Vosoritide
生产企业：BioMarin Pharmaceutical Inc.
Vosoritide is an analogue of C-type naturietic peptide used to promote bone growth in pediatric patients with achondroplasia.
Vosoritide is an analogue of C-type naturietic peptide used to promote bone growth in pediatric patients with achondroplasia.
Achondroplasia is a congenital disease resulting from a missense mutation in the fibroblast growth factor receptor 3 (FGFR3) gene, resulting in a gain-of-function that negatively regulates endochondral bone growth. Under normal conditions, FGFR3 is expressed during both embryonic and postnatal development, but serves a different role in each. During initial development, FGFR3 signaling promotes proliferation of chondrocytes (i.e. growth), whereas postnatal skeletal growth is actually inhibited by FGFR3 - as a result, the pathologic activation of FGFR3 observed in patients with achondroplasia leads to suppressed pre-pubertal skeletal growth. Vosoritide is an analog of C-type natriuretic peptide (CNP), a signaling molecule that appears primarily responsible for the stimulation of chondrocytes and the growth of long bones. The binding of CNP (or vosoritide) with its corresponding receptor, NPR-B, results in a signaling cascade that ultimately inhibits the MAPK/ERK pathway via inhibition of RAF-1 and stimulates the proliferation and differentiation of chondrocytes. This activity serves to antagonize the downstream signaling resulting from FGFR3 and its resultant effects on bone growth.
Vosoritide is indicated for the promotion of linear growth in pediatric patients with achondroplasia who are 5 years of age and older with open epiphyses. This indication is approved under accelerated approval based on an improvement in annualized growth velocity. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
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