hrp0097rfc4.5 | Growth and syndromes (to include Turner syndrome) | ESPE2023

Real-world safety and effectiveness of vosoritide: Results from an early access program in France

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

Introduction: Achondroplasia is the most common skeletal dysplasia, in which the main clinical feature is short stature. Vosoritide, the first specific treatment for achondroplasia; administered as a daily subcutaneous injection, was approved by the European Medicines Agency in August 2021 for patients aged ≥2 years until closure of epiphyses. French Health Authorities granted early access to vosoritide treatment in France on 24 June 2021, which continued u...