Real-World Safety and Effectiveness of Vosoritide in Children with Achondroplasia: French Early Access Program
Authors' conclusion
In this first use of vosoritide in a real-world setting, vosoritide had a positive benefit-risk ratio similar to that observed in vosoritide clinical trials. The French EAP provides a model that may be adapted and adopted for use in other countries.
Study context
- Population
- Children aged ≥5 years with achondroplasia and open epiphyses, six French rare disease reference centers, 24 June 2021 to 13 December 202257 started treatment
- Participants
- 62 children
- Outcome
- Effectiveness after 12 months of treatment
- Follow-up
- 12 months
Methodological detailsreal-world, single-arm, multicentre, six centers, 38 completed at least 6 months and 22 at least 12 months of treatment
- Real-world, single-arm
- Multicentre
- Six centers
- 38 completed at least 6 months and 22 at least 12 months of treatment
Main finding
As reported in the abstract
Mean AGV
6.0 cm/year
Absolute gain in height
6.2 cm
Increase in height Z-score (average stature reference)
0.38
Safety
Adverse events · 12-month follow-up
- All adverse events were mild (mainly injection site reactions)
- No discontinuations related to vosoritide treatment
Original publication
Real-World Safety and Effectiveness of Vosoritide in Children with Achondroplasia: French Early Access Program. Horm Res Paediatr. Published 25 January 2025. doi:10.
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