BioMarin Voxzogo Phase 3 Success Triggers Label Expansion Plan BioMarin's Voxzogo met Phase 3 endpoints. Results exceeded company expectations. Label expansion filing is now planned. This targets hypochondroplasia. BioMarin announced positive Phase 3 results for Voxzogo (vosoritide) in children with hypochondroplasia (HCH). The trial successfully met its primary endpoint, demonstrating a statistically significant increase in annualized growth velocity compared to placebo. This outcome, exceeding internal projections, positions Voxzogo to address another form of dwarfism, expanding its therapeutic reach beyond achondroplasia. Following these robust results, BioMarin intends to file for a label expansion with both the FDA and EMA. This process will involve submitting a Supplemental New Drug Application (sNDA) to the FDA and a Type II variation application to the EMA, leveraging existing regulatory pathways for drug modifications. The company will compile comprehensive data, adhering to 21 CFR Part 314 for sNDA submissions. This success boosts Voxzogo's market potential, solidifying its position as a key growth driver for BioMarin. It offers a new treatment option for HCH patients, a population with limited alternatives. Competitors like Ascendis Pharma, developing TransCon CNP, will face increased pressure. BioMarin anticipates submitting regulatory filings by early Q4 2026.