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Vosoritide Beneficial for Children Younger Than 5 Years With Achondroplasia

Medically reviewed by Carmen Pope, BPharm. Last updated on Dec 27, 2023.

By Elana Gotkine HealthDay Reporter

WEDNESDAY, Dec. 27, 2023 -- For children with achondroplasia, vosoritide is associated with a gain in the change in height Z score from baseline and a mild adverse event profile, according to a study published in the January issue of The Lancet Child & Adolescent Health.

Ravi Savarirayan, M.D., from the Royal Children's Hospital at the University of Melbourne in Australia, and colleagues conducted a double-blind, randomized phase 2 trial in 16 hospitals across Australia, Japan, the United Kingdom, and the United States to examine the safety and efficacy of vosoritide in children younger than 60 months with a clinical diagnosis of achondroplasia confirmed by genetic testing. Participants were enrolled into three cohorts based on age at screening: 24 to 59 months, 6 to 23 months, and 0 to 5 months (cohorts 1, 2, and 3, respectively). Seventy-five participants were recruited: 11 were assigned as sentinels who received vosoritide to determine appropriate daily dose, and 32 were randomly assigned to receive vosoritide or placebo for 52 weeks.

The researchers found that adverse events occurred in all participants, most of which were transient injection-site reactions and injection-site erythema. Three patients in the vosoritide group and six in the placebo group (7 and 19 percent, respectively) had serious adverse events. For change from baseline in height Z score, the least-squares mean difference between the vosoritide and placebo groups was 0.25.

"We hope these data will support pediatricians and other health care specialists who are assessing the risks and benefits of initiating vosoritide treatment in children with achondroplasia younger than 5 years," the authors write.

Several authors disclosed ties to biopharmaceutical companies, including BioMarin, which manufactures vosoritide and funded the study.

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Disclaimer: Statistical data in medical articles provide general trends and do not pertain to individuals. Individual factors can vary greatly. Always seek personalized medical advice for individual healthcare decisions.

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Posted December 2023

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