Description
Cormier-Daire et al. shared the organisational structure, initiation, follow-up protocol, and findings of a vosoritide early access program (EAP) in France. Real-world outcomes support a positive benefit–risk profile for vosoritide that are consistent with clinical trial data.
The vosoritide EAP was conducted at 6 centres from 24 June 2021 to 13 December 2022. Necker-Enfants Malades Hospital was the coordinating centre and the other 5 rare disease competence centres were the University Hospitals of Lyon, Marseille, Nantes, Strasbourg, and Toulouse. Participants enrolled in the EAP demonstrated good adherence. Height z-scores increased from baseline to Month 18 in boys and girls. While mean height increased to Month 18, annualised growth velocity decreased to Month 18. The post hoc 18-month outcomes of this study were in line with previous findings. Safety findings during the EAP were consistent with clinical trial data.
References:
Cormier-Daire V, Edouard T, Isidor B, Mukherjee S, Pimenta JM, Rossi M, Schaefer E, Sigaudy S, Baujat G
Horm Res Paediatr. 2025.
doi: 10.1159/000543743.