Efficacy of vosoritide treatment in patients with achondroplasia

Objectives: The objective was to evaluate the efficacy and safety profile of vosoritide treatment in children with achondroplasia after 1 year of administration. Material and Methods: This was a retrospective study including 14 patients with a confirmed diagnosis of achondroplasia who received vosoritide treatment for 12 months. Changes in height Z-score, annualized growth velocity (AGV), arm span/height ratio, sitting height/standing height ratio, changes in bone age, and reported adverse events were analyzed. Results: A significant increase in AGV of 0.89 ± 1.05 cm/year ( P = 0.021) was observed, along with an increase in AGV Z-score of 1.3 ± 2.2 ( P = 0.042) and a height Z-score increase of 0.34 ± 0.38 ( P = 0.007). A group of five patients did not show clinically relevant improvement in height Z-score. No serious adverse events were reported. Conclusion: Vosoritide treatment was generally effective and well tolerated, leading to an average increase in height Z-score after 1 year, with no serious side effects. Although some patients showed limited response, our findings align with previous studies. AGV is helpful but may not fully reflect treatment efficacy, especially in small cohorts. Larger, long-term studies are needed to confirm these results and clarify the clinical impact.