What is the mode of action of Vosoritide (vosoritide) for achondroplasia?

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From the Research

Mode of Action of Vosoritide

• Vosoritide is a biologic analogue of C-type natriuretic peptide, a potent stimulator of endochondral ossification 1, 2
• It acts on the growth plate through the natriuretic peptide receptor-B (NPR-B) causing the transformation of guanosine 5'-triphosphate into cyclic guanosine monophosphate 2
• Vosoritide has a half-life 10 times that of C-type natriuretic peptide, making it suitable for treatment of achondroplasia 2
• The mechanism of action of vosoritide involves stimulating endochondral bone growth, which is impaired in achondroplasia due to a mutation in the fibroblast growth factor receptor 3 (FGFR3) gene 1, 3, 4

Efficacy of Vosoritide

• Clinical trials have demonstrated that vosoritide is effective in significantly increasing the annualized growth velocity in children with achondroplasia before the fusion of the epiphyses 5, 1, 2, 4
• Vosoritide treatment has been shown to have a positive benefit-risk ratio, with a side-effect profile that appears generally mild 5, 1, 4
• The efficacy of vosoritide has been consistently demonstrated in multiple studies, with sustained increases in annualized growth velocity observed for up to 42 months 1 and 2 years 4

Administration and Dosage

• Vosoritide is administered via once-daily subcutaneous injection, with a typical dose of 15 μg/kg/day 5, 1, 4
• The dosage of vosoritide has been evaluated in clinical trials, with a dose-dependent increase in annualized growth velocity observed up to a dose of 15.0 μg/kg 1