BioMarin Pharmaceutical has announced that a phase 3 study evaluating treatment with Voxzogo® (vosoritide) in children with hypochondroplasia met its primary endpoint, demonstrating a statistically significant increase in the change from baseline at week 52 in annualised growth velocity (AGV) compared to placebo (LS mean difference +2.33 cm/yr, p<0.0001).
Children who received Voxzogo also showed a statistically significant increase in standing height (p<0.0001) and height Z-score (p<0.0001) versus placebo after one year of treatment.
Additionally, significant improvements in arm span were demonstrated (p=0.004), a key prespecified secondary endpoint in the study.
These positive findings could have important implications for children with hypochondroplasia, offering improvements in reach, daily activities and independence, which have been highlighted as meaningful outcomes by the community.
‘For the first time, a pivotal phase 3 study in hypochondroplasia has demonstrated impressive gains in growth, including improvements in arm span, highlighting Voxzogo’s potential to fundamentally change the treatment landscape for this condition’, said Greg Friberg, MD, Executive Vice President and Chief Research & Development Officer at BioMarin. A supplemental New Drug Application (sNDA) submission to the US Food and Drug Administration (FDA) is planned for the third quarter of 2026, followed by submissions to the European Medicines Agency (EMA) and other regional health authorities.
