Real-world Outcome of Vosoritide Treatment in Children With Achondroplasia: A 12-month Retrospective Observational Study.

Reincke S, Semler O, Junghänel-Welzing S, Stasek S, Rehberg M, Pfeiffer E, Hoyer-Kuhn H

Journal of the Endocrine Society8 Mar 2025Cited by 19 (Europe PMC)

PMID40134702PMCIDPMC11932077DOI10.1210/jendso/bvaf041PepBankPUB-40134702

Summary

ContextVosoritide is the first approved targeted therapy for achondroplasia (ACH) based on increased annualized growth velocity in clinical trials. The aim of our project was an assessment of the real-world setting and treatment with vosoritide.DesignThis was a 12-month, retrospective observational study on an inception cohort of 34 patients with ACH treated with vosoritide.Patients and methodsThirty-four patients with ACH (22 males; aged 2.8 to 15.3 years at treatment initiation) who received vosoritide treatment for at least 12 months at a specialized clinic for skeletal dysplasia in childhood were included in the analysis. Auxological measurements at baseline and after 12 months of therapy were converted into disease-specific (ACH) and general population [Centers for Disease Control and Prevention (CDC)] z-scores. Physical function assessed by a 6-minute walk test was converted into z-scores and compared to an unaffected reference cohort.ResultsAfter 12 months of treatment, both ACH and CDC height z-scores showed significant increases, with mean changes (mean ± SD) of 0.52 ± 0.35 and 0.38 ± 0.44, respectively (both P < .0001). The annualized growth velocity exceeded reference values for untreated children with ACH. No significant changes were observed in body mass index, upper to lower body segment ratio (sitting height/height), or head circumference. The 6-minute walking distance improved, with z-scores increasing from -2.00 ± 1.12 to -1.39 ± 1.23 (P = .0215).ConclusionIn a real-world setting, children with ACH showed significant improvements in growth and physical function after 12 months of treatment with vosoritide.

CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study7

7 results from studies that gave the peptide. 7 results from people.

01HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, ACH height z-score increased by 0.52 ± 0.35 after 12 months.
In plain wordsIn children with achondroplasia receiving vosoritide, disease-specific height scores increased from baseline.
Peptide
Vosoritide
Outcome
IncreaseACH height z-score
Study
n = 34
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 12 monthsAs given in this study (human).
Measurements
ACH height z-score mean change
0.52 z-score
after 12 months of vosoritide versus baseline; SD 0.35
Source sentences
Summary
After 12 months of treatment, both ACH and CDC height z-scores showed significant increases, with mean changes (mean ± SD) of 0.52 ± 0.35 and 0.38 ± 0.44, respectively (both P < .0001).
1 context sentence(design, population, supporting numbers)
Summary · context
This was a 12-month, retrospective observational study on an inception cohort of 34 patients with ACH treated with vosoritide.
Show in the study text Report a problemFND-4013470201
02HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, CDC height z-score increased by 0.38 ± 0.44 after 12 months.
In plain wordsIn children with achondroplasia receiving vosoritide, general-population height scores increased from baseline.
Peptide
Vosoritide
Outcome
IncreaseCDC height z-score
Study
n = 34
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 12 monthsAs given in this study (human).
Measurements
CDC height z-score mean change
0.38 z-score
after 12 months of vosoritide versus baseline; SD 0.44
Source sentences
Summary
After 12 months of treatment, both ACH and CDC height z-scores showed significant increases, with mean changes (mean ± SD) of 0.52 ± 0.35 and 0.38 ± 0.44, respectively (both P < .0001).
1 context sentence(design, population, supporting numbers)
Summary · context
This was a 12-month, retrospective observational study on an inception cohort of 34 patients with ACH treated with vosoritide.
Show in the study text Report a problemFND-4013470202
03HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, annualized growth velocity exceeded untreated reference values.
In plain wordsChildren with achondroplasia receiving vosoritide grew faster than untreated reference values.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity
Study
n = 34
Comparator
Untreated achondroplasia reference values
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 12 monthsAs given in this study (human).
Source sentences
Summary
The annualized growth velocity exceeded reference values for untreated children with ACH.
1 context sentence(design, population, supporting numbers)
Summary · context
This was a 12-month, retrospective observational study on an inception cohort of 34 patients with ACH treated with vosoritide.
Show in the study text Report a problemFND-4013470203
04HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, body mass index did not change significantly from baseline.
In plain wordsIn children receiving vosoritide, body mass index did not change significantly from baseline.
Peptide
Vosoritide
Outcome
No significant changeBody mass index
Study
n = 34
Comparator
Baseline
Outcome group
Body weight · Metabolic and weight
Studied dose
Dose not stated · 12 monthsAs given in this study (human).
Source sentences
Summary
No significant changes were observed in body mass index, upper to lower body segment ratio (sitting height/height), or head circumference.
1 context sentence(design, population, supporting numbers)
Summary · context
This was a 12-month, retrospective observational study on an inception cohort of 34 patients with ACH treated with vosoritide.
Show in the study text Report a problemFND-4013470204
05HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, sitting height/height did not change significantly from baseline.
In plain wordsIn children receiving vosoritide, body segment ratio did not change significantly from baseline.
Peptide
Vosoritide
Outcome
No significant changeBody segment ratio
Study
n = 34
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 12 monthsAs given in this study (human).
Source sentences
Summary
No significant changes were observed in body mass index, upper to lower body segment ratio (sitting height/height), or head circumference.
1 context sentence(design, population, supporting numbers)
Summary · context
This was a 12-month, retrospective observational study on an inception cohort of 34 patients with ACH treated with vosoritide.
Show in the study text Report a problemFND-4013470205
06HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, head circumference did not change significantly from baseline.
In plain wordsIn children receiving vosoritide, head circumference did not change significantly from baseline.
Peptide
Vosoritide
Outcome
No significant changeHead circumference
Study
n = 34
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 12 monthsAs given in this study (human).
Source sentences
Summary
No significant changes were observed in body mass index, upper to lower body segment ratio (sitting height/height), or head circumference.
1 context sentence(design, population, supporting numbers)
Summary · context
This was a 12-month, retrospective observational study on an inception cohort of 34 patients with ACH treated with vosoritide.
Show in the study text Report a problemFND-4013470206
07HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, walking distance z-score increased from -2.00 ± 1.12 to -1.39 ± 1.23 (P = .0215).
In plain wordsIn children with achondroplasia receiving vosoritide, walking distance improved from baseline.
Peptide
Vosoritide
Outcome
IncreaseSix-minute walking distance
Study
n = 34
Comparator
Baseline
Outcome group
Exercise and physical function · Musculoskeletal
Studied dose
Dose not stated · 12 monthsAs given in this study (human).
Measurements
6-minute walk distance z-score at baseline
−2 z-score
baseline; SD 1.12
6-minute walk distance z-score after 12 months
−1.39 z-score
after 12 months of vosoritide; SD 1.23
Source sentences
Summary
The 6-minute walking distance improved, with z-scores increasing from -2.00 ± 1.12 to -1.39 ± 1.23 (P = .0215).
1 context sentence(design, population, supporting numbers)
Summary · context
This was a 12-month, retrospective observational study on an inception cohort of 34 patients with ACH treated with vosoritide.
Show in the study text Report a problemFND-4013470207

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

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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

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