Real-world growth outcomes of vosoritide in children with achondroplasia: a single-center cohort study.

Regev R, Waksman Y, Cohen-Sela E, Brener A, Dechovich N, Gal M, Borger O, Zeitlin L, Lebenthal Y

Journal of the Endocrine Society28 Mar 2026Cited by 1 (Europe PMC)

PMID42238253PMCIDPMC13227001DOI10.1210/jendso/bvag072PepBankPUB-42238253

Summary

ContextVosoritide, a C-type natriuretic peptide analogue, is the first approved therapy targeting the pathophysiology of achondroplasia. While clinical trials demonstrate efficacy, individuals with prior orthopedic surgeries are typically excluded, and real-world data remain limited.ObjectiveTo evaluate real-world growth outcomes of vosoritide therapy in children with achondroplasia, including those with prior surgical interventions.MethodsThis retrospective analysis included 25 children with achondroplasia (17 boys, age range 2.9-14.3 years) treated with vosoritide for a mean duration of 12.7 months at a multidisciplinary center. Z-scores were calculated using an AI-assisted growth assessment tool previously validated for children with achondroplasia. Δ z-scores for height, sitting height, arm span, and body mass index (BMI) were calculated from baseline to follow-up. Multiple linear regression analyses assessed predictors of response.ResultsMean height z-scores improved from -0.62 ± 1.09 to -0.24 ± 1.20 (P < .001). Mean arm span z-score increased from -1.28 ± 0.93 to -0.96 ± 0.91 (P = .007). Sitting height z-scores showed a non-significant trend toward improvement; BMI z-scores remained stable. Mean Δ height and Δ arm span z-score gains were +0.38 ± 0.45 and +0.32 ± 0.48, respectively. A subgroup analysis of 8 patients with vs 17 without prior limb-lengthening surgery showed no significant differences. Regression analyses adjusted for sex, age, BMI z-scores, and surgical history were likely underpowered to detect meaningful predictors of response. There were no serious treatment-related adverse events.ConclusionVosoritide therapy enhanced linear and appendicular growth in a real-world cohort of children with achondroplasia, including post-surgical patients. These findings align with clinical trial data and support the therapy's applicability across diverse clinical profiles.

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

Results from this study6

6 results from studies that gave the peptide. 6 results from people.

01HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, mean height z-score increased from -0.62 ± 1.09 to -0.24 ± 1.20 (P < .001).
In plain wordsIn children with achondroplasia, vosoritide treatment was linked to higher height scores than baseline.
Peptide
Vosoritide
Outcome
IncreaseHeight z-score
Study
n = 25
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · Mean 12.7 monthsAs given in this study (human).
Measurements
Mean height z-score
−0.62 z-score
baseline
SD of height z-score
1.09 z-score
baseline
Mean height z-score
−0.24 z-score
follow-up
SD of height z-score
1.2 z-score
follow-up
Mean height z-score change
0.38 z-score
vosoritide
SD of height z-score change
0.45 z-score
vosoritide
Source sentences
Summary
Mean height z-scores improved from -0.62 ± 1.09 to -0.24 ± 1.20 (P < .001).
2 context sentences(design, population, supporting numbers)
Summary · context
Mean Δ height and Δ arm span z-score gains were +0.38 ± 0.45 and +0.32 ± 0.48, respectively.
Summary · context
This retrospective analysis included 25 children with achondroplasia (17 boys, age range 2.9-14.3 years) treated with vosoritide for a mean duration of 12.7 months at a multidisciplinary center.
Show in the study text Report a problemFND-4223825301
02HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, mean arm span z-score increased from -1.28 ± 0.93 to -0.96 ± 0.91 (P = .007).
In plain wordsIn children with achondroplasia, vosoritide treatment was linked to higher arm span scores than baseline.
Peptide
Vosoritide
Outcome
IncreaseArm span z-score
Study
n = 25
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · Mean 12.7 monthsAs given in this study (human).
Measurements
Mean arm span z-score
−1.28 z-score
baseline
SD of arm span z-score
0.93 z-score
baseline
Mean arm span z-score
−0.96 z-score
follow-up
SD of arm span z-score
0.91 z-score
follow-up
Mean arm span z-score change
0.32 z-score
vosoritide
SD of arm span z-score change
0.48 z-score
vosoritide
Source sentences
Summary
Mean arm span z-score increased from -1.28 ± 0.93 to -0.96 ± 0.91 (P = .007).
2 context sentences(design, population, supporting numbers)
Summary · context
Mean Δ height and Δ arm span z-score gains were +0.38 ± 0.45 and +0.32 ± 0.48, respectively.
Summary · context
This retrospective analysis included 25 children with achondroplasia (17 boys, age range 2.9-14.3 years) treated with vosoritide for a mean duration of 12.7 months at a multidisciplinary center.
Show in the study text Report a problemFND-4223825302
03HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, sitting height z-scores showed a non-significant trend toward improvement versus baseline.
In plain wordsIn children with achondroplasia, sitting height scores showed a non-significant improvement trend during vosoritide treatment versus baseline.
Peptide
Vosoritide
Outcome
No significant changeSitting height z-score
Study
n = 25
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · Mean 12.7 monthsAs given in this study (human).
Source sentences
Summary
Sitting height z-scores showed a non-significant trend toward improvement; BMI z-scores remained stable.
1 context sentence(design, population, supporting numbers)
Summary · context
This retrospective analysis included 25 children with achondroplasia (17 boys, age range 2.9-14.3 years) treated with vosoritide for a mean duration of 12.7 months at a multidisciplinary center.
Show in the study text Report a problemFND-4223825303
04HumanObservational studyEfficacy outcome
In children with achondroplasia receiving vosoritide, BMI z-scores remained stable versus baseline.
In plain wordsIn children with achondroplasia, BMI scores remained stable during vosoritide treatment compared with baseline.
Peptide
Vosoritide
Outcome
No significant changeBMI z-score
Study
n = 25
Comparator
Baseline
Outcome group
Body weight · Metabolic and weight
Studied dose
Dose not stated · Mean 12.7 monthsAs given in this study (human).
Source sentences
Summary
Sitting height z-scores showed a non-significant trend toward improvement; BMI z-scores remained stable.
1 context sentence(design, population, supporting numbers)
Summary · context
This retrospective analysis included 25 children with achondroplasia (17 boys, age range 2.9-14.3 years) treated with vosoritide for a mean duration of 12.7 months at a multidisciplinary center.
Show in the study text Report a problemFND-4223825304
05HumanObservational studyEfficacy outcome
In vosoritide-treated children with achondroplasia, growth response showed no significant difference between 8 with and 17 without prior limb-lengthening surgery.
In plain wordsIn vosoritide-treated children, growth response did not differ significantly between those with and without prior limb-lengthening surgery.
Peptide
Vosoritide
Outcome
No significant changeGrowth response by prior limb-lengthening surgery
Study
n = 25
Comparator
Children without prior limb-lengthening surgery
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · Mean 12.7 monthsAs given in this study (human).
Source sentences
Summary
A subgroup analysis of 8 patients with vs 17 without prior limb-lengthening surgery showed no significant differences.
1 context sentence(design, population, supporting numbers)
Summary · context
This retrospective analysis included 25 children with achondroplasia (17 boys, age range 2.9-14.3 years) treated with vosoritide for a mean duration of 12.7 months at a multidisciplinary center.
Show in the study text Report a problemFND-4223825305
06HumanObservational studySafety outcome
In children with achondroplasia receiving vosoritide, no serious treatment-related adverse events occurred.
In plain wordsIn children with achondroplasia receiving vosoritide, no serious treatment-related adverse events occurred.
Peptide
Vosoritide
Outcome
Not applicableSerious treatment-related adverse events
Study
n = 25
Outcome group
Adverse events · Whole body
Studied dose
Dose not stated · Mean 12.7 monthsAs given in this study (human).
Source sentences
Summary
There were no serious treatment-related adverse events.
1 context sentence(design, population, supporting numbers)
Summary · context
This retrospective analysis included 25 children with achondroplasia (17 boys, age range 2.9-14.3 years) treated with vosoritide for a mean duration of 12.7 months at a multidisciplinary center.
Show in the study text Report a problemFND-4223825306

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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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