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.authorSavarirayan, Ravi
dc.contributor.authorTofts, Louise
dc.contributor.authorIrving, Melita
dc.contributor.authorWilcox, William R.
dc.contributor.authorBacino, Carlos A.
dc.contributor.authorHoover-Fong, Julie
dc.contributor.authorFont, Rosendo Ullot
dc.contributor.authorHarmatz, Paul
dc.contributor.authorRutsch, Frank
dc.contributor.authorBober, Michael B.
dc.contributor.authorPolgreen, Lynda E.
dc.contributor.authorGinebreda, Ignacio
dc.contributor.authorMohnike, Klaus
dc.contributor.authorCharrow, Joel
dc.contributor.authorHoernschemeyer, Daniel
dc.contributor.authorOzono, Keiichi
dc.contributor.authorAlanay, Yasemin
dc.contributor.authorArundel, Paul
dc.contributor.authorKotani, Yumiko
dc.contributor.authorYasui, Natsuo
dc.contributor.authorWhite, Klane K.
dc.contributor.authorSaal, Howard M.
dc.contributor.authorLeiva-Gea, Antonio
dc.contributor.authorLuna-Gonzalez, Felipe
dc.contributor.authorMochizuki, Hiroshi
dc.contributor.authorBasel, Donald
dc.contributor.authorPorco, Dania M.
dc.contributor.authorJayaram, Kala
dc.contributor.authorFisheleva, Elena
dc.contributor.authorHuntsman-Labed, Alice
dc.contributor.authorDay, Jonathan R. S.
dc.date.accessioned2023-02-21T12:42:36Z
dc.date.available2023-02-21T12:42:36Z
dc.date.issued2021-01-01
dc.description.abstractPurpose 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.issue12
dc.description.issueDEC
dc.description.pages2443-2447
dc.description.volume23
dc.identifier.doi10.1038/s41436-021-01287-7
dc.identifier.urihttps://hdl.handle.net/11443/2830
dc.identifier.urihttp://dx.doi.org/10.1038/s41436-021-01287-7
dc.identifier.wosWOS:000680348600002
dc.publisherSPRINGERNATURE
dc.relation.ispartofGENETICS IN MEDICINE
dc.titleSafe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study
dc.typeArticle

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