vosoritide
Voxzogo (vosoritide) is a C-type natriuretic peptide (CNP) analog from BioMarin for achondroplasia, the most common form of disproportionate short stature. In the United States it increases linear growth in children with open epiphyses, and in the European Union it is used from 4 months of age while growth plates remain open.
This page is based on the official US label for VOXZOGO and the EMA medicine record for Voxzogo, cross-checked with MedlinePlus; the mechanism is explained in plain language, and analogies are used only where they stay accurate.
How it works
Explains what the medicine does in the body, not dosing, side effects, or safety.
An analogy
In achondroplasia a mutated FGFR3 receptor keeps the brakes pressed on bone growth. Vosoritide works like a second signal that releases those brakes, letting the growth plates lay down new bone again.
In simple terms
Vosoritide is a daily injection that copies C-type natriuretic peptide, a natural growth-promoting signal. In achondroplasia, an overactive FGFR3 signal slows bone growth; vosoritide counters that signal at the growth plates, helping children grow faster while their growth plates are still open.
In depth
Vosoritide is an analog of C-type natriuretic peptide (CNP) with an added N-terminal extension that prolongs its half-life. It binds natriuretic peptide receptor B (NPR-B) on chondrocytes, activating guanylyl cyclase and raising cyclic GMP. This inhibits the overactive FGFR3-MAPK signalling that suppresses chondrocyte proliferation and differentiation in achondroplasia, restoring endochondral bone growth and increasing linear growth velocity while epiphyses remain open.
See it in motion
A schematic animation of the mechanism — simplified for clarity, not drawn to scale.
How vosoritide releases the brake on bone growth
- In achondroplasia, an overactive FGFR3 signal slows bone growth; vosoritide counters that signal at the growth plates, helping children grow faster while their growth plates are still open.
- It binds natriuretic peptide receptor B (NPR-B) on chondrocytes, activating guanylyl cyclase and raising cyclic GMP.
- Vosoritide works like a second signal that releases the brake a mutated FGFR3 receptor keeps pressed on bone growth.
- The growth plates can lay down new bone again.
Target: Natriuretic peptide receptor B (NPR-B)
A receptor on growth-plate cartilage cells that, when activated, counteracts the overactive signal that restricts bone growth in achondroplasia.
Approved uses
Approved uses differ by country and change over time. Always follow your local prescribing information.
Approved uses in United States
FDA- Increase linear growth in pediatric patients with achondroplasia with open epiphyses
Approved uses in European Union
EMA- Treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed; the diagnosis of achondroplasia should be confirmed by appropriate genetic testing
Official sources
The statements on this page trace back to these records.
- Voxzogo (vosoritide) — Summary of product characteristics and package leaflet, English version
European Medicines Agency
- openFDA drug label record for vosoritide (Voxzogo)
U.S. Food and Drug Administration, openFDA
- MedlinePlus: vosoritide
MedlinePlus, U.S. National Library of Medicine
This page explains how the medicine works — it isn’t the patient leaflet. For anything that applies to you, read the leaflet that came with your medicine or ask your pharmacist.
MedUnfold provides educational content and is not medical advice. Always follow your local prescribing information and healthcare professional.