A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia

BACKGROUND: )-related skeletal condition characterized by disproportionate short stature and a spectrum of clinical features, has no available targeted therapies. Vosoritide, a C-type natriuretic peptide analogue approved for the treatment of achondroplasia, is being investigated for hypochondroplasia. METHODS: In this phase 3, multicenter trial, children with hypochondroplasia who were 3 to less than 18 years of age were randomly assigned to receive once-daily subcutaneous injections of vosoritide or placebo for 52 weeks per weight-band dosing regimen. The primary end point was change from baseline in annualized growth velocity at week 52 versus placebo. Confirmatory statistical testing using hierarchical procedures to control for type I error at the one-sided 0.025 significance level (equivalent to the two-sided 0.05 level) was performed for the primary and six key secondary efficacy end points. The safety and side effect profile of vosoritide versus placebo was assessed. RESULTS: A total of 81 participants were randomly assigned to receive vosoritide (n=41) or placebo (n=40). At week 52, the least squares mean (LSM) change from baseline in annualized growth velocity was 1.95 cm/year with vosoritide versus -0.39 cm/year with placebo (LSM difference of 2.33 cm/year; 95% confidence interval, 1.85-2.82 cm/year; two-sided P<0.0001). Most participants in the vosoritide group (87.8%) and the placebo group (72.5%) experienced at least one adverse event (AE). There were no reports of grade 3 or higher AEs, AEs leading to treatment discontinuation, or deaths. CONCLUSIONS: One year of vosoritide treatment significantly increased linear growth in children with hypochondroplasia. (Funded by BioMarin Pharmaceutical; ClinicalTrials.gov number, NCT06455059.).

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Journal
NEJM Evidence
Published
2026-09-09
DOI
https://doi.org/10.1056/evidoa2600257
Primary Topic
Connective tissue disorders research
Type
article
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article

A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia

Noriyuki Namba, Roberto Mendoza‐Londono, Ricki Carroll, Kei Takasawa et al.
NEJM Evidence
Connective tissue disorders research
article

A Phase 3 Trial of Vosoritide in Children with Hypochondroplasia

Noriyuki Namba, Roberto Mendoza‐Londono, Ricki Carroll, Kei Takasawa, Rachel Reynaud, Yutaka Kinoshita, Michaela Veronika Gonfiantini, Yasuhisa Ohata, Takuo Kubota, Alice Huntsman Labed, Roberta Onesimo, Oliver Semler, Josep Maria De Bergua, Katja Palm, Philippe M. Campeau, Peter Kannu, Dane Osmond, Sajda Ghani, Jeanette White, Carlos E. Prada, Moira Cheung, Ian Sabir, Roberto Bassi, Valérie Cormier-Daire, Mohamad Maghnie, Thomas Edouard, Andrew Dauber, Ravi Savarirayan, Julie Hoover-Fong, Massimiliano Rossi
article en

Abstract

BACKGROUND: )-related skeletal condition characterized by disproportionate short stature and a spectrum of clinical features, has no available targeted therapies. Vosoritide, a C-type natriuretic peptide analogue approved for the treatment of achondroplasia, is being investigated for hypochondroplasia. METHODS: In this phase 3, multicenter trial, children with hypochondroplasia who were 3 to less than 18 years of age were randomly assigned to receive once-daily subcutaneous injections of vosoritide or placebo for 52 weeks per weight-band dosing regimen. The primary end point was change from baseline in annualized growth velocity at week 52 versus placebo. Confirmatory statistical testing using hierarchical procedures to control for type I error at the one-sided 0.025 significance level (equivalent to the two-sided 0.05 level) was performed for the primary and six key secondary efficacy end points. The safety and side effect profile of vosoritide versus placebo was assessed. RESULTS: A total of 81 participants were randomly assigned to receive vosoritide (n=41) or placebo (n=40). At week 52, the least squares mean (LSM) change from baseline in annualized growth velocity was 1.95 cm/year with vosoritide versus -0.39 cm/year with placebo (LSM difference of 2.33 cm/year; 95% confidence interval, 1.85-2.82 cm/year; two-sided P<0.0001). Most participants in the vosoritide group (87.8%) and the placebo group (72.5%) experienced at least one adverse event (AE). There were no reports of grade 3 or higher AEs, AEs leading to treatment discontinuation, or deaths. CONCLUSIONS: One year of vosoritide treatment significantly increased linear growth in children with hypochondroplasia. (Funded by BioMarin Pharmaceutical; ClinicalTrials.gov number, NCT06455059.).

NEJM Evidence
Université Claude Bernard Lyon 1 (FR), Università Cattolica del Sacro Cuore (IT), Northwestern University (US), Osaka Gakuin University (JP), Royal Children's Hospital (AU), Children's National (US), Johns Hopkins University (US), The University of Melbourne (AU), Université Fédérale de Toulouse Midi-Pyrénées (FR), University of Cologne (DE), George Washington University (US), Université Paris Cité (FR), Johns Hopkins Medicine (US), Great Ormond Street Hospital (GB), Hospital for Sick Children (CA), Community Health Systems - Dupont Hospital (US), Istituto Giannina Gaslini (IT), Centre Hospitalier Universitaire Sainte-Justine (CA), National Institute for Health and Care Research (GB), Hôpital Femme Mère Enfant (FR), Assistance Publique Hôpitaux de Marseille (FR), Osaka Women's and Children's Hospital (JP), BioMarin (United Kingdom) (GB), Nemours Children's Health System (US), Unidad de Cirugía Artroscópica (ES), Bambino Gesù Children's Hospital (IT), Lurie Children's Hospital (US), Murdoch Children's Research Institute (AU), University of the Sacred Heart (JP), University Hospital Cologne (DE), Stollery Children's Hospital (CA), Institut des Maladies Génétiques Imagine (FR), University College London (GB), Tottori University (JP), University of Genoa (IT), BioMarin (United States) (US), Tokushima University (JP), Otto-von-Guericke-Universität Magdeburg (DE), The University of Osaka (JP)
Good health and well-being
Openalex Percentile: Top 11%
Connective tissue disorders research
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