VOXZOGO (vosoritide) is a recombinant analog of C-type natriuretic peptide (CNP), designed to address the underlying pathophysiology of achondroplasia, the most common form of disproportionate short stature. By binding to natriuretic peptide receptor-B (NPR-B), vosoritide antagonizes the overactive fibroblast growth factor receptor 3 (FGFR3) signaling pathway, which is responsible for impaired endochondral bone growth in achondroplasia. This mechanism promotes chondrocyte proliferation and differentiation, thereby enhancing linear bone growth.

Clinically, VOXZOGO is administered as a once-daily subcutaneous injection, with the dosage individualized according to the patient’s actual body weight. The drug is supplied as a lyophilized powder requiring reconstitution prior to administration. The most common adverse reactions include injection site reactions, vomiting, arthralgia, decreased blood pressure, and elevated alkaline phosphatase. VOXZOGO is not recommended for patients with significant renal impairment (eGFR < 60 mL/min/1.73 m²). Use in pregnancy, lactation, and other special populations is either not recommended or not specified in the specification.