What is vosoritide?

Voxzogo (vosoritide) is a C-type natriuretic peptide (CNP) analog used to increase linear growth in children with achondroplasia. Voxzogo facilitates linear growth in pediatric patients with achondroplasia with open epiphyses. This indication was approved under accelerated approval based on improvements in annualized growth rates. Continued approval for this indication may be contingent upon verification and characterization of clinical benefit in confirmatory trials.

Achondroplasia is a genetic disorder of bone growth characterized by disproportionate short stature (dwarfism) and structural disorders of the long bones, spine, face, and skull base. It is caused by functional mutations in the fibroblast growth factor receptor 3 gene (FGFR3) that lead to impaired endochondral bone growth, which is negatively regulated. Voxzogo acts as a positive regulator of the FGFR3 downstream signaling pathway and promotes endochondral bone growth. Therefore, vosoritide, like CNP, acts as a positive regulator of endochondral bone growth as it promotes chondrocyte proliferation and differentiation.

In animal models with open growth plates, administration of vosoritide resulted in promotion of chondrocyte proliferation and differentiation, which resulted in widening of the growth plate and subsequent increase in bone growth. In mouse models of FGFR3-related chondrodysplasia, partial or complete normalization of the dwarfism phenotype is observed.

Voxzogo is administered by subcutaneous injection once daily. Common adverse reactions include injection site erythema, injection site swelling, vomiting, injection site urticaria, arthralgia, decreased blood pressure, and gastroenteritis. Voxzogo may cause serious adverse reactions, including risk of hypotension. To reduce this risk, patients should be well hydrated and eat enough food before receiving Voxzogo.