It does so by inhibiting the development (cell proliferation and differentiation) of chondrocytes, the cells that produce and maintain the cartilaginous matrix which is also necessary for bone growth. Children with achondroplasia have one of several possible FGFR3 mutations resulting in constitutive (permanent) activity of this receptor, resulting in overall reduced chondrocyte activity and thus bone growth.The protein C-type natriuretic peptide (CNP), naturally found in humans, reduces the effects of over-active FGFR3. Vosoritide is a CNP analogue with the same effect but prolonged half-life, allowing for once-daily administration. Chemistry
Vosoritide is an analog of CNP. It is a peptide consisting of the amino acids proline and glycine plus the 37 C-terminal amino acids from natural human CNP. The complete peptide sequence is

PGQEHPNARK YKGANKKGLS KGCFGLKLDR IGSMSGLGCwith a disulfide bridge between positions 23 and 39 (underlined). The drug must be administered by injection as it would be rendered ineffective by the digestive system if taken by mouth. History
Vosoritide was developed by BioMarin Pharmaceutical and, being the only available causal treatment for this condition, got orphan drug status in the US as well as the European Union. It was in Phase II clinical trials in around 2015.The safety and efficacy of vosoritide in improving growth were evaluated in a year-long, double-blind, placebo-controlled, phase III study in participants five years and older with achondroplasia who have open epiphyses. In the study, 121 participants were randomly assigned to receive either vosoritide injections under the skin or a placebo.
Mannosidosis is a deficiency in mannosidase, an enzyme.There are two types:

Alpha-mannosidosis
Beta-mannosidosis

See also
Swainsonine


== References ==