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Vosoritide in achondroplasia: a profile of its use

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Abstract

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.

Plain Language Summary

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.

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Acknowledgements

The manuscript was reviewed by: H. Kitoh, Department of Orthopaedic Surgery, Aichi Children's Health and Medical Center, Obu, Japan; K. Mohnike, Otto-von-Guericke-Universität, Magdeburg, Germany. During the peer review process, BioMarin Pharmaceuticals Inc., the marketing authorization holder of vosoritide, was also offered an opportunity to provide a scientific accuracy review of their data. Changes resulting from comments received were made on the basis of scientific and editorial merit.

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    Young-A Heo

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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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Heo, YA. Vosoritide in achondroplasia: a profile of its use. Drugs Ther Perspect 38, 417–422 (2022). https://doi.org/10.1007/s40267-022-00942-6

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