Biotechnology company BioMarin Pharmaceutical Inc (Nasdaq:BMRN) announced on Wednesday that new data from the Phase 3 CANOPY-HCH-3 study evaluating VOXZOGO (vosoritide) in children with hypochondroplasia were published in New England Journal of Medicine (NEJM) Evidence and presented at the European Society for Paediatric Endocrinology (ESPE) 2026 Annual Meeting.
The data included new results on the magnitude of benefit seen in children receiving VOXZOGO, including statistically significant improvements in annualised growth velocity (AGV), standing height, height Z-score and arm span after 52 weeks, with safety findings consistent with the established profile of VOXZOGO in achondroplasia.
According to BioMarin, the CANOPY-HCH-3 study showed that treatment with VOXZOGO led to a statistically significant improvement in AGV compared with placebo after 52 weeks, meeting the study's primary endpoint (least squares [LS] mean difference of 2.33 cm/year; p
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