Vosoritide in achondroplasia: a profile of its use

<p>  </p> <p><strong>Declarations</strong></p> <p><strong>Funding</strong> The preparation of this review was not supported by any external funding.</p> <p><strong>Authorship and conflict of interest</strong> Young-A Heo, a salaried employee of Adis International Ltd/Springer Nature and an editor of <em>Drugs & Therapy Perspectives, was not involved in any publishing decisions for the manuscript and declares </em>no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content</p> <p><strong>Ethics approval, Consent to participate, Consent for publication, Availability of data and material, Code availability</strong> Not applicable </p> <p><br></p> <p> Additional information about this Adis Drug Review can be found <a href="http://www.springer.com/gp/adis/products-services/adis-journals-newsletters/adis-drug-reviews" target="_blank">here</a> </p> <p><br></p> <p>Abstract </p> <p>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.</p>