Voxzogo

Voxzogo is a medicine for treating achondroplasia in patients aged 4 months and older whose bones are still growing. Achondroplasia is an inherited disease caused by a mutation (change) in a gene called fibroblast growth-factor receptor 3 (FGFR3). The mutation affects growth of almost all bones in the body including the skull, spine, arms and legs, resulting in very short stature with a characteristic appearance. Achondroplasia is rare, and Voxzogo was designated an ‘orphan medicine’ (a medicine used in rare diseases) on 24 January 2013. Further information on the orphan designation can be found here: [ema.europa.eu/medicines/human/orphan-designations/EU3121094](https://beta.ema.europa.eu/en/medicines/human/orphan-designations/eu-3-12-1094). Voxzogo contains the active substance vosoritide.