A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia.

Dauber A, Savarirayan R, Campeau PM, Carroll RS, Cheung M, Cormier-Daire V, de Bergua JM, Edouard T, Gonfiantini MV, Hoover-Fong J, Kannu P, Kinoshita Y and 18 more

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Kubota T, Mendoza-Londono R, Namba N, Ohata Y, Onesimo R, Palm K, Prada CE, Reynaud R, Rossi M, Semler O, Takasawa K, Bassi R, Ghani S, Huntsman Labed A, Osmond D, Sabir I, White J, Maghnie M

NEJM evidence9 Sept 2026

PMID42713641DOI10.1056/evidoa2600257PepBankPUB-42713641

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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: Publisher copyright. Results still quote short attributed sentences.

Results from this study5

5 results from studies that gave the peptide. 5 results from people.

01HumanRandomized controlled trialEfficacy outcome
In children with hypochondroplasia, vosoritide increased annualized growth velocity more than placebo at week 52, with an LSM difference of 2.33 cm/year.
In plain wordsIn children with hypochondroplasia, vosoritide increased growth velocity more than placebo at week 52.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity change at week 52
Study
n = 81
Comparator
Placebo
Analysis
Primary
Outcome group
Linear growth · Musculoskeletal
Studied dose
Dose not stated · subcutaneous · 52 weeksAs given in this study (human).
Measurements
LSM change
1.95 cm/year
vosoritide
LSM change
−0.39 cm/year
placebo
LSM difference
2.33 cm/year
95% CI
1.85 to 2.82 cm/year
Two-sided P
< 0.0001
Source sentences
Summary
At week 52, the least squares mean (LSM) change from baseline in annualized growth velocity was 1.95 cm/year with vosoritide versus -0.39 cm/year with placebo (LSM difference of 2.33 cm/year; 95% confidence interval, 1.85-2.82 cm/year; two-sided P<0.0001).
2 context sentences(design, population, supporting numbers)
Summary · context
In this phase 3, multicenter trial, children with hypochondroplasia who were 3 to less than 18 years of age were randomly assigned to receive once-daily subcutaneous injections of vosoritide or placebo for 52 weeks per weight-band dosing regimen.
Summary · context
A total of 81 participants were randomly assigned to receive vosoritide (n=41) or placebo (n=40).
Report a problemFND-4271364101
02HumanRandomized controlled trialSafety outcome
In children with hypochondroplasia, at least one adverse event occurred in 87.8% with vosoritide versus 72.5% with placebo.
In plain wordsIn children with hypochondroplasia, adverse events occurred in 87.8% with vosoritide versus 72.5% with placebo.
Peptide
Vosoritide
Outcome
IncreaseAt least one adverse event
Study
n = 81
Comparator
Placebo
Outcome group
Adverse events · Whole body
Studied dose
Dose not stated · subcutaneous · 52 weeksAs given in this study (human).
Measurements
At least one AE
87.8%
vosoritide
At least one AE
72.5%
placebo
Source sentences
Summary
Most participants in the vosoritide group (87.8%) and the placebo group (72.5%) experienced at least one adverse event (AE).
2 context sentences(design, population, supporting numbers)
Summary · context
In this phase 3, multicenter trial, children with hypochondroplasia who were 3 to less than 18 years of age were randomly assigned to receive once-daily subcutaneous injections of vosoritide or placebo for 52 weeks per weight-band dosing regimen.
Summary · context
A total of 81 participants were randomly assigned to receive vosoritide (n=41) or placebo (n=40).
Report a problemFND-4271364102
03HumanNon-randomized trialSafety outcome
No grade 3 or higher adverse events were reported in the vosoritide trial in children with hypochondroplasia.
In plain wordsNo grade 3 or higher adverse events were reported in the vosoritide trial in children with hypochondroplasia.
Peptide
Vosoritide
Outcome
Not applicableGrade 3 or higher adverse events
Study
n = 41
Outcome group
Adverse events · Whole body
Studied dose
Dose not stated · subcutaneous · 52 weeksAs given in this study (human).
Source sentences
Summary
There were no reports of grade 3 or higher AEs, AEs leading to treatment discontinuation, or deaths.
2 context sentences(design, population, supporting numbers)
Summary · context
In this phase 3, multicenter trial, children with hypochondroplasia who were 3 to less than 18 years of age were randomly assigned to receive once-daily subcutaneous injections of vosoritide or placebo for 52 weeks per weight-band dosing regimen.
Summary · context
A total of 81 participants were randomly assigned to receive vosoritide (n=41) or placebo (n=40).
Report a problemFND-4271364103
04HumanNon-randomized trialSafety outcome
No adverse events leading to treatment discontinuation were reported in the vosoritide trial in children with hypochondroplasia.
In plain wordsNo adverse events caused treatment discontinuation in the vosoritide trial in children with hypochondroplasia.
Peptide
Vosoritide
Outcome
Not applicableAdverse events leading to treatment discontinuation
Study
n = 41
Outcome group
Adverse events · Whole body
Studied dose
Dose not stated · subcutaneous · 52 weeksAs given in this study (human).
Source sentences
Summary
There were no reports of grade 3 or higher AEs, AEs leading to treatment discontinuation, or deaths.
2 context sentences(design, population, supporting numbers)
Summary · context
In this phase 3, multicenter trial, children with hypochondroplasia who were 3 to less than 18 years of age were randomly assigned to receive once-daily subcutaneous injections of vosoritide or placebo for 52 weeks per weight-band dosing regimen.
Summary · context
A total of 81 participants were randomly assigned to receive vosoritide (n=41) or placebo (n=40).
Report a problemFND-4271364104
05HumanNon-randomized trialSafety outcome
No deaths were reported in the vosoritide trial in children with hypochondroplasia.
In plain wordsNo deaths were reported in the vosoritide trial in children with hypochondroplasia.
Peptide
Vosoritide
Outcome
Not applicableDeaths
Study
n = 41
Outcome group
Mortality · Whole body
Studied dose
Dose not stated · subcutaneous · 52 weeksAs given in this study (human).
Source sentences
Summary
There were no reports of grade 3 or higher AEs, AEs leading to treatment discontinuation, or deaths.
2 context sentences(design, population, supporting numbers)
Summary · context
In this phase 3, multicenter trial, children with hypochondroplasia who were 3 to less than 18 years of age were randomly assigned to receive once-daily subcutaneous injections of vosoritide or placebo for 52 weeks per weight-band dosing regimen.
Summary · context
A total of 81 participants were randomly assigned to receive vosoritide (n=41) or placebo (n=40).
Report a problemFND-4271364105

AI wrote and checked these results. AI can make mistakes, so check important results against the source.

Registered trials1

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

1 trial · 1 ongoing · source ClinicalTrials.gov (opens in a new tab)

Active, not recruitingPhase 3NCT06455059

Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia

BioMarin Pharmaceuticaln = 8017 Jun 2024 to est. 1 Aug 2026

Hypochondroplasia

Trial page ClinicalTrials.govUpdated 20 Jan 2026

Version history

PepBank keeps every version of the data on this study. Status changes from Retraction Watch, Europe PMC and Crossref are listed with their source.

Version 2Current
28 Oct 2026, 02:03 UTC
Study results updated
Version 1
27 Oct 2026, 16:37 UTC
First added · source update from Europe PMC

Subjects

Terms as the source lists them.

Publication types

Journal Article

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