Most adverse events were transient injection-site reactions, injection-site erythema; 7 percent in vosoritide group had serious adverse events<\/p>\n<\/h3>\n
By Elana Gotkine HealthDay Reporter<\/b><\/p>\n
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WEDNESDAY, Dec. 27, 2023 (HealthDay News) — 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<\/em>.<\/p>\n 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.<\/p>\n 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.<\/p>\n “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.<\/p>\n Several authors disclosed ties to biopharmaceutical companies, including BioMarin, which manufactures vosoritide and funded the study.<\/p>\n Abstract\/Full Text (subscription or payment may be required)<\/a><\/p>\n <\/i><\/p>\n