hrp0098p2-56 | Bone, Growth Plate and Mineral Metabolism | ESPE2024

Real-world effectiveness of vosoritide in children with achondroplasia: Results from 18 months follow-up in France

Cormier-Daire Valerie , Edouard Thomas , Isidor Bertrand , Pimenta Jeanne , Mukherjee Swati , Marcos Valeria , Dee Anne , Rossi Massimiliano , Schaefer Elise , Sigaudy Sabine , Baujat Geneviève

Introduction: Achondroplasia is the most common skeletal dysplasia, caused by a pathogenic FGFR3 variant, leading to impaired endochondral bone growth and multiple medical complications. Vosoritide, a modified recombinant human C-type natriuretic peptide (rhCNP), was first approved by the European Medicines Agency in August 2021 and is now approved for treating genetically-confirmed achondroplasia in patients aged ≥4 months until closure of epiphys...