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.
Chamgap Semantic Classification Code
Candidate index · review held
M.vosoritide-by-once-subcutaneous-injection.subcutaneous.annualized-growth-velocity-over-with-achondroplasia.increase.MULTIMedicinal interventions > Vosoritide by once- subcutaneous injection > Subcutaneous > annualized growth velocity over with achondroplasia > Increase claim > Multiple: primary unresolved
An automated migration candidate, not an issued permanent code; exact claim scope remains under review. This machine-generated migration candidate helps retrieval but is not a permanent assignment. The original verdict ID and URL remain authoritative.
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 |
Stored derived and displayed grades match; this is not a current recalculation or validity check (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 — AI research and Codex final gate
Evidence Table
| Study | Design | Sample | Funding | Endpoint | Result | Weight |
|---|---|---|---|---|---|---|
| Savarirayan R et al. 2020 phase 3 trial | Double-blind placebo-controlled phase 3 randomized trial at 24 sites | 121 randomized, 60 vosoritide and 61 placebo; 119 treated | 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).
Final verification and publication gate: Codex · Evidence 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
Chamgap is an information source. It reports what research has and has not confirmed; it does not tell readers what to take or buy. That decision belongs to readers and, when needed, medical or legal professionals. This verdict reflects literature available up to the search date and may change as new research appears. Nothing here is medical advice.