Analog of a natural peptide that stimulates bone growth. Used in children with achondroplasia while their growth plates are still open.
Regulatory status
Approved (FDA / EMA)
Family
C-type natriuretic peptide (CNP) analog
Scientific evidence
Moderate
Number of findings
2
Where it acts
Bones and muscles
Also known as
vosoritide, Voxzogo, BMN 111, CNP analog
Regulatory status
FDA (US): Approved (2021, Voxzogo, accelerated approval) to increase growth in children with achondroplasia with open growth plates; expanded in 2023 to all pediatric ages.
EMA (EU): Approved (2021, Voxzogo) for achondroplasia in children with open growth plates; minimum age later extended to infants.
Dose
One subcutaneous injection daily according to weight (about 15 µg/kg in the phase 3 trial); stopped when the epiphyses close (FDA/EMA Voxzogo label).
Safety
Injection-site reactions (redness, swelling), vomiting, transient drop in blood pressure with dizziness; it is advisable to eat and drink before each dose. Not recommended with low kidney filtration. Only with specialized supervision.
Interactions
Caution with drugs that lower blood pressure.
Effect by goal · level of evidence
Faster growth velocity in achondroplasia
increasesHighRCT (AA)in people
+1.57 cm/year
Phase 3 with 121 children aged 5 to 14 years over 52 weeks: they grew 1.57 cm/year more than with placebo. There were no serious drug-related adverse effects.
Phase 2 with 75 children aged 3 to 59 months over 52 weeks: trend toward improved height (Z-score difference of 0.25), not statistically significant. Well tolerated.
In achondroplasia, a receptor (FGFR3) is overactive and slows cartilage growth. Vosoritide activates another receptor (NPR-B) that counteracts that brake. The growth plates then produce more bone and the child grows faster.
What the evidence says
In people
Faster growth velocity in achondroplasia. Phase 3 trial with 121 children aged 5 to 14 years over 52 weeks: they grew 1.57 cm/year more than with placebo. There were no serious drug-related adverse effects. PubMed 32891212
Infants and young children. Phase 2 trial with 75 children aged 3 to 59 months over 52 weeks: trend toward improved height (Z-score difference of 0.25), not statistically significant. Well tolerated. PubMed 37984383