Ravi Savarirayan et al demonstrated a sustained increase in the annualized growth velocity in children with achondroplasia on treatment with vosoritide for up to 42 months. The study revealed that vosoritide- a synthetic version of C-type natriuretic peptide, interferes with certain proteins that block bone growth, allowing the increase in growth rate. In the study, a total of 35 children were enrolled in four sequential cohorts to receive vosoritide at a once-daily subcutaneous dose of 2.5 μg per kilogram of body weight, 7.5 μg per kilogram, 15.0 μg per kilogram, or 30.0 μg per kilogram. After 6 months, the dose in cohort 1 and cohort 2 was gradually raised to 15.0μg per kilogram while patients in cohorts 3 and 4 continued to receive their initial doses. During the first 6 months of treatment, a dose-dependent increase in the annualized growth velocity was observed with vosoritide up to a dose of 15.0 μg per kilogram, and a sustained increase in the annualized growth velocity was observed at doses of 15.0 and 30.0 μg per kilogram for up to 42 months. The researchers reported that the administration of vosoritide was associated with side effects of the drug which were mostly mild.
Source: New England Journal of Medicine 4 July 2019 DOI: 10.1056/NEJMoa1813446 https://www.nejm.org/doi/full/10.1056/NEJMoa1813446