REVIEW ON VOSORITIDE: ACHLONDROPLASIA

BioMarin Pharmaceutical is developing Vosoritide, a modified recombinant human C-type natriuretic peptide (CNP) analogue for the treatment of achondroplasia. A gain-of-function mutation in the fibroblast growth factor receptor 3 gene (FGFR3), a negative regulator of bone growth, causes achondroplasia. Vosoritide works to restore chondrogenesis by attaching to the natriuretic peptide receptor B (NPR-B), which inhibits the hyperactive FGFR3 gene’s downstream signalling pathways. In August 2021, the European Union approved Vosoritide for the treatment of achondroplasia in children under the age of two whose epiphyses are not closed; the diagnosis of achondroplasia should be validated by genetic testing. In the United States, the medication is also undergoing regulatory assessment for the treatment of achondroplasia and clinical trials. Several countries are in the process of developing. The milestones in the development of vosoritide that led to this first approval for achondroplasia in children under the age of two whose epiphyses are not closed are summarized in this article. Vosoritide is a medication that was designed to treat achondroplasia and has shown to improve the growth rate of children with the condition. Achondroplasia, often known as dwarfism, is a skeletal dysplasia (a disorder affecting children’s bones and joints that causes them to grow abnormally). Achondroplasia currently has no approved treatments, with the exception of growth hormone in Japan. Clinical studies are required after many other processes in the development of a new treatment to determine how effectively the drug works and whether it is safe.