EMA validates application for extension of indication for Voxzogo for injection to treat children with achondroplasia under the age of 2

The Company also announced that it submitted a supplemental New Drug Application (sNDA) to the FDA to treat children with achondroplasia under the age of 5. Approval of the submissions would mean Voxzogo could potentially be prescribed as early as birth with more than 1,000 additional children eligible for treatment for achondroplasia, the most common form of disproportionate short stature in humans. The Company anticipates action by health authorities in the second half of 2023.

The supplemental marketing applications are based on the outcomes from a Phase 2 randomized, double-blind, placebo-controlled clinical trial to evaluate the safety and efficacy of oxzogo (vosoritide) for injection in infants and children aged 3 months to less than five years old.

Voxzogo is the first FDA and EMA approved treatment for children with achondroplasia with open epiphyses (bone growth plates). 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 modified C-type natriuretic peptide (CNP), directly targets the underlying pathophysiology of achondroplasia by down regulating fibroblast growth factor receptor 3 (FGFR3) signaling and consequently promoting endochondral bone formation.