hrp0097t8 | Section | ESPE2023

Design and Objectives of the Acorn Study: A Non-Interventional Study Evaluating Long-term Safety in Achondroplasia Patients Treated with Vosoritide

M Pimenta Jeanne , Cohen Shelda , Mukherjee Swati , Fettes Fiona , Jayaram Kala , Lausch Ekkehart , Mallya Usha G. , Min Yang , Caroline Huber , Cala Mary Lynn , Ali Greatsinger , Jeremy Pomeroy , Haqq Andrea M.

Introduction: Achondroplasia is caused by a pathogenic mutation in the FGFR3 gene, leading to impaired endochondral bone growth and multiple medical complications. Vosoritide, a modified recombinant human C-type natriuretic peptide (rhCNP), was approved by the European Medicines Agency (EMA) in August 2021 for treating genetically confirmed achondroplasia in patients aged ≥2 years until closure of epiphyses. Acorn is the first treatment-based registry for a...