BioMarin reported new data from studies of VOXZOGO (vosoritide) in children with achondroplasia that will be presented at the 2026 ACMG Annual Clinical Genetics Meeting. In a study of children who started treatment before age 2, the least squares mean difference in upper-to-lower body segment ratio versus untreated children was −0.33 at year 1 (p=0.0005) and −0.53 at year 4 (p<0.0001). Among more than 20 children who began VOXZOGO between 0.5 to <2 years of age, average additional height gain after four years was 4.7 cm (p=0.0002) versus untreated children. BioMarin also cited real-world findings in Japan showing mean height change from baseline of 9.91 cm at 12 months and 15.62 cm at 24 months in children under age 2 receiving VOXZOGO.

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