vosoritide

Vosoritide is a recombinant peptide drug and an analog of C-type natriuretic peptide (CNP) used to increase linear growth in children with achondroplasia, a genetic disorder that results in short stature due to impaired bone growth. Vosoritide works by binding to the natriuretic peptide receptor B (NPR-B), which antagonizes the downstream signaling of fibroblast growth factor receptor 3 (FGFR3). This action inhibits the MAPK/ERK pathway at the level of RAF-1 kinase, promoting chondrocyte proliferation and differentiation, thereby enhancing endochondral bone growth. Vosoritide is administered as a daily subcutaneous injection and was developed as a peptidase-resistant formulation for improved therapeutic efficacy. It is approved under the brand name Voxzogo for use in children with open epiphyses (growth plates) who have achondroplasia[1][2][3][4][6][7].

Brand names

Voxzogo

Other names

C-type natriuretic peptide analogCNP analog