BioMarin Announces New Three-Year VOXZOGO® (vosoritide) Data in Hypochondroplasia and BMN 333 Early Results at ENDO 2026
BioMarin presented three-year data showing sustained growth improvements and a favorable safety profile for VOXZOGO in children with hypochondroplasia, supporting a planned supplemental New Drug Application in the third quarter of 2026. In the Phase 2 extension study, mean height standard deviation score improved by 0.72 SD and annualized growth velocity increased significantly from baseline to year one.
Key figures
- Guidance
- sNDA submission for VOXZOGO in hypochondroplasia planned for Q3 2026; BMN 333 Phase 2/3 data update expected in 2027
- Bmn 333 max dose
- 500 μg/kg
- Voxzogo agv year1
- 7.24 cm/year
- Voxzogo agv baseline
- 4.27 cm/year
- Cnp exposure increase
- >13 times
- Voxzogo sds improvement
- 0.72 SD
- Hypochondroplasia patient pool
- 14,000
AI analysis
The rest of the AI analysis, red flags and sentiment are part of Signal8 Pro.
AI-generated analysis of a public disclosure. Not investment advice; verify against the original document.