Once-Weekly Navepegritide in Children With Achondroplasia: The APPROACH Randomized Clinical Trial.

ABSTRACT:

Importance

Historically considered a skeletal dysplasia characterized by disproportionate short stature, achondroplasia is a condition with multisystemic effects due to the widespread expression of the fibroblast growth factor receptor 3 variant throughout the body, impacting muscle, neurological function, cardiorespiratory health, and health-related quality of life.

Objective

To evaluate the efficacy, safety, and tolerability of once-weekly navepegritide, an investigational prodrug of C-type natriuretic peptide, while assessing benefits beyond growth that may have important implications for complications and health-related quality of life in children with achondroplasia.

Design, setting, and participants

Enrollment for this pivotal phase 2b, randomized, double-blind,

SUBMITTER: Savarirayan R 

PROVIDER: S-EPMC12624480 | biostudies-literature | 2026 Jan

REPOSITORIES: biostudies-literature