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Study · Randomized controlled trial · n = 57 · 2026

7 results

Effect of vosoritide on genu varum in children with achondroplasia after 1 year in randomized placebo-controlled trials.

White KK, Irving M, Mukherjee S, Rivat C, Sabir I, Dee A, Savarirayan R

Journal of the Endocrine Society6 Feb 2026Cited by 1 (Europe PMC)

PMID41766933 (opens in a new tab)PMCIDPMC12946844 (opens in a new tab)DOI10.1210/jendso/bvag024 (opens in a new tab)PepBankPUB-41766933
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Summary

Achondroplasia is a skeletal dysplasia associated with multisystem complications including genu varum, which causes pain and limits mobility. Vosoritide, a targeted treatment for achondroplasia, improves growth in children and has an established safety profile, but its effect on genu varum is unclear. Data were collected from 183 participants from randomized, double-blind, placebo-controlled phase 2 (CANOPY ACH-2I [111-206; NCT03583697]) and phase 3 (CANOPY ACH-3 [111-301; NCT03197766]) studies evaluating vosoritide (weight-based dose of 15 or 30 µg/kg/day) in children aged 0-5 and >5 years, respectively. Anterior/posterior lower limb radiographs were taken at baseline and 1 year to measure parameters associated with genu varum. Differences in least-squares mean (LSM) change from baseline were calculated for vosoritide vs placebo using an analysis of covariance model. In both studies, age at treatment initiation and sex distribution were generally balanced between vosoritide and placebo (except 58% males received vosoritide in CANOPY ACH-2I). After 1 year of vosoritide treatment vs placebo, tibial bowing decreased in children who initiated treatment aged n = 40) and remained stable in those ≥5 years (n = 57). Vosoritide-treated participants with evidence of abnormal fibular growth at baseline had reduced overall and distal fibular overgrowth vs placebo. Children aged ≥5 years improved in ankle joint to distal fibula physis distance (difference in LSM change from baseline, -0.07, P = .010) and fibula/tibia ratio (-0.025, P < .0001), and children aged 2-5 years had improved fibula/tibia ratio (-0.033, P = .0032). Preliminary results suggest vosoritide may improve or limit genu varum in children with achondroplasia.
CC BYText from Europe PMC. Highlighted sentences are the source of results.

Results from this study7

7 results from studies that gave the peptide. 7 results from people.

  1. 01HumanRandomized controlled trialEfficacy outcome

    In children with achondroplasia aged ≥5 years at initiation, tibial bowing remained stable with vosoritide versus placebo after 1 year.

    In plain wordsIn older children with achondroplasia, tibial bowing remained stable with vosoritide compared with placebo.

    Peptide
    Vosoritide
    Outcome
    No significant changeTibial bowing, age five years or older
    Study

    n = 57

    Comparator
    Placebo
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 or 30 µg/kg/day · 1 yearAs given in this study (human).

    Source sentences

    Summary
    After 1 year of vosoritide treatment vs placebo, tibial bowing decreased in children who initiated treatment aged n = 40) and remained stable in those ≥5 years (n = 57).
    2 context sentences
    Summary · context
    Data were collected from 183 participants from randomized, double-blind, placebo-controlled phase 2 (CANOPY ACH-2I [111-206; NCT03583697]) and phase 3 (CANOPY ACH-3 [111-301; NCT03197766]) studies evaluating vosoritide (weight-based dose of 15 or 30 µg/kg/day) in children aged 0-5 and >5 years, respectively.
    Summary · context
    Differences in least-squares mean (LSM) change from baseline were calculated for vosoritide vs placebo using an analysis of covariance model.
  2. 02HumanRandomized controlled trialEfficacy outcome

    In children with achondroplasia aged <5 years at initiation, vosoritide reduced tibial bowing versus placebo after 1 year.

    In plain wordsIn younger children with achondroplasia, vosoritide reduced tibial bowing compared with placebo.

    Peptide
    Vosoritide
    Outcome
    DecreaseTibial bowing, age below five years
    Study

    n = 40

    Comparator
    Placebo
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 or 30 µg/kg/day · 1 yearAs given in this study (human).

    Source sentences

    Summary
    After 1 year of vosoritide treatment vs placebo, tibial bowing decreased in children who initiated treatment aged n = 40) and remained stable in those ≥5 years (n = 57).
    2 context sentences
    Summary · context
    Data were collected from 183 participants from randomized, double-blind, placebo-controlled phase 2 (CANOPY ACH-2I [111-206; NCT03583697]) and phase 3 (CANOPY ACH-3 [111-301; NCT03197766]) studies evaluating vosoritide (weight-based dose of 15 or 30 µg/kg/day) in children aged 0-5 and >5 years, respectively.
    Summary · context
    Differences in least-squares mean (LSM) change from baseline were calculated for vosoritide vs placebo using an analysis of covariance model.
  3. 03HumanRandomized controlled trialEfficacy outcome

    In children with abnormal baseline fibular growth, vosoritide reduced overall fibular overgrowth versus placebo.

    In plain wordsIn children with abnormal baseline fibular growth, vosoritide reduced overall fibular overgrowth compared with placebo.

    Peptide
    Vosoritide
    Outcome
    DecreaseOverall fibular overgrowth
    Study

    n: not reported in source

    Comparator
    Placebo
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 or 30 µg/kg/day · 1 yearAs given in this study (human).

    Source sentences

    Summary
    Vosoritide-treated participants with evidence of abnormal fibular growth at baseline had reduced overall and distal fibular overgrowth vs placebo.
    2 context sentences
    Summary · context
    Data were collected from 183 participants from randomized, double-blind, placebo-controlled phase 2 (CANOPY ACH-2I [111-206; NCT03583697]) and phase 3 (CANOPY ACH-3 [111-301; NCT03197766]) studies evaluating vosoritide (weight-based dose of 15 or 30 µg/kg/day) in children aged 0-5 and >5 years, respectively.
    Summary · context
    Differences in least-squares mean (LSM) change from baseline were calculated for vosoritide vs placebo using an analysis of covariance model.
  4. 04HumanRandomized controlled trialEfficacy outcome

    In children with abnormal baseline fibular growth, vosoritide reduced distal fibular overgrowth versus placebo.

    In plain wordsIn children with abnormal baseline fibular growth, vosoritide reduced distal fibular overgrowth compared with placebo.

    Peptide
    Vosoritide
    Outcome
    DecreaseDistal fibular overgrowth
    Study

    n: not reported in source

    Comparator
    Placebo
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 or 30 µg/kg/day · 1 yearAs given in this study (human).

    Source sentences

    Summary
    Vosoritide-treated participants with evidence of abnormal fibular growth at baseline had reduced overall and distal fibular overgrowth vs placebo.
    2 context sentences
    Summary · context
    Data were collected from 183 participants from randomized, double-blind, placebo-controlled phase 2 (CANOPY ACH-2I [111-206; NCT03583697]) and phase 3 (CANOPY ACH-3 [111-301; NCT03197766]) studies evaluating vosoritide (weight-based dose of 15 or 30 µg/kg/day) in children aged 0-5 and >5 years, respectively.
    Summary · context
    Differences in least-squares mean (LSM) change from baseline were calculated for vosoritide vs placebo using an analysis of covariance model.
  5. 05HumanRandomized controlled trialEfficacy outcome

    In children aged ≥5 years, vosoritide reduced ankle joint to distal fibula physis distance versus placebo (LSM difference -0.07, P = .010).

    In plain wordsIn older children with achondroplasia, vosoritide reduced ankle joint to distal fibula physis distance compared with placebo.

    Peptide
    Vosoritide
    Outcome
    DecreaseAnkle joint to distal fibula physis distance, older children
    Study

    n: not reported in source

    Comparator
    Placebo
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 or 30 µg/kg/day · 1 yearAs given in this study (human).

    Measurements

    Difference in LSM change from baseline in ankle joint to distal fibula physis distance, vosoritide vs placebo
    −0.07 not stated
    children aged ≥5 years

    Source sentences

    Summary
    Children aged ≥5 years improved in ankle joint to distal fibula physis distance (difference in LSM change from baseline, -0.07, P = .010) and fibula/tibia ratio (-0.025, P < .0001), and children aged 2-5 years had improved fibula/tibia ratio (-0.033, P = .0032).
    2 context sentences
    Summary · context
    Data were collected from 183 participants from randomized, double-blind, placebo-controlled phase 2 (CANOPY ACH-2I [111-206; NCT03583697]) and phase 3 (CANOPY ACH-3 [111-301; NCT03197766]) studies evaluating vosoritide (weight-based dose of 15 or 30 µg/kg/day) in children aged 0-5 and >5 years, respectively.
    Summary · context
    Differences in least-squares mean (LSM) change from baseline were calculated for vosoritide vs placebo using an analysis of covariance model.
  6. 06HumanRandomized controlled trialEfficacy outcome

    In children aged ≥5 years, vosoritide reduced fibula/tibia ratio versus placebo (LSM difference -0.025, P < .0001).

    In plain wordsIn older children with achondroplasia, vosoritide reduced the fibula/tibia ratio compared with placebo.

    Peptide
    Vosoritide
    Outcome
    DecreaseFibula/tibia ratio, older children
    Study

    n: not reported in source

    Comparator
    Placebo
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 or 30 µg/kg/day · 1 yearAs given in this study (human).

    Measurements

    Difference in LSM change from baseline in fibula/tibia ratio, vosoritide vs placebo
    −0.025 ratio
    children aged ≥5 years

    Source sentences

    Summary
    Children aged ≥5 years improved in ankle joint to distal fibula physis distance (difference in LSM change from baseline, -0.07, P = .010) and fibula/tibia ratio (-0.025, P < .0001), and children aged 2-5 years had improved fibula/tibia ratio (-0.033, P = .0032).
    2 context sentences
    Summary · context
    Data were collected from 183 participants from randomized, double-blind, placebo-controlled phase 2 (CANOPY ACH-2I [111-206; NCT03583697]) and phase 3 (CANOPY ACH-3 [111-301; NCT03197766]) studies evaluating vosoritide (weight-based dose of 15 or 30 µg/kg/day) in children aged 0-5 and >5 years, respectively.
    Summary · context
    Differences in least-squares mean (LSM) change from baseline were calculated for vosoritide vs placebo using an analysis of covariance model.
  7. 07HumanRandomized controlled trialEfficacy outcome

    In children aged 2-5 years, vosoritide reduced fibula/tibia ratio versus placebo (LSM difference -0.033, P = .0032).

    In plain wordsIn younger children with achondroplasia, vosoritide reduced the fibula/tibia ratio compared with placebo.

    Peptide
    Vosoritide
    Outcome
    DecreaseFibula/tibia ratio, younger children
    Study

    n: not reported in source

    Comparator
    Placebo
    Outcome group
    Bone and joint · Musculoskeletal
    Studied dose
    15 or 30 µg/kg/day · 1 yearAs given in this study (human).

    Measurements

    Difference in LSM change from baseline in fibula/tibia ratio, vosoritide vs placebo
    −0.033 ratio
    children aged 2-5 years

    Source sentences

    Summary
    Children aged ≥5 years improved in ankle joint to distal fibula physis distance (difference in LSM change from baseline, -0.07, P = .010) and fibula/tibia ratio (-0.025, P < .0001), and children aged 2-5 years had improved fibula/tibia ratio (-0.033, P = .0032).
    2 context sentences
    Summary · context
    Data were collected from 183 participants from randomized, double-blind, placebo-controlled phase 2 (CANOPY ACH-2I [111-206; NCT03583697]) and phase 3 (CANOPY ACH-3 [111-301; NCT03197766]) studies evaluating vosoritide (weight-based dose of 15 or 30 µg/kg/day) in children aged 0-5 and >5 years, respectively.
    Summary · context
    Differences in least-squares mean (LSM) change from baseline were calculated for vosoritide vs placebo using an analysis of covariance model.

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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