BioMarin Reveals New Data Showing How VOXZOGO® Boosts Quality Of Life For Kids With Achondroplasia

BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) has announced that at the 16th International Skeletal Dysplasia Society (ISDS) meeting in Madrid, running from September 18-21, 2024, they will present promising findings from their CANOPY clinical program. This program assesses the efficacy of VOXZOGO® (vosoritide) for children with achondroplasia and other genetic skeletal disorders. 

The new data highlights significant improvements not only in height but also in health-related quality of life (HRQoL) and bone length, while preserving bone strength. Additionally, researchers will share positive updates from ongoing studies exploring the treatment's impact on children with other genetic skeletal conditions, such as hypochondroplasia, Noonan syndrome, and genetic variants linked to idiopathic short stature, including aggrecan (ACAN) deficiency and heterozygous NPR2 mutations.

Ravi Savarirayan, M.D., Ph.D., group leader of Skeletal Biology & Disease at Murdoch Children's Research Institute in Melbourne, Australia, stated “VOXZOGO is now becoming the standard of care in achondroplasia, based on its proven effects on growth velocity, its safety profile and the clinical data demonstrating positive impact on proportionality and quality of life in treated children with achondroplasia.”

Qualitative feedback from caregivers of children with achondroplasia highlighted the positive effects of VOXZOGO on various aspects of health-related quality of life. These benefits were observed in physical, emotional, and social domains. Notably, improvements in physical abilities were reported, including enhanced motor skills such as reaching, walking, running, balancing, and cycling, as well as better self-care. These improvements hold particular significance for children and their families affected by the condition. 

Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin, mentioned “VOXZOGO is the first and only approved treatment for children with achondroplasia, providing families with an option that can be initiated in infants, and we are excited to continue investigating its possibilities in other genetic skeletal conditions through our CANOPY clinical program.”

He further added, “The data at ISDS continue to demonstrate the safety and efficacy of VOXZOGO, underscoring the clinical value of our approach with CNP as an effective, central regulator of growth-related development as our research seeks to transform the treatment landscape for families impacted by achondroplasia and other genetic skeletal conditions.”

On the psychosocial front, there were gains in confidence and social interactions. Furthermore, data from an investigator-led analysis of BioMarin's Phase 2 111-205 study, presented at the 2024 International Conference on Children's Bone Health, revealed that after around five years of receiving VOXZOGO, children (n=30) experienced substantial increases in bone length and metacarpal cortical area. This suggests that the treatment helps maintain bone strength as it grows longer.

At the ISDS conference, additional presentations covered the efficacy of VOXZOGO in ongoing studies targeting a range of genetic skeletal disorders beyond just achondroplasia. The safety profile of VOXZOGO remained consistent with what has been well-established in achondroplasia.

One investigator-sponsored study highlighted promising results, demonstrating continued improvements in mean annualized growth velocity (AGV) and height standard deviation (SD) among 24 children with various genetic skeletal conditions, such as Noonan syndrome and genetic variants linked to idiopathic short stature (including ACAN deficiency and heterozygous NPR2 mutations), over the course of one year.

Updated findings from an initial clinical trial of VOXZOGO in children with hypochondroplasia showed sustained enhancements in AGV and height SD specific to hypochondroplasia in 26 participants over the same period. The treatment's effectiveness was comparable to previous results in achondroplasia, with no new safety concerns reported.