Early initiation of therapy with vosoritide: case report. Description of the first Italian patient affected by achondroplasia treated with vosoritide before 2 years of age.

Achondroplasia, the most common skeletal dysplasia, is mainly characterized by disproportionate short stature. Affected patients present a reduced linear growth, and the greatest decrease in growth velocity is evident in infancy. Vosoritide is the first approved drug to improve linear growth in children affected by achondroplasia. Since 2023, it is approved in children from birth by the FDA, and from 4 months of age by the EMA. However, until October 2025, the prescription and reimbursement of vosoritide in Italy was only possible starting from 2 years of age. We present the case of the first patient affected by achondroplasia treated with vosoritide in Italy before 2 years of age, managed thanks to the approval by the local rare diseases commission. Therapy with vosoritide was initiated at 7 months of age. The infant presented no severe adverse effects. She completed 12 months of therapy, at which time an improvement of +0.35 SD in length, according to growth curves specific for girls affected by achondroplasia, was documented. This result is in line with previous studies and underlines the importance of an early therapy initiation.