Subcutaneous vosoritide (Voxzogo®), a modified recombinant human C-type natriuretic peptide (CNP) analogue, is the first precision therapy approved for the treatment of achondroplasia in the EU, USA and multiple other countries. It is indicated for patients aged ≥ 2 years with open epiphyses in the EU and those aged ≥ 5 years with open epiphyses in the USA. In a phase 3 trial (study 111-301), vosoritide 15 μg/kg/day significantly improved annualized growth velocity and height Z-score compared with placebo, without worsening upper-to-lower body segment ratio in children with achondroplasia. The growth-promoting effects of vosoritide persisted with continued treatment for up to 60 months. Vosoritide is generally well tolerated, with most adverse events being mild in severity. Longer-term safety data revealed no new safety signals. Achondroplasia is the most common form of skeletal dysplasia and is characterized by short stature with disproportionate bone growth. The condition is caused by a gain-of-function mutation in the gene for fibroblast growth factor receptor 3 (FGFR3), which is a negative regulator of bone growth. With increased understanding of the pathogenesis of achondroplasia, vosoritide (Voxzogo®), a modified recombinant human C-type natriuretic peptide (CNP) analogue, has been approved as the first precision therapy for the treatment of achondroplasia in patients aged ≥ 2 years with open epiphyses in the EU and those aged ≥ 5 years with open epiphyses in the USA. In children with achondroplasia, daily subcutaneous administration of vosoritide was associated with growth-promoting effects. Vosoritide is generally well tolerated, with most adverse events being mild in severity. Thus, vosoritide is a valuable, effective and generally well-tolerated treatment option for children with achondroplasia.