P138: Interim real-world results from the Acorn Study: Long-term safety and growth outcomes of vosoritide in children with achondroplasia

Genetics in Medicine Open 2026 Open access

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Authors: Oliver Semler, Valérie Cormier‐Daire, Veronika Horváthová, Jeanne M. Pimenta, Anne Dee, Sheetal Ingole, Soraya Azmi, Mohamad Maghnie, Zdenĕk Šumnı́k
DOI: 10.1016/j.gimo.2026.103632
Cited by: 0

Abstract

Achondroplasia is a rare skeletal dysplasia caused by pathogenic variants in the gene Fibroblast Growth Factor Receptor 3 (FGFR3) associated with impaired endochondral bone growth and multiple well-characterized complications. Vosoritide is a targeted recombinant C-type natriuretic peptide analog approved by the European Medicines Agency (EMA) for treating achondroplasia in children ≥4 months of age until closure of epiphyses. Vosoritide was first approved by the EMA in 2021. Here, we present interim results from the post-approval real-world study in Europe designed to monitor the long-term safety of vosoritide (Acorn; 111-603).

Fields of Study & Subjects

Connective tissue disorders research Growth Hormone and Insulin-like Growth Factors Craniofacial Disorders and Treatments

Publication details

Journal / source
Genetics in Medicine Open
Volume
4
Pages
103632–103632
Publication date
1 January 2026
Language
EN