The US FDA has approved vosoritide (Voxzogo) daily injection for the treatment of children with achondroplasia. Indicated to increase linear growth in pediatric patients aged ≥5 years who have achondroplasia with open epiphyses.
Route: SC Inj.
MOA: Analog of C-type natriuretic peptide (CNP); this peptide binds to natriuretic-peptide receptor B (NPR B), which induces synthesis of cGMP molecules, which, in turn, inhibits the MAPK pathway.
Adverse Effects: Injection site erythema (75%), Injection site swelling (62%), Vomiting (27%), Injection site urticaria (25%), Arthralgia (15%), Decreased blood pressure (13%), Gastroenteritis (13%)
Source: Medscape