A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia
Primary result · LSM change from baseline in annualized growth velocityResult — go to the resultLSM difference 2.33 cm/year 95% CI 1.85–2.82Authors' conclusion
One year of vosoritide treatment significantly increased linear growth in children with hypochondroplasia.
Study context
- Population
- Children with hypochondroplasia aged 3 to <18 years
- Participants
- 81 children
- Comparison
- Vosoritide vs placebo
- Vosoritiden = 41
- Placebon = 40reference
- Primary endpoint
- Change from baseline in annualized growth velocity vs placebo
- Follow-up
- Week 52
Methodological detailsphase 3, multicenter, placebo-controlled, Hierarchical testing controlling type I error at one-sided 0.025 level for primary and six key secondary end points
- Phase 3
- Multicenter
- Placebo-controlled
- Hierarchical testing controlling type I error at one-sided 0.025 level for primary and six key secondary end points
Primary result
As reported in the abstract
LSM change from baseline in annualized growth velocity
Least squares mean (LSM) difference
2.33 cm/year
95% CI 1.85–2.82
two-sided P<0.0001
Change by group
Vosoritide
1.95
least squares mean · cm/year
Placebo
−0.39
least squares mean · cm/year
Vosoritidevs Placebo
LSM difference 2.33 cm/year (95% CI 1.85–2.82); two-sided P<0.0001
Safety
At least one AE · 52 weeks
Serious adverse event (AE) outcomes
- There were no reports of grade 3 or higher AEs, AEs leading to treatment discontinuation, or deaths
Original publication
A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia. NEJM Evid. Published 9 September 2026. doi:10.
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