Achondroplasia Treatments

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Brand Name

Voxzogo

Generic Name
Vosoritide
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FDA approval date: November 19, 2021
Form: Kit

What is Voxzogo (Vosoritide)?

VOXZOGO is indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial. VOXZOGO is a C type natriuretic peptide analog indicated to increase linear growth in pediatric patients with achondroplasia with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial.

Approved To Treat

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Related Clinical Trials

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to < 36 Months
A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Safety and Efficacy of Vosoritide in Infants and Young Children With Hypochondroplasia, Aged 0 to < 36 Months
Enrollment Status: Recruiting
Publish Date: March 31, 2026
Intervention Type: Drug
Study Phase: Phase 2

Summary: The purpose of this study is to evaluate the safety and efficacy of daily administration of vosoritide in participants with HCH aged 0 to \< 36 months over a 52-week period.

A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature
A Phase 2, Randomized, Controlled, Multicenter Study of Vosoritide in Children With Idiopathic Short Stature
Enrollment Status: Recruiting
Publish Date: March 13, 2026
Intervention Type: Drug
Study Phase: Phase 2

Summary: The purpose of this study is to evaluate i) the effect of multiple doses of vosoritide and ii) the effect of the therapeutic dose of vosoritide compared to human growth hormone (hGH)(hGH; only in the United States), in children with idiopathic short stature (ISS).

A Phase 2, Randomized, Multicenter, Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment.
A Phase 2, Randomized, Multicenter, Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment.
Enrollment Status: Recruiting
Publish Date: February 27, 2026
Intervention Type: Drug
Study Phase: Phase 2

Summary: The purpose of this basket study in children with Turner syndrome, SHOX deficiency, and Noonan syndrome is to evaluate the effect of 3 doses of vosoritide on growth as measured by AGV after 6 months of treatment. The long-term efficacy and safety of vosoritide at the therapeutic dose will be evaluated up to FAH.

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Related Latest Advances

Development of Muscle Function in Children with Achondroplasia Under Vosoritide Treatment: A Retrospective Single-Centre Observational Study.
Development of Muscle Function in Children with Achondroplasia Under Vosoritide Treatment: A Retrospective Single-Centre Observational Study.
Journal: Journal of musculoskeletal & neuronal interactions
Published: March 01, 2026
A Phase II Basket Trial of Vosoritide in Children with RASopathies, ACAN and NPR2 Deficiency.
A Phase II Basket Trial of Vosoritide in Children with RASopathies, ACAN and NPR2 Deficiency.
Journal: The Journal of clinical endocrinology and metabolism
Published: February 06, 2026
Key points of the International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with Achondroplasia
Key points of the International consensus guidelines on the implementation and monitoring of vosoritide therapy in individuals with Achondroplasia
Journal: Zhonghua yi xue yi chuan xue za zhi = Zhonghua yixue yichuanxue zazhi = Chinese journal of medical genetics
Published: February 01, 2026
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