Real-world safety and age-dependent effectiveness of vosoritide in achondroplasia: A single-center retrospective analysis of transition from growth hormone to vosoritide.

Shimada T, Nakayama H, Ueda I, Ishimi T, Yamada C, Nakano Y, Yamamoto K, Fujiwara M, Kitaoka T, Ozono K, Kubota T, Ohata Y, Kitabatake Y

Bone11 Nov 2025

PMID41232919DOI10.1016/j.bone.2025.117722PepBankPUB-41232919

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Summary

Achondroplasia (ACH), a skeletal dysplasia, is characterized by disproportionate short stature and impaired quality of life. In Japan, growth hormone (GH) therapy has been available since 1997, and vosoritide, a C-type natriuretic peptide analog, was approved in 2022. Although clinical trials have demonstrated vosoritide's efficacy, real-world comparative data in Japan are scarce. We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025. Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment. Growth outcomes included annualized growth velocity (AGV) and height standard deviation scores (HT-SDS) calculated using Japanese and ACH-specific (HT-SDS_ACH) references. Group A (median age: 2.10 years) demonstrated significant improvements in HT-SDS_ACH at years 3 (p = 0.0022), 4 (p = 0.0004), 5 (p = 0.007), and 6 (p = 0.0012). Group B (median age: 10.1 years), with a median of 6.10 years of GH exposure, showed significant increases at 1 year (p = 0.047) and 2.5 years (p = 0.0054) after switching. In Group B, HT-SDS_ACH remained stable among patients starting vosoritide before age 4, but improved significantly in those beginning later. Injection site pain occurred in three patients, leading to one discontinuation. Vosoritide improves linear growth in children with ACH, including those previously treated with GH, showing greater benefit when started at ≥4 years, though earlier use may offer advantages.

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

Results from this study10

10 results from studies that gave the peptide. 10 results from people.

01HumanObservational studySafety outcome
In children with ACH receiving vosoritide, injection site pain occurred in three patients.
In plain wordsIn children with achondroplasia receiving vosoritide, three patients reported injection site pain.
Peptide
Vosoritide
Outcome
Not applicableInjection site pain
Study
n = 22
Outcome group
Adverse events · Skin and wound
Studied dose
Dose not stated
Measurements
patients with injection site pain
3 patients
all 22 treated children
Source sentences
Summary
Injection site pain occurred in three patients, leading to one discontinuation.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025.
Summary · context
Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment.
Show in the study text Report a problemFND-4123291909
02HumanObservational studySafety outcome
In children with ACH receiving vosoritide, injection site pain led to one discontinuation.
In plain wordsIn children with achondroplasia receiving vosoritide, one patient stopped because of injection site pain.
Peptide
Vosoritide
Outcome
Not applicableDiscontinuation due to injection site pain
Study
n = 22
Outcome group
Adverse events · Skin and wound
Studied dose
Dose not stated
Measurements
discontinuations due to injection site pain
1 patient
all 22 treated children
Source sentences
Summary
Injection site pain occurred in three patients, leading to one discontinuation.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025.
Summary · context
Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment.
Show in the study text Report a problemFND-4123291910
03HumanObservational studyEfficacy outcome
In GH-naïve children with ACH, vosoritide treatment was associated with improved HT-SDS_ACH at year 3 (p = 0.0022).
In plain wordsIn GH-naïve children with achondroplasia, vosoritide treatment was linked to higher ACH-specific height scores at year three versus baseline.
Peptide
Vosoritide
Outcome
IncreaseGH-naïve HT-SDS_ACH at year 3
Study
n: not reported in source
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Measurements
p-value
0.0022
GH-naive group, HT-SDS_ACH at year 3
Source sentences
Summary
Group A (median age: 2.10 years) demonstrated significant improvements in HT-SDS_ACH at years 3 (p = 0.0022), 4 (p = 0.0004), 5 (p = 0.007), and 6 (p = 0.0012).
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025.
Summary · context
Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment.
Show in the study text Report a problemFND-4123291901
04HumanObservational studyEfficacy outcome
In GH-naïve children with ACH, vosoritide treatment was associated with improved HT-SDS_ACH at year 4 (p = 0.0004).
In plain wordsIn GH-naïve children with achondroplasia, vosoritide treatment was linked to higher ACH-specific height scores at year four versus baseline.
Peptide
Vosoritide
Outcome
IncreaseGH-naïve HT-SDS_ACH at year 4
Study
n: not reported in source
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Measurements
p-value
0.0004
GH-naive group, HT-SDS_ACH at year 4
Source sentences
Summary
Group A (median age: 2.10 years) demonstrated significant improvements in HT-SDS_ACH at years 3 (p = 0.0022), 4 (p = 0.0004), 5 (p = 0.007), and 6 (p = 0.0012).
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025.
Summary · context
Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment.
Show in the study text Report a problemFND-4123291902
05HumanObservational studyEfficacy outcome
In GH-naïve children with ACH, vosoritide treatment was associated with improved HT-SDS_ACH at year 5 (p = 0.007).
In plain wordsIn GH-naïve children with achondroplasia, vosoritide treatment was linked to higher ACH-specific height scores at year five versus baseline.
Peptide
Vosoritide
Outcome
IncreaseGH-naïve HT-SDS_ACH at year 5
Study
n: not reported in source
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Measurements
p-value
0.007
GH-naive group, HT-SDS_ACH at year 5
Source sentences
Summary
Group A (median age: 2.10 years) demonstrated significant improvements in HT-SDS_ACH at years 3 (p = 0.0022), 4 (p = 0.0004), 5 (p = 0.007), and 6 (p = 0.0012).
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025.
Summary · context
Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment.
Show in the study text Report a problemFND-4123291903
06HumanObservational studyEfficacy outcome
In GH-naïve children with ACH, vosoritide treatment was associated with improved HT-SDS_ACH at year 6 (p = 0.0012).
In plain wordsIn GH-naïve children with achondroplasia, vosoritide treatment was linked to higher ACH-specific height scores at year six versus baseline.
Peptide
Vosoritide
Outcome
IncreaseGH-naïve HT-SDS_ACH at year 6
Study
n: not reported in source
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Measurements
p-value
0.0012
GH-naive group, HT-SDS_ACH at year 6
Source sentences
Summary
Group A (median age: 2.10 years) demonstrated significant improvements in HT-SDS_ACH at years 3 (p = 0.0022), 4 (p = 0.0004), 5 (p = 0.007), and 6 (p = 0.0012).
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025.
Summary · context
Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment.
Show in the study text Report a problemFND-4123291904
07HumanObservational studyEfficacy outcome
In children with ACH previously receiving GH, switching to vosoritide was associated with higher HT-SDS_ACH at 1 year (p = 0.047).
In plain wordsIn children with achondroplasia previously receiving GH, switching to vosoritide was linked to higher height scores at one year versus baseline.
Peptide
Vosoritide
Outcome
IncreaseSwitch-group HT-SDS_ACH at year 1
Study
n: not reported in source
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Measurements
p-value
0.047
switch group, HT-SDS_ACH 1 year after switching
Source sentences
Summary
Group B (median age: 10.1 years), with a median of 6.10 years of GH exposure, showed significant increases at 1 year (p = 0.047) and 2.5 years (p = 0.0054) after switching.
3 context sentences(design, population, supporting numbers)
Summary · context
Growth outcomes included annualized growth velocity (AGV) and height standard deviation scores (HT-SDS) calculated using Japanese and ACH-specific (HT-SDS_ACH) references.
Summary · context
We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025.
Summary · context
Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment.
Show in the study text Report a problemFND-4123291905
08HumanObservational studyEfficacy outcome
In children with ACH previously receiving GH, switching to vosoritide was associated with higher HT-SDS_ACH at 2.5 years (p = 0.0054).
In plain wordsIn children with achondroplasia previously receiving GH, switching to vosoritide was linked to higher height scores at 2.5 years versus baseline.
Peptide
Vosoritide
Outcome
IncreaseSwitch-group HT-SDS_ACH at year 2.5
Study
n: not reported in source
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Measurements
p-value
0.0054
switch group, HT-SDS_ACH 2.5 years after switching
Source sentences
Summary
Group B (median age: 10.1 years), with a median of 6.10 years of GH exposure, showed significant increases at 1 year (p = 0.047) and 2.5 years (p = 0.0054) after switching.
3 context sentences(design, population, supporting numbers)
Summary · context
Growth outcomes included annualized growth velocity (AGV) and height standard deviation scores (HT-SDS) calculated using Japanese and ACH-specific (HT-SDS_ACH) references.
Summary · context
We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025.
Summary · context
Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment.
Show in the study text Report a problemFND-4123291906
09HumanObservational studyEfficacy outcome
In children with ACH previously receiving GH, HT-SDS_ACH remained stable when vosoritide started before age 4.
In plain wordsIn children with achondroplasia previously receiving GH, height scores remained stable versus baseline when vosoritide started before age four.
Peptide
Vosoritide
Outcome
No significant changeSwitch-group HT-SDS_ACH starting before age 4
Study
n: not reported in source
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Source sentences
Summary
In Group B, HT-SDS_ACH remained stable among patients starting vosoritide before age 4, but improved significantly in those beginning later.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025.
Summary · context
Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment.
Show in the study text Report a problemFND-4123291907
10HumanObservational studyEfficacy outcome
In children with ACH previously receiving GH, HT-SDS_ACH improved significantly in those who started vosoritide at age 4 or later.
In plain wordsIn children with achondroplasia previously receiving GH, height scores improved versus baseline among those who started vosoritide at age four or later.
Peptide
Vosoritide
Outcome
IncreaseSwitch-group HT-SDS_ACH starting at age 4 or later
Study
n: not reported in source
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated
Source sentences
Summary
In Group B, HT-SDS_ACH remained stable among patients starting vosoritide before age 4, but improved significantly in those beginning later.
2 context sentences(design, population, supporting numbers)
Summary · context
We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025.
Summary · context
Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment.
Show in the study text Report a problemFND-4123291908

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

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.

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Real-world safety and age-dependent effectiveness of vosoritide in achondroplasia: A single-center retrospective analysis of transition from growth hormone to vosoritide.

Summary

Results from this study10

Version history

Subjects

Publication types

MeSH terms

Keywords