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.
Mechanism of Action
By stimulating the NPR-B/cGMP/MAPK pathway, it antagonizes the growth-inhibitory FGFR3 signal and restores more normal linear bone growth..
Research Summary & Key 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.
Clinical Status
Vosoritide (Voxzogo) has received FDA approval and is available for clinical use under appropriate medical supervision. Consult a qualified healthcare provider for prescribing information.
Administration Routes
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