Vosoritide in achondroplasia: a profile of its use
Funding The preparation of this review was not supported by any external funding.
Authorship and conflict of interest Young-A Heo, a salaried employee of Adis International Ltd/Springer Nature and an editor of Drugs & Therapy Perspectives, was not involved in any publishing decisions for the manuscript and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content
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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.