May 21, 2024

Beneficial Effects of Vosoritide in Children with Achondroplasia Under 5 Years of Age

A recent study published in The Lancet Child & Adolescent Health has shown that the drug vosoritide can be beneficial for children under the age of five with achondroplasia, a genetic disorder that affects bone development. The study, conducted by researchers from the Royal Children’s Hospital at the University of Melbourne and other international institutions, found that vosoritide was associated with an increase in the change in height Z score from baseline and had a relatively mild adverse event profile.

The study involved a double-blind, randomized phase 2 trial conducted across 16 hospitals in Australia, Japan, the United Kingdom, and the United States. Participants in the study were children under 60 months of age who had been diagnosed with achondroplasia through genetic testing. They were divided into three cohorts based on age at screening: 24 to 59 months, 6 to 23 months, and 0 to 5 months.

In total, 75 participants were recruited, with 11 assigned as sentinels to determine the appropriate daily dose of vosoritide. The remaining 32 participants were randomly assigned to either receive vosoritide or a placebo for a duration of 52 weeks. The researchers monitored and recorded any adverse events that occurred during the study.

The study found that adverse events did occur in all participants, but the majority of these events were transient injection-site reactions and injection-site erythema. Only a small percentage of participants experienced serious adverse events, with three patients in the vosoritide group and six in the placebo group reporting such events. The difference in the change from baseline in height Z score between the vosoritide and placebo groups was 0.25.

The findings of this study are promising for children with achondroplasia and suggest that vosoritide could provide a viable treatment option. The authors of the study hope that these results will assist pediatricians and other healthcare specialists in accurately assessing the risks and benefits of initiating vosoritide treatment in children under the age of five with achondroplasia.

It is important to note that several authors of the study have disclosed affiliations with biopharmaceutical companies, including BioMarin, the manufacturer of vosoritide, which also funded the study. Further research and clinical trials will be necessary to validate these findings and ensure the safety and efficacy of vosoritide in treating achondroplasia in young children.

1. Source: Coherent Market Insights, Public sources, Desk research
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