RT Journal Article
T1 Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study.
A1 Savarirayan, Ravi
A1 Tofts, Louise
A1 Irving, Melita
A1 Wilcox, William R
A1 Bacino, Carlos A
A1 Hoover-Fong, Julie
A1 Font, Rosendo Ullot
A1 Harmatz, Paul
A1 Rutsch, Frank
A1 Bober, Michael B
A1 Polgreen, Lynda E
A1 Ginebreda, Ignacio
A1 Mohnike, Klaus
A1 Charrow, Joel
A1 Hoernschemeyer, Daniel
A1 Ozono, Keiichi
A1 Alanay, Yasemin
A1 Arundel, Paul
A1 Kotani, Yumiko
A1 Yasui, Natsuo
A1 White, Klane K
A1 Saal, Howard M
A1 Leiva-Gea, Antonio
A1 Luna-González, Felipe
A1 Mochizuki, Hiroshi
A1 Basel, Donald
A1 Porco, Dania M
A1 Jayaram, Kala
A1 Fisheleva, Elena
A1 Huntsman-Labed, Alice
A1 Day, Jonathan R S
AB 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. 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. 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. Vosoritide treatment has safe and persistent growth-promoting effects in children with achondroplasia treated daily for two years.
YR 2021
FD 2021-08-02
LK http://hdl.handle.net/10668/18285
UL http://hdl.handle.net/10668/18285
LA en
DS RISalud
RD Apr 7, 2025