Safe and persistent growth-promoting effects of vosoritide in children with achondroplasia: 2-year results from an open-label, phase 3 extension study.
Journal
Genetics in medicine : official journal of the American College of Medical Genetics
ISSN: 1530-0366
Titre abrégé: Genet Med
Pays: United States
ID NLM: 9815831
Informations de publication
Date de publication:
12 2021
12 2021
Historique:
received:
01
05
2021
accepted:
12
07
2021
revised:
08
07
2021
pubmed:
4
8
2021
medline:
23
3
2022
entrez:
3
8
2021
Statut:
ppublish
Résumé
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.
Identifiants
pubmed: 34341520
doi: 10.1038/s41436-021-01287-7
pii: S1098-3600(21)05439-3
pmc: PMC8327889
doi:
Substances chimiques
Natriuretic Peptide, C-Type
127869-51-6
vosoritide
7SE5582Q2P
Types de publication
Clinical Trial, Phase III
Journal Article
Randomized Controlled Trial
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
2443-2447Informations de copyright
© 2021. The Author(s).
Références
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