Real-world data: effectiveness and safety of vosoritide in the treatment of achondroplasia in chinese population

Lifen Chen; Xiaofei Zhang; Xiwen Zhang; Wen Su; Xiaoyu Ma; Yuan Xiao; Wenli Lu; Fang Wang; Zhidong Gu; Zhiya Dong

doi:10.1530/endoabs.110.P636

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Endocrine Abstracts (2025) 110 P636 | DOI: 10.1530/endoabs.110.P636

ECEESPE2025 Poster Presentations Growth Axis and Syndromes (91 abstracts)

Real-world data: effectiveness and safety of vosoritide in the treatment of achondroplasia in chinese population

Lifen Chen ¹ , Xiaofei Zhang ¹ , Xiwen Zhang ² , Wen Su ¹ , Xiaoyu Ma ¹ , Yuan Xiao ¹ , Wenli Lu ¹ , Fang Wang ³ , Zhidong Gu ⁴ & Zhiya Dong ¹

Author affiliations

¹Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, RuiJin Hainan Hospital, Shanghai Jiao Tong University School of Medicine, Pediatrics, Shanghai, China; ²RuiJin Hospital, Shanghai Jiao Tong University School of Medicine, Pediatrics, Shanghai, China; ³RuiJin Hospital, Shanghai Jiao Tong University School of Medicine, Cardiovascular Medicine, Shanghai, China; ⁴RuiJin Hainan Hospital, Shanghai Jiao Tong University School of Medicine, Clinical Laboratory, Shanghai, China

JOINT2060

Background: Achondroplasia (ACH) is the most common form of hereditary skeletal dysplasia, caused by activating mutations in the FGFR3 gene. It leads to severe disproportionate short stature and multiple complications. Current management strategies focus on multidisciplinary care, but effective pharmacological treatments are limited. Vosoritide, a CNP analogue, is the first available molecular therapy for ACH in China, showing significant improvement in annual growth rate in children.

Methods: This retrospective cohort study collected data from 30 ACH patients treated with vosoritide at the Hainan Boao Lecheng International Medical Tourism Pilot Zone from June 2022 to August 2024. After excluding four cases due to irregular medication, 26 cases were analyzed. Inclusion criteria included a diagnosis of ACH confirmed by genetic testing and open or partially closed growth plates. Vosoritide was administered at 15 µg/kg by subcutaneous injection once daily. Data on physical conditions, growth measurements, and biochemical indices were collected and analyzed using SPSS 22. 0 software.

Results: The study included 26 patients (11 males, 15 females) from 3. 4 to 11. 7 years old. Significant improvements were observed in height SDS, annual growth velocity (AGV), and BMI SDS after 12 months of treatment. Height SDS increased by 0. 4, and AGV increased by 2. 3 cm/year. The SH/H ratio decreased, indicating improved body proportions. No significant difference was found in BA-CA. Vosoritide treatment was well-tolerated, with no serious adverse events reported.

Conclusion: Vosoritide effectively promotes linear growth and improves body proportions in Chinese children with ACH, providing an optimized treatment option. Further studies with larger sample sizes are needed to confirm these findings and explore potential racial differences in treatment efficacy.