Vosoritide,
does it really help with Increased annualized growth velocity over 52 weeks in children with achondroplasia?
research showsThe grade is C with 50 points. In a 121-patient double-blind placebo-controlled trial, the 52-week change in annualized growth velocity was 1.57 cm/year greater with vosoritide (95% CI 1.22 to 1.93; P<.0001). The difference was large by statistical convention, but the primary outcome was one-year growth velocity rather than adult height, function, or complications, and evidence came from one BioMarin-funded trial.
ads claimA one-year growth-velocity difference cannot be restated as improved adult height, mobility, self-care, spinal stenosis, or sleep apnea. The original report also stated that adult height and long-term harms were unknown.
Useful facts when choosing a product
- The trial is registered as NCT03197766 and EudraCT 2015-003836-11.
- All 119 treated participants reported at least one adverse event.
- No treatment-related serious adverse event or death was reported.
What the research actually shows
At 24 sites, 121 children aged 5 to under 18 were randomized to vosoritide, 60, or placebo, 61. Randomization used a voice or web-response system, and participants, investigators, and the sponsor were masked. The adjusted primary difference was 1.57 cm/year (95% CI 1.22 to 1.93). With no validated clinical threshold, magnitude was classified by statistical convention. From the interval, SE=(1.93−1.22)/(2×1.96)=0.181, SD=0.181/√(1/60+1/61)=1.00, and d≈1.58. Cross-checking the reported change SDs of 1.71 and 1.74 gave d≈0.91. The adjusted and raw-data calculations differed, but both exceeded Cohen 0.5. Enrollment below 200 was counted as one limitation. BioMarin Pharmaceutical funded the study.
Why this is classified as C (50)
A large 52-week growth-velocity difference was limited by an intermediate outcome, one manufacturer-funded trial, and enrollment below 200, giving C with 50 points.
Counterpoint. Final height, function, complications, and long-term safety were not conclusions of this 52-week trial.
Rejudgment record. Cross-check applied — Cross-checked the Lancet report and NCT03197766 for the adjusted primary result, randomization and masking, analysis counts, BioMarin funding, reported standard deviations, and unmeasured long-term outcomes
| Endpoint | S | Surrogate marker - laboratory or imaging measures |
| Replication | R1 | Single confirmatory trial |
| Independence | I0 | Evidence comes only from manufacturer studies |
| Effect size | E+ | Meets the clinically important threshold |
The scoring table and the verdict agree (C).
Sub-claim grades by effect
This ingredient is marketed for several effects. A single overall grade blends strong and weak claims together, so each effect is graded separately here. The overall grade reflects the strongest disconfirming or core claim.
| Effect (sub-claim) | Grade | Basis |
|---|---|---|
| Increased annualized growth velocity at 52 weeks | C | The adjusted difference was 1.57 cm/year. |
| Improved adult height, function, or complications | ? | The 52-week trial did not measure these outcomes. |
Cross-check — Codex and Claude
Evidence Table
| Study | Design | Sample | Funding | Endpoint | Result | Weight |
|---|---|---|---|---|---|---|
| Study 1 | Double-blind placebo-controlled phase 3 randomized trial at 24 sites | 119 | BioMarin Pharmaceutical | Change from baseline in annualized growth velocity at week 52 | Adjusted difference 1.57 cm/year (95% CI 1.22 to 1.93; P<.0001); no treatment-related serious adverse event or death | Single manufacturer-funded trial with an intermediate outcome |
Receipt — 1 References
All 1 cited sources were verified for existence at the original page (as of 2026-08-18).
Reviewed and approved: Chamgap Editorial Team · Approval date: 2026-08-18 · Corrections: none
Cite this verdict
[Chamgap] Vosoritide x growth velocity in pediatric achondroplasia — Evidence Grade C·50. 1 cited sources checked. Source: https://chamgap.com/en/verdicts/joint-bone/vosoritide-pediatric-achondroplasia-annualized-growth-velocity/ · CC BY 4.0CC BY 4.0 — free to use with attribution; do not distort grades, numbers, or verdict meaning.
What this document does and does not do
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