BioMarin reports early VOXZOGO treatment benefits for achondroplasia

BioMarin Pharmaceutical Inc. (NASDAQ: BMRN) announced new data showing that early treatment with VOXZOGO (vosoritide) in children with achondroplasia leads to improvements in body proportionality and arm span compared to untreated children.

The company presented findings at the 2026 American College of Medical Genetics and Genomics Annual Clinical Genetics Meeting demonstrating that children who started VOXZOGO before age 2 showed sustained improvements over four years of treatment.

For proportionality measures, children starting treatment under age 2 showed a least squares mean difference of -0.33 versus untreated children in year one, improving to -0.53 by year four. Among children who began treatment between 0.5 to under 2 years of age, average additional height gain after four years was 4.7 cm compared to untreated children.

The data also included real-world evidence from Japan, Europe and the United States. More than 5,000 infants and children across over 50 countries have received VOXZOGO to date, according to the company's press release.

In Japan, children under age 2 demonstrated mean height changes from baseline of 9.91 cm at 12 months and 15.62 cm at 24 months. French real-world study participants showed mean height gains of 16.7 cm over 36 months of treatment.

VOXZOGO is approved in the United States, Japan and Australia to increase linear growth in children with achondroplasia and open growth plates. In the United States, the indication was approved under accelerated approval based on improvement in annualized growth velocity.

Achondroplasia affects approximately one in 25,000 live births worldwide and is the most common form of skeletal dysplasia leading to disproportionate short stature.

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