Vosoritide daily injections in prepubertal hypochondroplasia patients over 12 months

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The Journal of clinical endocrinology and metabolism Published April 22, 2026 Medically reviewed May 25, 2026 Study authors: Kanakatti Shankar Roopa, Galetaki Despoina M, Zhang Anqing, Shafaei Niusha, Pitner Kimberly, Seafort… PubMed ↗ DOI ↗ By Dr. Sofia Müller, MD · Lifespan & Whole-Person Care

Key Takeaway

Consider the correlation between IGF-1 and NTproCNP changes, but note the single-arm design and lack of clinical outcome data.

This was a phase 2, single-arm, unblinded, standardized clinical trial in prepubertal patients aged 3 to 11 years with hypochondroplasia and height less than -2.25 SD. The intervention was daily subcutaneous vosoritide 15 μg/kg/day, with a baseline 6-month observation period as a comparator. The follow-up duration was 12.0 months.

Main results showed that IGF-1 levels reduced at baseline and increased throughout the study, but IGF-1 SD did not change significantly. NTproCNP levels raised at baseline and declined throughout the study, and NTproCNP SD was significantly lower at months 6 and 12 on treatment. The correlation between change in height SD or AGV and IGF-1 or NTproCNP SD was not correlated, while the correlation between change in IGF-1 concentrations and change in NTproCNP plasma concentrations was positively correlated (ρ = 0.57, P = .024).

Safety data, including adverse events, serious adverse events, discontinuations, and tolerability, were not reported. Key limitations include the single-arm, unblinded design. The practice relevance was not reported. The causality note suggests a possible interaction between IGF-1 and CNP signaling, but the evidence is observational and does not establish causation.

Study Details

Study typePhase2

EvidenceLevel 3

Follow-up12.0 mo

PublishedApr 2026

PMID41157964

View Original Abstract ↓

CONTEXT: Insulin-like growth factor-1 (IGF-1) and C-type natriuretic peptide (CNP) promote endochondral bone growth. We previously reported that vosoritide, a CNP analogue, increases annualized growth velocity (AGV) and height SD in prepubertal children with hypochondroplasia (HCH). OBJECTIVE: We hypothesized that IGF-1 and CNP will be inversely associated at baseline and will respond differentially during a single-arm, unblinded, standardized 12-month phase 2 clinical trial of vosoritide in prepubertal patients aged 3 to 11 years with HCH and height less than -2.25 SD. Participants were followed for a baseline 6-month observation period and then received a dose of 15 μg/kg/day of vosoritide subcutaneous daily injections for 12 months. Anthropometrics, IGF-1, and NTproCNP were measured every 6 months. Longitudinal changes in IGF-1 and NTproCNP as well as the correlations between these 2 analytes and changes in height SD and AGV were analyzed. RESULTS: Baseline IGF-1 values are reduced in children with HCH and increased throughout the study, but IGF-1 SD did not change significantly. NTproCNP was raised at baseline and declined throughout the study, with NTproCNP SD significantly lower at months 6 and 12 on treatment. Change in height SD or AGV were not correlated to IGF-1 or NTproCNP SD at 12 months. The change in IGF-1 concentrations at 12 months was positively correlated with the change in NTproCNP plasma concentrations (ρ = 0.57; P = .024). CONCLUSION: In children with HCH, IGF-1 levels are reduced and NTproCNP levels are elevated at baseline. A correlation between changes in these levels during treatment suggests a possible interaction between IGF-1 and CNP signaling.