Peer-reviewed article
Real-world safety and age-dependent effectiveness of vosoritide in achondroplasia: A single-center retrospective analysis of transition from growth hormone to vosoritide.
Shimada T, Nakayama H, Ueda I, Ishimi T, Yamada C et al. ·
Summary
Written by AI, not yet AICS's view · claude-haiku-4-5-20251001This study examined how vosoritide, a medication approved in Japan in 2022, affects growth in children with achondroplasia, a genetic condition causing short stature. Researchers followed 22 children: 13 who received vosoritide alone from the start, and 9 who switched from previous growth hormone therapy. Children who started vosoritide early (under 4 years old) showed the most growth improvement, while older children who switched from growth hormone also benefited but with different patterns. The main side effect was injection site pain in three children, with one stopping treatment.
Key points
- Group A (starting vosoritide early, median age 2.10 years) showed significant improvement in height measurements at years 3, 4, 5, and 6 of treatment
- Group B (switching from growth hormone, median age 10.1 years) showed significant height improvements at 1 year and 2.5 years after switching
- In Group B, children who started vosoritide before age 4 maintained stable height, while those starting at age 4 or older showed significant improvement
- Injection site pain occurred in 3 out of 22 patients (13.6%), with one patient discontinuing treatment due to this side effect
- Study provides real-world comparative safety and effectiveness data for vosoritide in Japan following its 2022 approval
Limitations
This is a small single-center retrospective study with only 22 children and varying follow-up periods, which limits generalizability to other populations and settings. The lack of a concurrent control group and the retrospective design prevent definitive causal conclusions about vosoritide's superiority over continued growth hormone therapy.
Abstract
Achondroplasia (ACH), a skeletal dysplasia, is characterized by disproportionate short stature and impaired quality of life. In Japan, growth hormone (GH) therapy has been available since 1997, and vosoritide, a C-type natriuretic peptide analog, was approved in 2022. Although clinical trials have demonstrated vosoritide's efficacy, real-world comparative data in Japan are scarce. We conducted a retrospective study of 22 children with ACH treated with vosoritide for over 6 months at a single institution between February 2018 and May 2025. Patients were divided into two groups: GH-naïve group (Group A, n = 13), receiving vosoritide alone, and a switch group (Group B, n = 9), who transitioned from prior GH treatment. Growth outcomes included annualized growth velocity (AGV) and height standard deviation scores (HT-SDS) calculated using Japanese and ACH-specific (HT-SDS_ACH) references. Group A (median age: 2.10 years) demonstrated significant improvements in HT-SDS_ACH at years 3 (p = 0.0022), 4 (p = 0.0004), 5 (p = 0.007), and 6 (p = 0.0012). Group B (median age: 10.1 years), with a median of 6.10 years of GH exposure, showed significant increases at 1 year (p = 0.047) and 2.5 years (p = 0.0054) after switching. In Group B, HT-SDS_ACH remained stable among patients starting vosoritide before age 4, but improved significantly in those beginning later. Injection site pain occurred in three patients, leading to one discontinuation. Vosoritide improves linear growth in children with ACH, including those previously treated with GH, showing greater benefit when started at ≥4 years, though earlier use may offer advantages.
Findings the Hub has extracted
Each claim below is labelled by its type of design and links back to this report.
- Intervention
- Treatment with vosoritide (C-type natriuretic peptide analog), either as initial therapy or following transition from growth hormone therapy
- Population
- 22 children with achondroplasia at a single Japanese institution; Group A (n=13, median age 2.10 years) receiving vosoritide alone; Group B (n=9, median age 10.1 years) transitioning from prior growth hormone treatment
Key finding
Group A showed significant improvements in height standard deviation scores at years 3, 4, 5, and 6 (p=0.0022 to p=0.0012)
T et al. · Europe PMC · 11 Nov 2025