Voxzogo data shows bone gains in kids

BioMarin Pharmaceutical Inc. announced new data from studies of  Voxzogo  (vosoritide), demonstrating meaningful impact on tibial bowing in children with achondroplasia and investigational early efficacy results in other skeletal conditions. The 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.

Voxzogo Demonstrated Positive Impact on Tibial Bowing: An analysis of data presented at PES from the Voxzogo  Phase II CANOPY clinical studies in younger children assessed the impact of treatment on tibial bowing, an orthopedic complication and significant cause of pain in children with achondroplasia. Children who received Voxzogo  had a significant reduction in the magnitude of tibial bowing compared to children who received placebo. Furthermore, the researchers found that this improvement was sustained in children who received treatment for several years.

"Outcomes beyond height have long been prioritized by the dwarfism community," said Michael Hughes, Chair, Little People of America's (LPA) Biotech Industry Liaison Committee. "Tibial bowing can be a source of pain and surgical intervention for some children with achondroplasia, and this research reflects an encouraging shift toward addressing a meaningful outcome to the community."

"In children with achondroplasia, tibial bowing can be painful and often requires surgical intervention," said Klane White, M.D., pediatric orthopedic surgeon and Rose Brown Endowed Chair of Pediatric Orthopedics at Children's Hospital Colorado. "Through my clinical practice, I have observed meaningful improvements that early treatment initiation with Voxzogo has offered children with achondroplasia, including in proportionality, quality of life and now tibial bowing, which have the potential to improve physical well-being for these children into adulthood."

The latest results from the BioMarin Pharmaceutical Inc. announced new data from studies of Voxzogo  (vosoritide), demonstrating meaningful impact on tibial bowing in children with achondroplasia and investigational early efficacy results in other skeletal conditions. The 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.

The latest results from the Voxzogo Phase III clinical trial reporting the longest follow-up to date demonstrated that continuous and early treatment was associated with sustained increases in annualized growth velocity (AGV). For the first time, it was possible to model potential final height gain for children with achondroplasia if they were treated early and continuously from 6 months until final adult height (FAH). Modeling indicated that Voxzogo treatment could result in a height increase of 21.7 centimeters (cm) (95% Cl, 18.7-24.6) in girls and 26.4 cm (95% Cl, 22.9-29.8) in boys compared to untreated children. These data were presented today at ESPE as an oral presentation and add to the totality of evidence demonstrating that early and continuous treatment with Voxzogo maximizes clinical benefits for children with achondroplasia.

Hypochondroplasia Associated with Significant Increase in Comorbidities; Researchers also presented data shedding light on the comorbidities associated with hypochondroplasia, a genetically defined form of skeletal dysplasia for which Voxzogo  is currently being evaluated in clinical trials. In more than 600 adults and children with hypochondroplasia who were studied using real-world electronic health primary care medical record data in England, 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.

"This research is critical to shaping a deeper understanding of hypochondroplasia and underscores the importance of early diagnosis, which allows physicians to begin working with children and their families as soon as possible," said Greg Friberg, M.D., Executive Vice President and Chief Research & Development Officer at BioMarin. "We remain committed to developing new options for skeletal conditions, such as hypochondroplasia and Turner syndrome, and these early data continue to inform our clinical trials." 

In a prospective, single-center Phase II open-label clinical study in girls with Turner syndrome, researchers found that treatment with Voxzogo  was associated with a promising increase in AGV at 6 months compared to their baseline AGV. Importantly, AGV was increased even in children who were previously treated with human growth hormone and discontinued due to suboptimal results prior to enrollment in this study. Voxzogo  was also well-tolerated with safety results consistent with its known profile in achondroplasia.

BioMarin continues to advance development across its CANOPY clinical program for Voxzogo  in hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome and SHOX deficiency. In April, BioMarin completed enrollment in its pivotal Phase III study with Voxzogo  in hypochondroplasia, and the company is on track to share topline data in 2026, with potential launch in 2027 Voxzogo  Phase III clinical trial reporting the longest follow-up to date demonstrated that continuous and early treatment was associated with sustained increases in annualized growth velocity (AGV). For the first time, it was possible to model potential final height gain for children with achondroplasia if they were treated early and continuously from 6 months until final adult height (FAH). Modeling indicated that VOXZOGO treatment could result in a height increase of 21.7 centimeters (cm) (95% Cl, 18.7-24.6) in girls and 26.4 cm (95% Cl, 22.9-29.8) in boys compared to untreated children. These data were presented today at ESPE as an oral presentation and add to the totality of evidence demonstrating that early and continuous treatment with VOXZOGO maximizes clinical benefits for children with achondroplasia.