Vosoritide (Voxzogo)
Overview
A C-type natriuretic peptide (CNP) analog that acts as an agonist at the natriuretic peptide receptor B (NPR-B) on growth plate chondrocytes. Vosoritide counteracts the constitutively activated FGFR3 signaling that suppresses endochondral ossification in achondroplasia. By stimulating the NPR-B/cGMP/MAPK pathway, it antagonizes the growth-inhibitory FGFR3 signal and restores more normal linear bone growth.
Key Research Findings
FDA-approved in 2021 for achondroplasia in children 5 years and older with open growth plates. Phase 3 trial demonstrated a sustained increase in annualized growth velocity of 1.57 cm/year vs. placebo over 52 weeks (Savarirayan et al., NEJM, 2020). Five-year extension data showed maintained growth velocity improvement. Well-tolerated with transient injection site reactions and blood pressure decreases as common side effects.
Subcutaneous injection (daily)
FDA Approved
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