BioMarin Pharmaceutical Inc. announced that the European Medicines Agency (EMA) validated its Type II Variation application to extend the indication for VOXZOGO® (vosoritide) for injection to treat children with achondroplasia under the age of 2. Validation confirms the submission is complete and begins the EMA's review process. The Company also announced that it submitted a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (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 VOXZOGO™ (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. Study Description: The 52-week phase 2 study enrolled 75 infants and young children with achondroplasia, aged zero to less than five years old (60 months). The study consists of three cohorts by age (24 months to less than 5 years, 6 months to less than 24 months, less than 6 months) and is followed by a subsequent open-label extension trial where all children receive active treatment.

Children in this study have completed a minimum three- or six-month baseline study to determine their respective baseline growth prior to entering the Phase 2 study. The objectives of the study are to evaluate safety, tolerability, and the effect of VOXZOGO on growth. The study also evaluated proportionality, functionality, quality of life, sleep apnea, and foramen magnum dimension, as well as the advent of major illnesses and surgeries which frequently occur in children with achondroplasia.