Skip to content

Study · Observational study · 2026

5 results

Evaluating the impact of vosoritide on complications of achondroplasia.

Savarirayan R, Hecht JT, Jayaram K, Ingole S, Dee A, Hoover-Fong J

Genetics in medicine : official journal of the American College of Medical Genetics17 Apr 2026

PMID42001280 (opens in a new tab)DOI10.1016/j.gim.2026.102584 (opens in a new tab)PepBankPUB-42001280
Publisher pagePubMedAsk about this study
Jump to 5 results

Summary

PurposeImpaired endochondral bone growth in people with achondroplasia causes complications that impair function, cause pain, require surgery, and contribute to mortality. Rates of complications in participants with achondroplasia enrolled in clinical trials of vosoritide, a targeted treatment, were analyzed and compared with published natural history (NH) rates.MethodsPrevalence (medical history + new events post-treatment initiation) and incidence (new/worsening events posttreatment initiation) were calculated for complications across 6 clinical trials (2.5-30 μg/kg/day vosoritide) in children with achondroplasia, stratified by age at treatment initiation (<5 years/≥5 years). Complication-related procedures and mortality rates were examined.ResultsBy August 2024, 230 participants received vosoritide (68 aged <5 years at treatment initiation [exposure, 289.1 person-years]; 162 aged ≥5 years at treatment initiation [exposure, 834.5 person-years]). Prevalence for most complications was consistent with or lower than NH rates. Incidence of leg malalignment; foramen magnum stenosis; spinal stenosis; kyphosis; lordosis; hydrocephalus; ear, nose, and throat complications; and pain was generally lower than NH rates. Foramen magnum decompression frequency was 3.0% overall (5.9% in participants who initiated treatment aged <2 years). Crude mortality was lower than NH rates.ConclusionVosoritide was not associated with worsening of complications in children with achondroplasia and may reduce their frequency.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study5

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

  1. 01HumanObservational studySafety outcome

    In vosoritide trial participants with achondroplasia, prevalence including medical history and new post-treatment events was generally consistent with or below natural history rates.

    In plain wordsIn vosoritide trial participants, complication prevalence included past medical history and new events, and was generally similar to or below natural history rates.

    Peptide
    Vosoritide
    Outcome
    MixedComplication prevalence
    Study

    n: not reported in source

    Comparator
    Published natural history rates
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    2.5-30 μg/kg/dayAs given in this study (human).

    Source sentences

    Summary
    Prevalence for most complications was consistent with or lower than NH rates.
    2 context sentences
    Summary · context
    Rates of complications in participants with achondroplasia enrolled in clinical trials of vosoritide, a targeted treatment, were analyzed and compared with published natural history (NH) rates.
    Summary · context
    Prevalence (medical history + new events post-treatment initiation) and incidence (new/worsening events posttreatment initiation) were calculated for complications across 6 clinical trials (2.5-30 μg/kg/day vosoritide) in children with achondroplasia, stratified by age at treatment initiation (<5 years/≥5 years).
  2. 02HumanObservational studySafety outcome

    In vosoritide-treated children with achondroplasia, incidence of leg malalignment, foramen magnum stenosis, spinal stenosis, kyphosis, lordosis, hydrocephalus, ENT complications and pain was generally below natural history rates.

    In plain wordsIn vosoritide-treated children, bone, spine, skull opening, hydrocephalus, ear, nose, throat and pain complications generally occurred less often than natural history rates.

    Peptide
    Vosoritide
    Outcome
    DecreaseComplication incidence
    Study

    n: not reported in source

    Comparator
    Published natural history rates
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    2.5-30 μg/kg/dayAs given in this study (human).

    Source sentences

    Summary
    Incidence of leg malalignment; foramen magnum stenosis; spinal stenosis; kyphosis; lordosis; hydrocephalus; ear, nose, and throat complications; and pain was generally lower than NH rates.
    2 context sentences
    Summary · context
    Rates of complications in participants with achondroplasia enrolled in clinical trials of vosoritide, a targeted treatment, were analyzed and compared with published natural history (NH) rates.
    Summary · context
    Prevalence (medical history + new events post-treatment initiation) and incidence (new/worsening events posttreatment initiation) were calculated for complications across 6 clinical trials (2.5-30 μg/kg/day vosoritide) in children with achondroplasia, stratified by age at treatment initiation (<5 years/≥5 years).
  3. 03HumanObservational studySafety outcome

    In children with achondroplasia receiving vosoritide, foramen magnum decompression frequency was 3.0% overall.

    In plain wordsIn children with achondroplasia receiving vosoritide, foramen magnum decompression frequency was 3.0% overall.

    Peptide
    Vosoritide
    Outcome
    Not applicableForamen magnum decompression, overall
    Study

    n: not reported in source

    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    2.5-30 μg/kg/dayAs given in this study (human).

    Measurements

    Foramen magnum decompression frequency
    3%
    overall

    Source sentences

    Summary
    Foramen magnum decompression frequency was 3.0% overall (5.9% in participants who initiated treatment aged <2 years).
    2 context sentences
    Summary · context
    Rates of complications in participants with achondroplasia enrolled in clinical trials of vosoritide, a targeted treatment, were analyzed and compared with published natural history (NH) rates.
    Summary · context
    Prevalence (medical history + new events post-treatment initiation) and incidence (new/worsening events posttreatment initiation) were calculated for complications across 6 clinical trials (2.5-30 μg/kg/day vosoritide) in children with achondroplasia, stratified by age at treatment initiation (<5 years/≥5 years).
  4. 04HumanObservational studySafety outcome

    In children who initiated vosoritide aged <2 years, foramen magnum decompression frequency was 5.9%.

    In plain wordsIn children who started vosoritide before age 2, foramen magnum decompression frequency was 5.9%.

    Peptide
    Vosoritide
    Outcome
    Not applicableForamen magnum decompression, initiation before age two
    Study

    n: not reported in source

    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    2.5-30 μg/kg/dayAs given in this study (human).

    Measurements

    Foramen magnum decompression frequency
    5.9%
    initiated treatment aged <2 years

    Source sentences

    Summary
    Foramen magnum decompression frequency was 3.0% overall (5.9% in participants who initiated treatment aged <2 years).
    2 context sentences
    Summary · context
    Rates of complications in participants with achondroplasia enrolled in clinical trials of vosoritide, a targeted treatment, were analyzed and compared with published natural history (NH) rates.
    Summary · context
    Prevalence (medical history + new events post-treatment initiation) and incidence (new/worsening events posttreatment initiation) were calculated for complications across 6 clinical trials (2.5-30 μg/kg/day vosoritide) in children with achondroplasia, stratified by age at treatment initiation (<5 years/≥5 years).
  5. 05HumanObservational studySafety outcome

    In children with achondroplasia, vosoritide exposure was linked to lower crude mortality than published natural history rates.

    In plain wordsIn children with achondroplasia, vosoritide exposure was linked to lower crude mortality than natural history rates.

    Peptide
    Vosoritide
    Outcome
    DecreaseCrude mortality
    Study

    n: not reported in source

    Comparator
    Published natural history rates
    Outcome group
    Mortality · Whole body
    Studied dose
    2.5-30 μg/kg/dayAs given in this study (human).

    Source sentences

    Summary
    Crude mortality was lower than NH rates.
    2 context sentences
    Summary · context
    Rates of complications in participants with achondroplasia enrolled in clinical trials of vosoritide, a targeted treatment, were analyzed and compared with published natural history (NH) rates.
    Summary · context
    Prevalence (medical history + new events post-treatment initiation) and incidence (new/worsening events posttreatment initiation) were calculated for complications across 6 clinical trials (2.5-30 μg/kg/day vosoritide) in children with achondroplasia, stratified by age at treatment initiation (<5 years/≥5 years).

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.

  1. Version 2Current

    28 Oct 2026, 02:03 UTC

    Study results updated

  2. Version 1

    27 Oct 2026, 16:37 UTC

    First added · source update from Europe PMC

Subjects

Terms as the source lists them.

PepBank is a research reference. Talk to a doctor before you use any peptide.