BioMarin reports VOXZOGO trial data showing 4.7 cm additional height gain at four years in children with achondroplasia treated before age 2

Reuters
Mar 12
BioMarin reports VOXZOGO trial data showing 4.7 cm additional height gain at four years in children with achondroplasia treated before age 2

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.

Disclaimer: This news brief was created by Public Technologies (PUBT) using generative artificial intelligence. While PUBT strives to provide accurate and timely information, this AI-generated content is for informational purposes only and should not be interpreted as financial, investment, or legal advice. BioMarin Pharmaceutical Inc. published the original content used to generate this news brief via PR Newswire (Ref. ID: 202603120905PR_NEWS_USPR_____SF07983) on March 12, 2026, and is solely responsible for the information contained therein.

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