BioMarin has debuted new data from a Phase III trial on its dwarfism drug, Voxzogo (vosoritide), which the company hopes will form the basis for the drug’s US approval in a subset of the genetic condition.
As per updated results from the late-stage CANOPY-HCH-3 study (NCT06455059), which were presented at the 2026 European Society for Paediatric Endocrinology (ESPE) meeting, Voxzogo-treated children with hypochondroplasia experienced a statistically significant 2.33cm uptick in yearly height growth compared with placebo-treated individuals after 52 weeks – meeting the trial’s primary endpoint.
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The drug also triggered a significant 2.35cm improvement in standing height and arm span, as well as height Z-score, which measures how far a child’s height is from the average for their age and sex.
On top of its potential to stimulate growth in height, Voxzogo numerically enhanced patient quality of life, while maintaining a consistent safety profile. Researchers identified no serious treatment-linked side effects in this study.
According to BioMarin’s EVP and chief R&D officer, Greg Friberg, these results mark the first time the company has been able to share “a comprehensive picture” of Voxzogo’s impact across multiple aspects of growth in children with hypochondroplasia – a rare condition characterised by stunted growth of the long bones in the body.
With this positive data in hand, BioMarin is eyeing a potential US approval for Voxzogo in hypochondroplasia, with the company having recently submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for Voxzogo in the condition.
If the drug were to secure the US regulatory greenlight, it could become the first targeted therapy to make a market debut in hypochondroplasia, which BioMarin estimates impacts between 8,500 and 23,000 people in the US.
It would also expand Voxzogo’s role in the treatment of dwarfism, which is currently limited to patients with achondroplasia who have open growth plates.
