BioMarin Reports New Positive CANOPY Studies Data For Voxzogo

(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) Monday announced new data from studies of Voxzogo in children with achondroplasia and other skeletal conditions.

Voxzogo is already approved for the treatment of children with achondroplasia, a genetic bone growth disorder that leads to short-limbed dwarfism.

New data from the Phase 2 CANOPY studies showed that treatment with Voxzogo improved tibial bowing, a common cause of pain and impaired function.

These data were presented at the 2025 Joint Congress of the European Society for Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) in Copenhagen, Denmark and will be shared at the Pediatric Endocrine Society Annual Meeting (PES) in National Harbor, Maryland.

Researchers also presented data providing information about the comorbidities associated with hypochondroplasia, a genetically defined form of skeletal dysplasia for which Voxzogo is currently being evaluated. In more than 600 adults and children with hypochondroplasia comorbidity event rates were more common compared to people without hypochondroplasia. The condition was also associated with an increase in doctor visits, surgeries and orthopedic interventions.

A Phase 3 study of Voxzogo in hypochondroplasia is underway, with topline data expected in 2026.

The drug is also being evaluated in idiopathic short stature, Noonan syndrome, Turner syndrome and SHOX deficiency.