Effect of vosoritide on spine morphology in children with achondroplasia: 1-year results from a randomized phase 2 study.

Irving M, Savarirayan R, Hoover-Fong JE, Dee A, Mukherjee S, Rivat C, Sabir I, White KK

Journal of the Endocrine Society20 Feb 2026Cited by 1 (Europe PMC)

PMID41783511PMCIDPMC12954483DOI10.1210/jendso/bvag008PepBankPUB-41783511

Summary

Achondroplasia is a skeletal dysplasia condition caused by reduced endochondral ossification resulting in disproportionate short stature and skeletal deformities, including thoracolumbar kyphosis (TLK) and spinal stenosis. Vosoritide, the first and only approved targeted therapy for achondroplasia, increases bone growth, but its impact on spinal morphology has not been assessed. The randomized, double-blind, placebo-controlled phase 2 CANOPY ACH-2I study (111-206; NCT03583697) evaluated the safety and efficacy of vosoritide in 75 children aged 0 to <5 years. Interpedicular distance (IPD), sagittal width of the lumbar spinal canal, and TLK angle were measured on spinal radiographs taken at baseline and 1 year after vosoritide or placebo treatment. Differences in least-squares mean (LSM) change from baseline (95% CI) between treatment groups were determined with an analysis of covariance model. Measurable improvements in IPD and spinal canal width across L1 to L5 were observed with vosoritide compared with placebo after 1 year. L4 was the most impacted by vosoritide for IPD (LSM difference [95% CI], 0.509 [-0.034 to 1.052] mm, P = .066) and canal width (1.433 [0.547 to 2.320] mm, P = .002). Vosoritide treatment also reduced the natural increase in TLK angle in children 0 to <0.5 years and provided greater improvements in children ≥0.5 to <5 years. After 1 year, fewer children treated with vosoritide (33.3%) vs placebo (59.3%) had pathological (≥20°) TLK angles (P = .037). These preliminary results suggest that early vosoritide treatment may improve spinal morphology and reduce the risk of spinal stenosis in children with achondroplasia. Clinical Trial Information: NCT03583697.

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.

01HumanRandomized controlled trialEfficacy outcome
In children with achondroplasia, measurable improvements in interpedicular distance across L1 to L5 were observed with vosoritide compared with placebo after 1 year.
In plain wordsIn children with achondroplasia, lumbar interpedicular distance showed measurable improvements with vosoritide compared with placebo.
Peptide
Vosoritide
Outcome
Direction not reportedLumbar interpedicular distance
Study
n: not reported in source
Comparator
Placebo
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 1 yearAs given in this study (human).
Source sentences
Summary
Measurable improvements in IPD and spinal canal width across L1 to L5 were observed with vosoritide compared with placebo after 1 year.
3 context sentences(design, population, supporting numbers)
Summary · context
The randomized, double-blind, placebo-controlled phase 2 CANOPY ACH-2I study (111-206; NCT03583697) evaluated the safety and efficacy of vosoritide in 75 children aged 0 to <5 years.
Summary · context
Interpedicular distance (IPD), sagittal width of the lumbar spinal canal, and TLK angle were measured on spinal radiographs taken at baseline and 1 year after vosoritide or placebo treatment.
Summary · context
Differences in least-squares mean (LSM) change from baseline (95% CI) between treatment groups were determined with an analysis of covariance model.
Show in the study text Report a problemFND-4178351101
02HumanRandomized controlled trialEfficacy outcome
In children with achondroplasia, measurable improvements in spinal canal width across L1 to L5 were observed with vosoritide compared with placebo after 1 year.
In plain wordsIn children with achondroplasia, lumbar spinal canal width showed measurable improvements with vosoritide compared with placebo.
Peptide
Vosoritide
Outcome
Direction not reportedLumbar spinal canal width
Study
n: not reported in source
Comparator
Placebo
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 1 yearAs given in this study (human).
Source sentences
Summary
Measurable improvements in IPD and spinal canal width across L1 to L5 were observed with vosoritide compared with placebo after 1 year.
3 context sentences(design, population, supporting numbers)
Summary · context
The randomized, double-blind, placebo-controlled phase 2 CANOPY ACH-2I study (111-206; NCT03583697) evaluated the safety and efficacy of vosoritide in 75 children aged 0 to <5 years.
Summary · context
Interpedicular distance (IPD), sagittal width of the lumbar spinal canal, and TLK angle were measured on spinal radiographs taken at baseline and 1 year after vosoritide or placebo treatment.
Summary · context
Differences in least-squares mean (LSM) change from baseline (95% CI) between treatment groups were determined with an analysis of covariance model.
Show in the study text Report a problemFND-4178351102
03HumanRandomized controlled trialEfficacy outcome
In children with achondroplasia, vosoritide versus placebo produced an L4 interpedicular distance change difference of 0.509 mm (95% CI -0.034 to 1.052, P = .066).
In plain wordsIn children with achondroplasia, L4 interpedicular distance was numerically higher with vosoritide than placebo (P = .066).
Peptide
Vosoritide
Outcome
IncreaseL4 interpedicular distance
Study
n: not reported in source
Comparator
Placebo
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 1 yearAs given in this study (human).
Measurements
LSM difference in L4 interpedicular distance change, vosoritide vs placebo
0.509 mm
L4, after 1 year
95% CI lower limit of LSM difference in L4 interpedicular distance change
−0.034 mm
L4, after 1 year
95% CI upper limit of LSM difference in L4 interpedicular distance change
1.052 mm
L4, after 1 year
Source sentences
Summary
L4 was the most impacted by vosoritide for IPD (LSM difference [95% CI], 0.509 [-0.034 to 1.052] mm, P = .066) and canal width (1.433 [0.547 to 2.320] mm, P = .002).
3 context sentences(design, population, supporting numbers)
Summary · context
The randomized, double-blind, placebo-controlled phase 2 CANOPY ACH-2I study (111-206; NCT03583697) evaluated the safety and efficacy of vosoritide in 75 children aged 0 to <5 years.
Summary · context
Interpedicular distance (IPD), sagittal width of the lumbar spinal canal, and TLK angle were measured on spinal radiographs taken at baseline and 1 year after vosoritide or placebo treatment.
Summary · context
Differences in least-squares mean (LSM) change from baseline (95% CI) between treatment groups were determined with an analysis of covariance model.
Show in the study text Report a problemFND-4178351103
04HumanRandomized controlled trialEfficacy outcome
In children with achondroplasia, vosoritide versus placebo produced an L4 canal width change difference of 1.433 mm (95% CI 0.547 to 2.320, P = .002).
In plain wordsIn children with achondroplasia, vosoritide increased L4 spinal canal width compared with placebo.
Peptide
Vosoritide
Outcome
IncreaseL4 spinal canal width
Study
n: not reported in source
Comparator
Placebo
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 1 yearAs given in this study (human).
Measurements
LSM difference in L4 spinal canal width change, vosoritide vs placebo
1.433 mm
L4, after 1 year
95% CI lower limit of LSM difference in L4 spinal canal width change
0.547 mm
L4, after 1 year
95% CI upper limit of LSM difference in L4 spinal canal width change
2.32 mm
L4, after 1 year
Source sentences
Summary
L4 was the most impacted by vosoritide for IPD (LSM difference [95% CI], 0.509 [-0.034 to 1.052] mm, P = .066) and canal width (1.433 [0.547 to 2.320] mm, P = .002).
3 context sentences(design, population, supporting numbers)
Summary · context
The randomized, double-blind, placebo-controlled phase 2 CANOPY ACH-2I study (111-206; NCT03583697) evaluated the safety and efficacy of vosoritide in 75 children aged 0 to <5 years.
Summary · context
Interpedicular distance (IPD), sagittal width of the lumbar spinal canal, and TLK angle were measured on spinal radiographs taken at baseline and 1 year after vosoritide or placebo treatment.
Summary · context
Differences in least-squares mean (LSM) change from baseline (95% CI) between treatment groups were determined with an analysis of covariance model.
Show in the study text Report a problemFND-4178351104
05HumanRandomized controlled trialEfficacy outcome
In children aged 0 to <0.5 years, vosoritide reduced the natural increase in TLK angle versus placebo.
In plain wordsIn young infants with achondroplasia, vosoritide limited the increase in spinal curvature compared with placebo.
Peptide
Vosoritide
Outcome
DecreaseTLK angle, infants below half a year
Study
n: not reported in source
Comparator
Placebo
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 1 yearAs given in this study (human).
Source sentences
Summary
Vosoritide treatment also reduced the natural increase in TLK angle in children 0 to <0.5 years and provided greater improvements in children ≥0.5 to <5 years.
3 context sentences(design, population, supporting numbers)
Summary · context
The randomized, double-blind, placebo-controlled phase 2 CANOPY ACH-2I study (111-206; NCT03583697) evaluated the safety and efficacy of vosoritide in 75 children aged 0 to <5 years.
Summary · context
Interpedicular distance (IPD), sagittal width of the lumbar spinal canal, and TLK angle were measured on spinal radiographs taken at baseline and 1 year after vosoritide or placebo treatment.
Summary · context
Differences in least-squares mean (LSM) change from baseline (95% CI) between treatment groups were determined with an analysis of covariance model.
Show in the study text Report a problemFND-4178351105
06HumanRandomized controlled trialEfficacy outcome
In children aged ≥0.5 to <5 years, vosoritide provided greater TLK angle improvements versus placebo.
In plain wordsIn older children with achondroplasia, vosoritide improved spinal curvature more than placebo.
Peptide
Vosoritide
Outcome
Direction not reportedTLK angle, older children
Study
n: not reported in source
Comparator
Placebo
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 1 yearAs given in this study (human).
Source sentences
Summary
Vosoritide treatment also reduced the natural increase in TLK angle in children 0 to <0.5 years and provided greater improvements in children ≥0.5 to <5 years.
3 context sentences(design, population, supporting numbers)
Summary · context
The randomized, double-blind, placebo-controlled phase 2 CANOPY ACH-2I study (111-206; NCT03583697) evaluated the safety and efficacy of vosoritide in 75 children aged 0 to <5 years.
Summary · context
Interpedicular distance (IPD), sagittal width of the lumbar spinal canal, and TLK angle were measured on spinal radiographs taken at baseline and 1 year after vosoritide or placebo treatment.
Summary · context
Differences in least-squares mean (LSM) change from baseline (95% CI) between treatment groups were determined with an analysis of covariance model.
Show in the study text Report a problemFND-4178351106
07HumanRandomized controlled trialEfficacy outcome
After 1 year, pathological TLK angles occurred in 33.3% of vosoritide-treated children versus 59.3% with placebo (P = .037).
In plain wordsIn children with achondroplasia, pathological spinal curvature was less frequent with vosoritide than placebo.
Peptide
Vosoritide
Outcome
DecreasePathological TLK angle
Study
n: not reported in source
Comparator
Placebo
Outcome group
Bone and joint · Musculoskeletal
Studied dose
Dose not stated · 1 yearAs given in this study (human).
Measurements
Children with pathological (≥20°) TLK angle
33.3%
vosoritide, after 1 year
Children with pathological (≥20°) TLK angle
59.3%
placebo, after 1 year
Source sentences
Summary
After 1 year, fewer children treated with vosoritide (33.3%) vs placebo (59.3%) had pathological (≥20°) TLK angles (P = .037).
3 context sentences(design, population, supporting numbers)
Summary · context
The randomized, double-blind, placebo-controlled phase 2 CANOPY ACH-2I study (111-206; NCT03583697) evaluated the safety and efficacy of vosoritide in 75 children aged 0 to <5 years.
Summary · context
Interpedicular distance (IPD), sagittal width of the lumbar spinal canal, and TLK angle were measured on spinal radiographs taken at baseline and 1 year after vosoritide or placebo treatment.
Summary · context
Differences in least-squares mean (LSM) change from baseline (95% CI) between treatment groups were determined with an analysis of covariance model.
Show in the study text Report a problemFND-4178351107

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

Registered trials1

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

1 trial · 1 completed · 1 with posted results · source ClinicalTrials.gov (opens in a new tab)

CompletedPhase 2Results postedNCT03583697

A Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children With Achondroplasia

BioMarin Pharmaceuticaln = 7513 Jun 2018 to 26 Jan 2022

Achondroplasia

Trial page ClinicalTrials.govUpdated 13 Jun 2024

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

Subjects

Terms as the source lists them.

Publication types

Keywords

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