{"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":"2026-08-25T14:33:22","modified_gmt":"2026-08-25T14:33:22","slug":"safety-data","status":"publish","type":"page","link":"https:\/\/hcp.biomarin.com\/en-us\/voxzogo\/safety-data\/","title":{"rendered":"VOXZOGO Safety"},"content":{"rendered":"
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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
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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 \n <\/div>\n \n

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VOXZOGO may cause serious side effects including a temporary decrease in blood pressure in some patients;\u00a0to reduce the risk of decreased 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 <\/div>\n\n

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*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<\/sup>
\n\u2020<\/sup>Injection site reactions occurring more frequently in VOXZOGO-treated patients than placebo.1<\/sup>
\n\u2021<\/sup>All events were transient and resolved without medical intervention; 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.4<\/sup>
\n\u00a7<\/sup>Includes the preferred terms: gastroenteritis and gastroenteritis, viral.1<\/sup>
\n||<\/sup>Includes the preferred terms: dizziness, presyncope, procedural dizziness, and vertigo.1<\/sup>
\n\u00b6<\/sup>Includes the preferred terms: fatigue, lethargy, and malaise.1<\/sup>
\n#<\/em><\/sup>1 patient discontinued treatment due to pain, and 1 discontinued treatment due to anxiety with injections.1<\/sup><\/small><\/p>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n

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VOXZOGO has over 10 years of clinical trial experience in achondroplasia.1,5<\/sup><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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Consistent safety profile in children aged <5 years1,3<\/sup><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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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 \n <\/div>\n \n

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ADL,<\/strong> activities of daily living; AE,<\/strong> adverse event; CTCAE,<\/strong> Common Terminology Criteria for Adverse Events; SAE,<\/strong> serious adverse event.<\/small>
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\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.6<\/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>
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“Knowing that VOXZOGO is available from birth made us confident in our decision to treat Sophie with it.”<\/h3>\n

\u2013 Amanda, Sophie\u2019s mom<\/p>\n \t\t<\/div>\n\t<\/div>\n\t

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Consistent safety profile in children aged <2 years3<\/sup><\/h2>\n <\/div>\n <\/div>\n <\/div>\n<\/div>\n\n
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Over 3 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 \n <\/div>\n \n

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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.6<\/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

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Hear from an experienced specialist\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

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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

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References:\n<\/h4>\n\t\t\t\t\t\t
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  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. 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
  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. 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 <\/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":"\nAchondroplasia Treatment Safety Profile | VOXZOGO\u00ae Data<\/title>\n<meta name=\"description\" content=\"Explore the safety data on VOXZOGO\u00ae (vosoritide) from pivotal and long-term clinical trials in infants and children with achondroplasia.\" \/>\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\/\" 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