hrp0095p1-508 | Growth and Syndromes | ESPE2022

Real-world experience with Vosoritide for achondroplasia: interim findings from an early access programme in France

Cormier-Daire Valérie , Cohen Shelda , Edouard Thomas , Isidor Bertrand , Mukherjee Swati , Pimenta Jeanne , Rossi Massimiliano , Schaefer Elise , Sigaudy Sabine , Baujat Geneviève

Introduction: Achondroplasia is caused by a pathogenic mutation in the FGFR3 gene, leading to impaired endochondral bone growth and multiple medical complications. Vosoritide (once daily, subcutaneous injection) has recently been approved by the European Medicines Agency (EMA) for treating achondroplasia in patients aged ≥2 years until closure of epiphyses. It has been made available in France via an early access program, a cohort Temporary Authorization fo...