How well does VOXZOGO(Vosoritide) work?

VOXZOGO has demonstrated efficacy in increasing linear growth velocity in pediatric patients with achondroplasia.

Efficacy in Promoting Linear Growth

Clinical studies have shown that VOXZOGO significantly increases annualized growth velocity in children with achondroplasia and open epiphyses compared to placebo. The drug acts by antagonizing FGFR3 signaling, thereby promoting chondrocyte proliferation and differentiation, which leads to enhanced endochondral bone growth. Improvements in height standard deviation scores and maintenance of growth velocity have been observed in long-term extension studies. The therapeutic benefit is contingent upon continued open growth plates, and treatment should be discontinued once epiphyseal closure is confirmed. Effects in other populations are not specified in the description.