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Study · Non-randomized trial · n = 60 · 2022

16 results

Pharmacokinetics and Exposure-Response of Vosoritide in Children with Achondroplasia.

Chan ML, Qi Y, Larimore K, Cherukuri A, Seid L, Jayaram K, Jeha G, Fisheleva E, Day J, Huntsman-Labed A, Savarirayan R, Irving M and 7 more

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Bacino CA, Hoover-Fong J, Ozono K, Mohnike K, Wilcox WR, Horton WA, Henshaw J

Clinical pharmacokinetics25 Aug 2021Cited by 30 (Europe PMC)

PMID34431071 (opens in a new tab)PMCIDPMC8813707 (opens in a new tab)DOI10.1007/s40262-021-01059-1 (opens in a new tab)PepBankPUB-34431071
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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 study16

16 results from studies that gave the peptide. 16 results from people.

  1. 01HumanNon-randomized trialPharmacokinetics

    In children with achondroplasia, vosoritide had a median time to maximal plasma concentration of 15 minutes.

    In plain wordsIn children with achondroplasia, vosoritide reached its maximal plasma concentration after a median of 15 minutes.

    Peptide
    Vosoritide
    Outcome
    Not applicableTime to maximal concentration
    Study

    n = 60

    Outcome group
    Pharmacokinetics · Pharmacology
    Studied dose
    15 μg/kg · subcutaneousAs given in this study (human).

    Measurements

    Median Tmax
    15 minutes
    Phase III study, 15 μg/kg subcutaneous

    Source sentences

    Summary
    In the phase III study, following subcutaneous administration at the recommended dose of 15 μg/kg to patients with achondroplasia aged 5-18 years, vosoritide was rapidly absorbed with a median time to maximal plasma concentration (Cmax) of 15 minutes,
  2. 02HumanNon-randomized trialPharmacokinetics

    In children with achondroplasia, vosoritide had a mean half-life of 27.9 minutes after 52 weeks of treatment.

    In plain wordsIn children with achondroplasia, vosoritide had a mean half-life of 27.9 minutes after 52 weeks of treatment.

    Peptide
    Vosoritide
    Outcome
    Not applicableElimination half-life
    Study

    n = 60

    Outcome group
    Pharmacokinetics · Pharmacology
    Studied dose
    15 μg/kg · subcutaneous · 52 weeksAs given in this study (human).

    Measurements

    Mean half-life
    27.9 minutes
    After 52 weeks

    Source sentences

    Summary
    and cleared with a mean half-life of 27.9 minutes after 52 weeks of treatment.
  3. 03HumanNon-randomized trialSafety outcome

    In children with achondroplasia, 25 of 60 vosoritide-treated patients developed total anti-vosoritide antibody responses (42%).

    In plain wordsIn children with achondroplasia, 25 of 60 vosoritide-treated patients developed antibody responses (42%).

    Peptide
    Vosoritide
    Outcome
    Not applicableAnti-vosoritide antibodies
    Study

    n = 60

    Outcome group
    Immune response · Immune and inflammation
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Measurements

    Patients with total anti-vosoritide antibody responses
    25 patients
    Of 60 treated patients, phase III study
    Patients with total anti-vosoritide antibody responses
    42%
    Phase III treated patients

    Source sentences

    Summary
    Total anti-vosoritide antibody (TAb) responses were detected in the serum of 25 of 60 (42%) treated patients in the phase III study, with no apparent impact of TAb development noted on annualized growth velocity or vosoritide exposure.
  4. 04HumanNon-randomized trialPharmacokinetics

    In children with achondroplasia, vosoritide Cmax was consistent across visits.

    In plain wordsIn children with achondroplasia, vosoritide peak concentrations were consistent across visits.

    Peptide
    Vosoritide
    Outcome
    Direction not reportedPeak concentration across visits
    Study

    n: not reported in source

    Outcome group
    Pharmacokinetics · Pharmacology
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Vosoritide exposure (Cmax and area under the concentration-time curve [AUC]) was consistent across visits.
  5. 05HumanNon-randomized trialPharmacokinetics

    In children with achondroplasia, once-daily vosoritide showed no evidence of accumulation.

    In plain wordsIn children with achondroplasia, once-daily vosoritide showed no evidence of accumulation.

    Peptide
    Vosoritide
    Outcome
    No significant changeAccumulation
    Study

    n: not reported in source

    Outcome group
    Pharmacokinetics · Pharmacology
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    No evidence of accumulation with once-daily dosing was observed.
  6. 06HumanNon-randomized trialPharmacokinetics

    In children with achondroplasia, vosoritide area under the concentration-time curve was consistent across visits.

    In plain wordsIn children with achondroplasia, total vosoritide exposure was consistent across visits.

    Peptide
    Vosoritide
    Outcome
    Direction not reportedTotal exposure across visits
    Study

    n: not reported in source

    Outcome group
    Pharmacokinetics · Pharmacology
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Vosoritide exposure (Cmax and area under the concentration-time curve [AUC]) was consistent across visits.
  7. 07HumanObservational studyEfficacy outcome

    In children with achondroplasia, the vosoritide exposure-response relationship for growth velocity saturated at 15 μg/kg.

    In plain wordsIn children with achondroplasia, the growth velocity response to vosoritide exposure saturated at 15 μg/kg.

    Peptide
    Vosoritide
    Outcome
    Not applicableGrowth velocity exposure-response saturation
    Study

    n: not reported in source

    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 μg/kg · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    The exposure-response relationships for changes in both annualized growth velocity and the CXM biomarker saturated at 15 μg/kg, while systemic pharmacological activity, as measured by urinary cGMP, was near maximal or saturated at exposures obtained at the highest dose studied (i.e. 30 μg/kg).
  8. 08HumanObservational studyActivity measurement

    In children with achondroplasia, the vosoritide exposure-response relationship for CXM saturated at 15 μg/kg.

    In plain wordsIn children with achondroplasia, the CXM response to vosoritide exposure saturated at 15 μg/kg.

    Peptide
    Vosoritide
    Outcome
    Not applicableCXM exposure-response saturation
    Study

    n: not reported in source

    Outcome group
    Pharmacokinetics · Pharmacology
    Studied dose
    15 μg/kg · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    The exposure-response relationships for changes in both annualized growth velocity and the CXM biomarker saturated at 15 μg/kg, while systemic pharmacological activity, as measured by urinary cGMP, was near maximal or saturated at exposures obtained at the highest dose studied (i.e. 30 μg/kg).
  9. 09HumanObservational studyActivity measurement

    In children with achondroplasia, urinary cGMP activity was near maximal or saturated at vosoritide exposures obtained at 30 μg/kg.

    In plain wordsIn children with achondroplasia, urinary cGMP activity approached or reached saturation at vosoritide exposures obtained at 30 μg/kg.

    Peptide
    Vosoritide
    Outcome
    Not applicableUrinary cGMP response saturation
    Study

    n: not reported in source

    Outcome group
    Pharmacokinetics · Pharmacology
    Studied dose
    30 μg/kg · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    The exposure-response relationships for changes in both annualized growth velocity and the CXM biomarker saturated at 15 μg/kg, while systemic pharmacological activity, as measured by urinary cGMP, was near maximal or saturated at exposures obtained at the highest dose studied (i.e. 30 μg/kg).
  10. 10HumanObservational studyEfficacy outcome

    In children with achondroplasia receiving vosoritide, antibody development showed no apparent impact on annualized growth velocity.

    In plain wordsIn children receiving vosoritide, antibody development showed no apparent impact on growth velocity.

    Peptide
    Vosoritide
    Outcome
    No significant changeAntibody impact on growth velocity
    Study

    n: not reported in source

    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Total anti-vosoritide antibody (TAb) responses were detected in the serum of 25 of 60 (42%) treated patients in the phase III study, with no apparent impact of TAb development noted on annualized growth velocity or vosoritide exposure.
  11. 11HumanObservational studyPharmacokinetics

    In children with achondroplasia receiving vosoritide, antibody development showed no apparent impact on vosoritide exposure.

    In plain wordsIn children receiving vosoritide, antibody development showed no apparent impact on drug exposure.

    Peptide
    Vosoritide
    Outcome
    No significant changeAntibody impact on exposure
    Study

    n: not reported in source

    Outcome group
    Pharmacokinetics · Pharmacology
    Studied dose
    Dose not stated · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Total anti-vosoritide antibody (TAb) responses were detected in the serum of 25 of 60 (42%) treated patients in the phase III study, with no apparent impact of TAb development noted on annualized growth velocity or vosoritide exposure.
  12. 12HumanObservational studyEfficacy outcome

    In children with achondroplasia, vosoritide exposure showed no meaningful correlation with changes in annualized growth velocity.

    In plain wordsIn children with achondroplasia, vosoritide exposure showed no meaningful link to changes in growth velocity.

    Peptide
    Vosoritide
    Outcome
    Direction not reportedExposure association with growth velocity
    Study

    n: not reported in source

    Outcome group
    Pharmacokinetics · Pharmacology
    Studied dose
    15 µg/kg · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Across the exposure range obtained with 15 µg/kg in the phase III study, no meaningful correlations between vosoritide plasma exposure and changes in annualized growth velocity or CXM, or changes from predose heart rate, and systolic or diastolic blood pressures were observed.
  13. 13HumanObservational studyActivity measurement

    In children with achondroplasia, vosoritide exposure showed no meaningful correlation with changes in CXM.

    In plain wordsIn children with achondroplasia, vosoritide exposure showed no meaningful link to changes in CXM.

    Peptide
    Vosoritide
    Outcome
    Direction not reportedExposure association with CXM
    Study

    n: not reported in source

    Outcome group
    Pharmacokinetics · Pharmacology
    Studied dose
    15 µg/kg · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Across the exposure range obtained with 15 µg/kg in the phase III study, no meaningful correlations between vosoritide plasma exposure and changes in annualized growth velocity or CXM, or changes from predose heart rate, and systolic or diastolic blood pressures were observed.
  14. 14HumanObservational studySafety outcome

    In children with achondroplasia, vosoritide exposure showed no meaningful correlation with changes from predose heart rate.

    In plain wordsIn children with achondroplasia, vosoritide exposure showed no meaningful link to heart rate changes from predose values.

    Peptide
    Vosoritide
    Outcome
    Direction not reportedExposure association with heart rate
    Study

    n: not reported in source

    Outcome group
    Heart function · Cardiovascular
    Studied dose
    15 µg/kg · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Across the exposure range obtained with 15 µg/kg in the phase III study, no meaningful correlations between vosoritide plasma exposure and changes in annualized growth velocity or CXM, or changes from predose heart rate, and systolic or diastolic blood pressures were observed.
  15. 15HumanObservational studySafety outcome

    In children with achondroplasia, vosoritide exposure showed no meaningful correlation with systolic blood pressure changes.

    In plain wordsIn children with achondroplasia, vosoritide exposure showed no meaningful link to systolic blood pressure changes.

    Peptide
    Vosoritide
    Outcome
    Direction not reportedExposure association with systolic blood pressure
    Study

    n: not reported in source

    Outcome group
    Blood pressure · Cardiovascular
    Studied dose
    15 µg/kg · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Across the exposure range obtained with 15 µg/kg in the phase III study, no meaningful correlations between vosoritide plasma exposure and changes in annualized growth velocity or CXM, or changes from predose heart rate, and systolic or diastolic blood pressures were observed.
  16. 16HumanObservational studySafety outcome

    In children with achondroplasia, vosoritide exposure showed no meaningful correlation with diastolic blood pressure changes.

    In plain wordsIn children with achondroplasia, vosoritide exposure showed no meaningful link to diastolic blood pressure changes.

    Peptide
    Vosoritide
    Outcome
    Direction not reportedExposure association with diastolic blood pressure
    Study

    n: not reported in source

    Outcome group
    Blood pressure · Cardiovascular
    Studied dose
    15 µg/kg · subcutaneousAs given in this study (human).

    Source sentences

    Summary
    Across the exposure range obtained with 15 µg/kg in the phase III study, no meaningful correlations between vosoritide plasma exposure and changes in annualized growth velocity or CXM, or changes from predose heart rate, and systolic or diastolic blood pressures were observed.

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

Registered trials2

Trials this study reports on, or that list this study as a reference. Registry data from ClinicalTrials.gov.

2 trials · 2 completed · 2 with posted results · source ClinicalTrials.gov (opens in a new tab)

CompletedPhase 3Results postedNCT03197766

A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia

BioMarin Pharmaceuticaln = 12112 Dec 2016 to 30 Oct 2019

Achondroplasia

Trial pageClinicalTrials.govUpdated 2 Mar 2022
CompletedPhase 2Results postedNCT02055157

A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With Achondroplasia

BioMarin Pharmaceuticaln = 3513 Jan 2014 to 2 Oct 2017

Achondroplasia

Trial pageClinicalTrials.govUpdated 15 Jan 2021

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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