BioMarin announces results from CANOPY clinical program evaluating VOXZOGO

BioMarin Pharmaceutical announced that positive data from the CANOPY clinical program evaluating VOXZOGO, or vosoritide, in children with achondroplasia and other genetic skeletal conditions will be presented at the 16th International Skeletal Dysplasia Society meeting, or ISDS, in Madrid, Sept. 18-21. These results include data showing that children with achondroplasia treated with VOXZOGO experienced meaningful improvements beyond height, such as in health-related quality of life, or HRQoL, and increased bone length while maintaining bone strength. Researchers will also present encouraging data from ongoing investigator-led studies investigating treatment in children with other genetic skeletal conditions, including hypochondroplasia and Noonan syndrome, as well as those with genetic variants often associated with idiopathic short stature such as aggrecan (ACAN) deficiency and heterozygous NPR2 mutations. Qualitative insights elucidating caregiver perspectives of children with achondroplasia revealed that VOXZOGO had positive impacts on HRQoL across several physical, emotional and social functioning domains. Notable physical functioning improvements reported included improved motor skills and self-care, which are particularly meaningful outcomes for children and families impacted by the condition. Psychosocial functioning outcomes included improved confidence and social interactions. Additional results from an investigator-led analysis of BioMarin’s Phase 2 111-205 study, previously shared at the 2024 International Conference on Children’s Bone Health, demonstrated that children who received VOXZOGO had significant increases in bone length and metacarpal cortical area after approximately five years of therapy, suggesting that treatment allowed the bone to remain strong as it lengthened.