Description
Savarirayan et al. reported on data from the 111-302 open label extension study, with the aim of exploring the long-term safety, tolerability, and efficacy of vosoritide treatment in children with achondroplasia. Vosoritide treatment was found to be well tolerated and resulted in sustained growth promoting effects in children with achondroplasia who were treated with daily subcutaneous injections of vosoritide for up to 6 years.
A total of 119 participants were enrolled in the 111-302 study out of the 121 participants from the 111-301 study. All participants received daily dose of vosoritide 15 µg/kg. Data from the CLARITY study was used to provide an external untreated population for use as reference data. Annualised growth velocity (AGV) in participants treated with vosoritide was found to be similar to the average stature population before puberty, with the greatest change in AGV being observed in patients with the lowest baseline AGV and steady increase in height Z-scores. A favourable safety profile was observed with continuous vosoritide treatment up to 6 years, with no long-term harms or deaths occurring.
References:
Savarirayan R, Irving M, Wilcox WR, Bacino CA, Hoover-Fong JE, Harmatz P, Polgreen LE, Palm K, Prada CE, Kubota T, Arundell P, Kotani Y, Leiva-Gea A, Bober MB, Hecht JT, Legare JM, Lawrinson S, Low A, Sabir I, Huntsman-Labed A, Day JRSGenet Med. 2024.
Med. 2024.
doi:10.1016/j.medj.2024.11.019.