","position":1,"name":"Home","item":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/"},{"@type":"ListItem","position":2,"name":"VOXZOGO Safety"}]},{"@type":"WebSite","@id":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/#website","url":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/","name":"BMRN Voxzogo HCP EN-US","description":"","potentialAction":[{"@type":"SearchAction","target":{"@type":"EntryPoint","urlTemplate":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/?s={search_term_string}"},"query-input":{"@type":"PropertyValueSpecification","valueRequired":true,"valueName":"search_term_string"}}],"inLanguage":"en-US"}]}},"_links":{"self":[{"href":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-json\/wp\/v2\/pages\/65","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-json\/wp\/v2\/pages"}],"about":[{"href":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-json\/wp\/v2\/types\/page"}],"author":[{"embeddable":true,"href":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-json\/wp\/v2\/users\/4"}],"replies":[{"embeddable":true,"href":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-json\/wp\/v2\/comments?post=65"}],"version-history":[{"count":379,"href":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-json\/wp\/v2\/pages\/65\/revisions"}],"predecessor-version":[{"id":6328,"href":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-json\/wp\/v2\/pages\/65\/revisions\/6328"}],"wp:attachment":[{"href":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/wp-json\/wp\/v2\/media?parent=65"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}