BioMarin Pharmaceutical developed vosoritide (previously known as BMN 111) for children with achondroplasia, the most common form of dwarfism. Achondroplasia is an inherited autosomal-dominant chondrodysplasia caused by a gain-of-function mutation in fibroblast-growth-factor-receptor 3 (FGFR3). Vosoritide has been granted orphan drug designation in both the U.S. and Europe, and this drug participated in phase III clinical trials in children with achondroplasia. Vosoritide mimics C-type natriuretic peptide (CNP) and acts as a key regulator of longitudinal bone growth by downregulating the mitogen-activated protein kinase pathway, which is activated because of a FGFR3 gain-of-function mutation.