Voxzogo is the first FDA approved treatment for children with achondroplasia. In patients with achondroplasia, endochondral bone growth, an essential process by which bone tissue is created, is negatively regulated due to a gain of function mutation in fibroblast growth factor receptor 3 gene (FGFR3). Voxzogo, a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
Figure 1: Role of FGFR3 in down-regulation of bone growth in the chondrocyte cell. M1, M2, M3, and M4 are the messengers that relay the down-regulation signal (their actual abbreviated names are Ras, Raf1, MEK, and ERK). CNP (as well as Vosoritide and TransCon CNP) act by blocking the signal at M2. Meclizine acts by blocking the signal between M3 and M4. Recifercept is a “decoy” that prevents binding of FGF to FGFR3. RBM-007 is an aptamer oligonucleotide that binds to FGF and prevents its binding to FGFR3. Vofatamab is a monoclonal antibody against FGFR3, while Infigratinib is a tyrosine kinase inhibitor that prevents FGFR3 from initiating the down-regulation signal. https://i0.wp.com/thechandlerproject.org/wp-content/uploads/2021/02/Screen- Shot-2021-02-16-at-8.00.42-PM.png?resize=1024%2C615&ssl=1
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Voxzogo is the first FDA approved treatment for children with achondroplasia. In patients with achondroplasia, endochondral bone growth, an
essential process by which bone tissue is created, is negatively regulated
due to a gain of function mutation in fibroblast growth factor receptor 3
gene (FGFR3). Voxzogo, a C-type natriuretic peptide (CNP) analog, represents a new class of therapy, which acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth.
Figure 1: Role of FGFR3 in down-regulation of bone growth in the chondrocyte cell.
M1, M2, M3, and M4 are the messengers that relay the down-regulation signal (their actual abbreviated names are Ras, Raf1, MEK, and ERK). CNP (as well
as Vosoritide and TransCon CNP) act by blocking the signal at M2. Meclizine acts by blocking the signal between M3 and M4. Recifercept is a “decoy”
that prevents binding of FGF to FGFR3. RBM-007 is an aptamer oligonucleotide that binds to FGF and prevents its binding to FGFR3. Vofatamab is a
monoclonal antibody against FGFR3, while Infigratinib is a tyrosine kinase
inhibitor that prevents FGFR3 from initiating the down-regulation signal.
https://i0.wp.com/thechandlerproject.org/wp-content/uploads/2021/02/Screen-
Shot-2021-02-16-at-8.00.42-PM.png?resize=1024%2C615&ssl=1