C-Type Natriuretic Peptide Analogue Therapy in Children with Achondroplasia

This phase 2 study evaluated the safety and efficacy of vosoritide, a C-type natriuretic peptide analogue, in 35 children (aged 5–14 years) with achondroplasia. Participants received daily subcutaneous vosoritide at escalating doses (2.5–30.0 μg/kg) over 42 months. Results showed a dose-dependent increase in annualized growth velocity (peaking at 15 μg/kg), with sustained improvements in height *z* scores and proportional growth. Adverse events were mild, primarily injection-site reactions, with no severe cardiovascular or skeletal complications. Biomarker analyses confirmed sustained pharmacologic activity. These findings support vosoritide as a promising therapy for achondroplasia, addressing the underlying FGFR3-mediated growth inhibition. Ongoing phase 3 trials (NCT03197766, NCT03424018) aim to further validate these results.

This phase 2 study evaluated the safety and efficacy of vosoritide, a C-type natriuretic peptide analogue, in 35 children (aged 5–14 years) with achondroplasia. Participants received daily subcutaneous vosoritide at escalating doses (2.5–30.0 μg/kg) over 42 months. Results showed a dose-dependent increase in annualized growth velocity (peaking at 15 μg/kg), with sustained improvements in height *z* scores and proportional growth. Adverse events were mild, primarily injection-site reactions, with no severe cardiovascular or skeletal complications. Biomarker analyses confirmed sustained pharmacologic activity. These findings support vosoritide as a promising therapy for achondroplasia, addressing the underlying FGFR3-mediated growth inhibition. Ongoing phase 3 trials (NCT03197766, NCT03424018) aim to further validate these results.