Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study
dc.contributor.author | Savarirayan, Ravi | |
dc.contributor.author | Tofts, Louise | |
dc.contributor.author | Irving, Melita | |
dc.contributor.author | Wilcox, William R. | |
dc.contributor.author | Bacino, Carlos A. | |
dc.contributor.author | Hoover-Fong, Julie | |
dc.contributor.author | Font, Rosendo Ullot | |
dc.contributor.author | Harmatz, Paul | |
dc.contributor.author | Rutsch, Frank | |
dc.contributor.author | Bober, Michael B. | |
dc.contributor.author | Polgreen, Lynda E. | |
dc.contributor.author | Ginebreda, Ignacio | |
dc.contributor.author | Mohnike, Klaus | |
dc.contributor.author | Charrow, Joel | |
dc.contributor.author | Hoernschemeyer, Daniel | |
dc.contributor.author | Ozono, Keiichi | |
dc.contributor.author | Alanay, Yasemin | |
dc.contributor.author | Arundel, Paul | |
dc.contributor.author | Kotani, Yumiko | |
dc.contributor.author | Yasui, Natsuo | |
dc.contributor.author | White, Klane K. | |
dc.contributor.author | Saal, Howard M. | |
dc.contributor.author | Leiva-Gea, Antonio | |
dc.contributor.author | Luna-Gonzalez, Felipe | |
dc.contributor.author | Mochizuki, Hiroshi | |
dc.contributor.author | Basel, Donald | |
dc.contributor.author | Porco, Dania M. | |
dc.contributor.author | Jayaram, Kala | |
dc.contributor.author | Fisheleva, Elena | |
dc.contributor.author | Huntsman-Labed, Alice | |
dc.contributor.author | Day, Jonathan R. S. | |
dc.date.accessioned | 2023-02-21T12:42:36Z | |
dc.date.available | 2023-02-21T12:42:36Z | |
dc.date.issued | 2021-01-01 | |
dc.description.abstract | 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 mu g/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. | |
dc.description.issue | 12 | |
dc.description.issue | DEC | |
dc.description.pages | 2443-2447 | |
dc.description.volume | 23 | |
dc.identifier.doi | 10.1038/s41436-021-01287-7 | |
dc.identifier.uri | https://hdl.handle.net/11443/2830 | |
dc.identifier.uri | http://dx.doi.org/10.1038/s41436-021-01287-7 | |
dc.identifier.wos | WOS:000680348600002 | |
dc.publisher | SPRINGERNATURE | |
dc.relation.ispartof | GENETICS IN MEDICINE | |
dc.title | Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study | |
dc.type | Article |
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