Achondroplasia is the most common form of short stature skeletal dysplasia, caused by a gain-of-function mutation in the fibroblast growth factor receptor 3 (FGFR3) gene; it is associated with multisystem complications which may occur throughout the life course. Multidisciplinary care over the entire lifespan is needed to provide optimal care for individuals with achondroplasia; until recently there have been no medical therapies available that address the underlying pathophysiology of the condition.

Vosoritide is a modified recombinant human C-type natriuretic peptide (CNP) analogue that leverages the CNP pathway to counteract overactive FGFR3 signaling and allow endochondral bone growth; it was approved by the European Medicines Agency and the US Food and Drug Administration in 2021 for use in children with achondroplasia who have open epiphyses and are aged ≥ 5 years in the US, and ≥ 2 years in the European Union.

Practical guidance on the use of vosoritide is lacking; the aim of this publication was to share early experiences of vosoritide in clinical practice to provide a framework for the approach to management.

Multidisciplinary team support, infrastructure, appropriate training for patients and families, as well as expectation management are all key considerations for initiating vosoritide in clinical practice; core assessments to monitor efficacy and safety may include pubertal staging, anthropometry, and X-rays to confirm open physes, the review of adverse events, and discussion of concomitant or new medications. Timing and assessments may vary by region or healthcare system.

Sharing of early experience by providers treating children with achondroplasia is useful in developing standards on vosoritide treatment for patients and families; international consensus guidance is needed to support the use of vosoritide in clinical practice.