BioMarin Reports Favorable Clinical Trial Data for Growth Disorders

BioMarin Pharmaceutical has released new findings from its clinical trials, indicating sustained growth improvements in children with hypochondroplasia treated with VOXZOGO over three years. Furthermore, initial Phase 1 results for BMN 333 suggest potential weekly dosing for achondroplasia, with both treatments demonstrating a positive safety profile. These updates were presented at ENDO 2026.