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Vosoritide: First Approval

Authors :
Sean T. Duggan
Source :
Drugs. 81:2057-2062
Publication Year :
2021
Publisher :
Springer Science and Business Media LLC, 2021.

Abstract

Vosoritide (VOXZOGO®) is a modified recombinant human C-type natriuretic peptide (CNP) analogue, being developed by BioMarin Pharmaceutical for the treatment of achondroplasia. Achondroplasia is caused by a gain-of-function mutation in the fibroblast growth factor receptor 3 gene (FGFR3), which is a negative regulator of bone growth. Vosoritide acts to restore chondrogenesis through its binding to natriuretic peptide receptor B (NPR-B), resulting in the inhibition of downstream signalling pathways of the overactive FGFR3 gene. Vosoritide was approved in August 2021 in the EU for the treatment of achondroplasia in patients aged ≥ 2 years whose epiphyses are not closed; the diagnosis of achondroplasia should be confirmed by appropriate genetic testing. The drug is also under regulatory review in the USA for the treatment of achondroplasia and clinical development is underway in several countries. This article summarizes the milestones in the development of vosoritide leading to this first approval for achondroplasia in patients aged ≥ 2 years whose epiphyses are not closed.

Details

ISSN :
11791950 and 00126667
Volume :
81
Database :
OpenAIRE
Journal :
Drugs
Accession number :
edsair.doi...........dde702007476d6167afc783a66586b75
Full Text :
https://doi.org/10.1007/s40265-021-01623-w