Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study.

Savarirayan R, Tofts L, Irving M, Wilcox WR, Bacino CA, Hoover-Fong J, Font RU, Harmatz P, Rutsch F, Bober MB, Polgreen LE, Ginebreda I and 19 more

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Mohnike K, Charrow J, Hoernschemeyer D, Ozono K, Alanay Y, Arundel P, Kotani Y, Yasui N, White KK, Saal HM, Leiva-Gea A, Luna-González F, Mochizuki H, Basel D, Porco DM, Jayaram K, Fisheleva E, Huntsman-Labed A, Day JRS

Genetics in medicine : official journal of the American College of Medical Genetics2 Aug 2021Cited by 77 (Europe PMC)

PMID34341520PMCIDPMC8327889DOI10.1038/s41436-021-01287-7PepBankPUB-34341520

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Summary

PurposeAchondroplasia is caused by pathogenic variants in the fibroblast growth factor receptor 3 gene that lead to impaired endochondral ossification. Vosoritide, an analog of C-type natriuretic peptide, stimulates endochondral bone growth and is in development for the treatment of achondroplasia. This phase 3 extension study was conducted to document the efficacy and safety of continuous, daily vosoritide treatment in children with achondroplasia, and the two-year results are reported.MethodsAfter completing at least six months of a baseline observational growth study, and 52 weeks in a double-blind, placebo-controlled study, participants were eligible to continue treatment in an open-label extension study, where all participants received vosoritide at a dose of 15.0 μg/kg/day.ResultsIn children randomized to vosoritide, annualized growth velocity increased from 4.26 cm/year at baseline to 5.39 cm/year at 52 weeks and 5.52 cm/year at week 104. In children who crossed over from placebo to vosoritide in the extension study, annualized growth velocity increased from 3.81 cm/year at week 52 to 5.43 cm/year at week 104. No new adverse effects of vosoritide were detected.ConclusionVosoritide treatment has safe and persistent growth-promoting effects in children with achondroplasia treated daily for two years.

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

Results from this study4

4 results from studies that gave the peptide. 4 results from people.

01HumanNon-randomized trialEfficacy outcome
In children with achondroplasia receiving vosoritide, annualized growth velocity increased from 4.26 cm/year at baseline to 5.39 cm/year at 52 weeks.
In plain wordsIn children receiving vosoritide, growth velocity increased from 4.26 cm/year at baseline to 5.39 cm/year at 52 weeks.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity at 52 weeks
Study
n: not reported in source
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 52 weeksAs given in this study (human).
Measurements
Annualized growth velocity
4.26 cm/year
Baseline, children randomized to vosoritide
Annualized growth velocity
5.39 cm/year
Week 52, children randomized to vosoritide
Source sentences
Summary
In children randomized to vosoritide, annualized growth velocity increased from 4.26 cm/year at baseline to 5.39 cm/year at 52 weeks and 5.52 cm/year at week 104.
1 context sentence(design, population, supporting numbers)
Summary · context
After completing at least six months of a baseline observational growth study, and 52 weeks in a double-blind, placebo-controlled study, participants were eligible to continue treatment in an open-label extension study, where all participants received vosoritide at a dose of 15.0 μg/kg/day.
Show in the study text Report a problemFND-3434152001
02HumanNon-randomized trialEfficacy outcome
In children with achondroplasia receiving vosoritide, annualized growth velocity increased from 4.26 cm/year at baseline to 5.52 cm/year at week 104.
In plain wordsIn children receiving vosoritide, growth velocity increased from 4.26 cm/year at baseline to 5.52 cm/year at week 104.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity at 104 weeks
Study
n: not reported in source
Comparator
Baseline
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15.0 μg/kg/day · two yearsAs given in this study (human).
Measurements
Annualized growth velocity
4.26 cm/year
Baseline, children randomized to vosoritide
Annualized growth velocity
5.52 cm/year
Week 104, children randomized to vosoritide
Source sentences
Summary
In children randomized to vosoritide, annualized growth velocity increased from 4.26 cm/year at baseline to 5.39 cm/year at 52 weeks and 5.52 cm/year at week 104.
1 context sentence(design, population, supporting numbers)
Summary · context
After completing at least six months of a baseline observational growth study, and 52 weeks in a double-blind, placebo-controlled study, participants were eligible to continue treatment in an open-label extension study, where all participants received vosoritide at a dose of 15.0 μg/kg/day.
Show in the study text Report a problemFND-3434152002
03HumanNon-randomized trialEfficacy outcome
In children crossing from placebo to vosoritide, annualized growth velocity increased from 3.81 cm/year at week 52 to 5.43 cm/year at week 104.
In plain wordsIn children crossing to vosoritide, growth velocity increased from 3.81 cm/year at week 52 to 5.43 cm/year at week 104.
Peptide
Vosoritide
Outcome
IncreaseAnnualized growth velocity after placebo crossover
Study
n: not reported in source
Comparator
Week 52 before crossover
Outcome group
Bone and joint · Musculoskeletal
Studied dose
15.0 μg/kg/dayAs given in this study (human).
Measurements
Annualized growth velocity
3.81 cm/year
Week 52, crossover from placebo
Annualized growth velocity
5.43 cm/year
Week 104, crossover from placebo
Source sentences
Summary
In children who crossed over from placebo to vosoritide in the extension study, annualized growth velocity increased from 3.81 cm/year at week 52 to 5.43 cm/year at week 104.
1 context sentence(design, population, supporting numbers)
Summary · context
After completing at least six months of a baseline observational growth study, and 52 weeks in a double-blind, placebo-controlled study, participants were eligible to continue treatment in an open-label extension study, where all participants received vosoritide at a dose of 15.0 μg/kg/day.
Show in the study text Report a problemFND-3434152003
04HumanNon-randomized trialSafety outcome
In children with achondroplasia receiving vosoritide, no new adverse effects were detected.
In plain wordsIn children with achondroplasia receiving vosoritide, no new adverse effects were detected.
Peptide
Vosoritide
Outcome
Not applicableNew adverse effects
Study
n: not reported in source
Outcome group
Adverse events · Whole body
Studied dose
15.0 μg/kg/dayAs given in this study (human).
Source sentences
Summary
No new adverse effects of vosoritide were detected.
1 context sentence(design, population, supporting numbers)
Summary · context
After completing at least six months of a baseline observational growth study, and 52 weeks in a double-blind, placebo-controlled study, participants were eligible to continue treatment in an open-label extension study, where all participants received vosoritide at a dose of 15.0 μg/kg/day.
Show in the study text Report a problemFND-3434152004

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

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