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Efficacy Data

The safety and efficacy of VOXZOGO® (vosoritide) were evaluated in patients with achondroplasia aged 5 to 15 years1,2

Statistically significant improvements in annualised growth velocity (AGV) were seen in patients aged 5 to 15 years1

Mean (SD) AGV results over baseline from a placebo-controlled clinical trial1-3

SD, standard deviation.
*LS means were estimated from the ANCOVA (analysis of covariance) model, which included treatment, stratum defined by sex and Tanner stage, baseline age, baseline AGV, and baseline height Z-score.
All randomised subjects. Two patients in the VOXZOGO group discontinued from the study before Week 52. The values for these 2 patients were imputed assuming baseline growth rate for the period with missing data.
Baseline AGV was based on standing height at least 6 months prior to enrollment into the study.
§Two-sided P value.

Consistent improvement in AGV in favour of VOXZOGO® observed across all subgroups1,2

Difference in mean change from baseline in AGV at Week 52 by subgroup

Data are based on 3 and 5 male patients taking VOXZOGO® and placebo, respectively, who were Tanner stage >|.
ANCOVA models were applied to determine the least squares mean change from baseline treatment difference at 52 weeks and 95% CIs. All models provide the treatment difference adjusted for the following baseline covariates: strata (male Tanner stage I, female Tanner stage I, male Tanner stage >I, female Tanner stage >I); age; annualised growth velocity; and height Z-score. Subgroup analyses were done by applying the ANCOVA model used for the primary analysis to each subgroup category. The vertical dashed line represents the change from baseline difference of 1.75 cm/year for which the study was powered.
AGV: annualised growth velocity; Z-score: height standard-deviation score.

VOXZOGO® improved height standard-deviation score (Z-score) compared to placebo1,2

References:

  1. VOXZOGO® Product Information, 2022
  2. Savarirayan R, Tofts L, Irving M, et al. Once-daily, subcutaneous vosoritide therapy in children with achondroplasia: a randomised, double-blind, phase 3, placebo-controlled, multicentre trial. Lancet. 2020;396:684-692.
  3. Savarirayan R. Persistent and stable growth promoting effects of vosoritide in children with achondroplasia for up to 2 years: results from the ongoing phase 3 extension study. Oral presentation at: Endocrine Society’s Annual Meeting; November 21-24, 2021; Victoria, Australia.