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Study · Observational study · n = 57 · 2026

6 results

Real-World Safety and Effectiveness of Vosoritide in Children with Achondroplasia: French Early Access Program.

Cormier-Daire V, Edouard T, Isidor B, Mukherjee S, Pimenta JM, Rossi M, Schaefer E, Sigaudy S, Baujat G

Hormone research in paediatrics25 Jan 2025Cited by 6 (Europe PMC)

PMID39864410 (opens in a new tab)PMCIDPMC13225797 (opens in a new tab)DOI10.1159/000543743 (opens in a new tab)PepBankPUB-39864410
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Summary

The text of this summary is not shown here, because the article license does not allow reuse. Read it at the source (opens in a new tab)

Text rights: CC BY-NC. Results still quote short attributed sentences.

Results from this study6

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

  1. 01HumanObservational studySafety outcome

    In children with achondroplasia receiving vosoritide, all adverse events were mild.

    In plain wordsIn children with achondroplasia receiving vosoritide, all reported adverse events were mild.

    Peptide
    Vosoritide
    Outcome
    Not applicableAdverse event severity
    Study

    n = 57

    Outcome group
    Adverse events · Whole body
    Studied dose
    15 μg/kg once daily · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    All adverse events were mild (mainly injection site reactions) and there were no discontinuations related to vosoritide treatment.
    3 context sentences
    Summary · context
    Participants aged ≥5 years with achondroplasia and open epiphyses were eligible for enrollment in the EAP, conducted by six centers within the French national rare disease reference center for constitutional bone diseases network, from 24 June 2021 to 13 December 2022.
    Summary · context
    Treatment consisted of once-daily subcutaneous vosoritide 15 μg/kg.
    Summary · context
    Among 62 enrolled participants, 57 started treatment with vosoritide within the EAP period with 38 completing at least 6 months and 22 at least 12 months of treatment.
  2. 02HumanObservational studySafety outcome

    In children with achondroplasia receiving vosoritide, adverse events were mainly mild injection site reactions.

    In plain wordsIn children receiving vosoritide, reported adverse events were mainly mild injection site reactions.

    Peptide
    Vosoritide
    Outcome
    Not applicableInjection site reactions
    Study

    n = 57

    Outcome group
    Adverse events · Skin and wound
    Studied dose
    15 μg/kg once daily · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    All adverse events were mild (mainly injection site reactions) and there were no discontinuations related to vosoritide treatment.
    3 context sentences
    Summary · context
    Participants aged ≥5 years with achondroplasia and open epiphyses were eligible for enrollment in the EAP, conducted by six centers within the French national rare disease reference center for constitutional bone diseases network, from 24 June 2021 to 13 December 2022.
    Summary · context
    Treatment consisted of once-daily subcutaneous vosoritide 15 μg/kg.
    Summary · context
    Among 62 enrolled participants, 57 started treatment with vosoritide within the EAP period with 38 completing at least 6 months and 22 at least 12 months of treatment.
  3. 03HumanObservational studySafety outcome

    In children with achondroplasia receiving vosoritide, no discontinuations were related to treatment.

    In plain wordsIn children receiving vosoritide, no participants discontinued treatment for reasons related to vosoritide.

    Peptide
    Vosoritide
    Outcome
    Not applicableTreatment-related discontinuations
    Study

    n = 57

    Outcome group
    Adverse events · Whole body
    Studied dose
    15 μg/kg once daily · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    All adverse events were mild (mainly injection site reactions) and there were no discontinuations related to vosoritide treatment.
    3 context sentences
    Summary · context
    Participants aged ≥5 years with achondroplasia and open epiphyses were eligible for enrollment in the EAP, conducted by six centers within the French national rare disease reference center for constitutional bone diseases network, from 24 June 2021 to 13 December 2022.
    Summary · context
    Treatment consisted of once-daily subcutaneous vosoritide 15 μg/kg.
    Summary · context
    Among 62 enrolled participants, 57 started treatment with vosoritide within the EAP period with 38 completing at least 6 months and 22 at least 12 months of treatment.
  4. 04HumanObservational studyEfficacy outcome

    In children with achondroplasia receiving vosoritide, mean annualized growth velocity was 6.0 cm/year after 12 months.

    In plain wordsIn children with achondroplasia receiving vosoritide, average growth velocity was 6.0 cm/year after 12 months.

    Peptide
    Vosoritide
    Outcome
    Not applicableAnnualized growth velocity
    Study

    n = 22

    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 μg/kg once daily · subcutaneous · 12 monthsAs given in this study (human).

    Measurements

    mean annualized growth velocity
    6 cm/year
    after 12 months of treatment

    Source sentences

    Summary
    After 12 months of treatment, participants achieved a mean AGV of 6.0 cm/year, absolute gain in height of 6.2 cm, and increase in height Z-score referenced to the average stature population of 0.38.
    3 context sentences
    Summary · context
    Participants aged ≥5 years with achondroplasia and open epiphyses were eligible for enrollment in the EAP, conducted by six centers within the French national rare disease reference center for constitutional bone diseases network, from 24 June 2021 to 13 December 2022.
    Summary · context
    Treatment consisted of once-daily subcutaneous vosoritide 15 μg/kg.
    Summary · context
    Among 62 enrolled participants, 57 started treatment with vosoritide within the EAP period with 38 completing at least 6 months and 22 at least 12 months of treatment.
  5. 05HumanObservational studyEfficacy outcome

    In children with achondroplasia, vosoritide treatment was associated with a 6.2 cm absolute height gain after 12 months.

    In plain wordsIn children with achondroplasia, vosoritide treatment was linked to a 6.2 cm height gain after 12 months.

    Peptide
    Vosoritide
    Outcome
    IncreaseAbsolute height gain
    Study

    n = 22

    Comparator
    Baseline
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 μg/kg once daily · subcutaneous · 12 monthsAs given in this study (human).

    Measurements

    absolute height gain
    6.2 cm
    after 12 months of treatment

    Source sentences

    Summary
    After 12 months of treatment, participants achieved a mean AGV of 6.0 cm/year, absolute gain in height of 6.2 cm, and increase in height Z-score referenced to the average stature population of 0.38.
    3 context sentences
    Summary · context
    Participants aged ≥5 years with achondroplasia and open epiphyses were eligible for enrollment in the EAP, conducted by six centers within the French national rare disease reference center for constitutional bone diseases network, from 24 June 2021 to 13 December 2022.
    Summary · context
    Treatment consisted of once-daily subcutaneous vosoritide 15 μg/kg.
    Summary · context
    Among 62 enrolled participants, 57 started treatment with vosoritide within the EAP period with 38 completing at least 6 months and 22 at least 12 months of treatment.
  6. 06HumanObservational studyEfficacy outcome

    In children with achondroplasia, vosoritide treatment was associated with a 0.38 increase in average-stature-referenced height Z-score after 12 months.

    In plain wordsIn children with achondroplasia, vosoritide treatment was linked to a 0.38 increase in height Z-score after 12 months.

    Peptide
    Vosoritide
    Outcome
    IncreaseHeight Z-score
    Study

    n = 22

    Comparator
    Baseline
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 μg/kg once daily · subcutaneous · 12 monthsAs given in this study (human).

    Measurements

    height Z-score increase (average stature population reference)
    0.38
    after 12 months of treatment

    Source sentences

    Summary
    After 12 months of treatment, participants achieved a mean AGV of 6.0 cm/year, absolute gain in height of 6.2 cm, and increase in height Z-score referenced to the average stature population of 0.38.
    3 context sentences
    Summary · context
    Participants aged ≥5 years with achondroplasia and open epiphyses were eligible for enrollment in the EAP, conducted by six centers within the French national rare disease reference center for constitutional bone diseases network, from 24 June 2021 to 13 December 2022.
    Summary · context
    Treatment consisted of once-daily subcutaneous vosoritide 15 μg/kg.
    Summary · context
    Among 62 enrolled participants, 57 started treatment with vosoritide within the EAP period with 38 completing at least 6 months and 22 at least 12 months of treatment.

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.

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