Journal Article

Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study

Tofts, Louise, Savarirayan, Ravi, Ginebreda, Ignacio, Ozono, Keiichi, Bacino, Carlos A., Mohnike, Klaus, Huntsman-Labed, Alice, Charrow, Joel, Day, Jonathan R. S., Irving, Melita, Wilcox, William R., Kotani, Yumiko, Saal, Howard M., Jayaram, Kala, Bober, Michael B., Hoernschemeyer, Daniel, Arundel, Paul, Porco, Dania M., Harmatz, Paul, Leiva-Gea, Antonio, Fisheleva, Elena, Rutsch, Frank, Yasui, Natsuo, White, Klane K., Basel, Donald, Hoover-Fong, Julie, Alanay, Yasemin, Polgreen, Lynda E., Mochizuki, Hiroshi, Luna-González, Felipe, Font, Rosendo Ullot

2021

Overview

Purpose Achondroplasia is caused by pathogenic variants in the fibroblast growth factor receptor 3 gene that lead to impaired endochondral ossification. Vosoritide, an analog of C-type natriuretic peptide, stimulates endochondral bone growth and is in development for the treatment of achondroplasia. This phase 3 extension study was conducted to document the efficacy and safety of continuous, daily vosoritide treatment in children with achondroplasia, and the two-year results are reported. Methods After completing at least six months of a baseline observational growth study, and 52 weeks in a double-blind, placebo-controlled study, participants were eligible to continue treatment in an open-label extension study, where all participants received vosoritide at a dose of 15.0 μg/kg/day. Results In children randomized to vosoritide, annualized growth velocity increased from 4.26 cm/year at baseline to 5.39 cm/year at 52 weeks and 5.52 cm/year at week 104. In children who crossed over from placebo to vosoritide in the extension study, annualized growth velocity increased from 3.81 cm/year at week 52 to 5.43 cm/year at week 104. No new adverse effects of vosoritide were detected. Conclusion Vosoritide treatment has safe and persistent growth-promoting effects in children with achondroplasia treated daily for two years.

• Publisher: Nature Publishing Group US,Elsevier Limited
• Subject: Achondroplasia - drug therapy / Achondroplasia - genetics / Biomedical and Life Sciences / Biomedicine / Brief Communication / Child / COVID-19 / Double-Blind Method / Drug dosages / Fibroblasts / Growth factors / Hospitals / Human Genetics / Humans / Laboratory Medicine / Medicine / Natriuretic Peptide, C-Type - analogs & derivatives / Natriuretic Peptide, C-Type - therapeutic use / Peptides / Treatment Outcome / Velocity