BioMarin to Present VOXZOGO® Data on Health, Growth, and Bone Strength in Achondroplasia at 2024 ISDS Meeting

BioMarin Pharmaceutical Inc. recently revealed positive data from its CANOPY clinical program, which evaluates VOXZOGO® (vosoritide) for children with achondroplasia and other genetic skeletal conditions. The data will be presented at the 16th International Skeletal Dysplasia Society meeting (ISDS) in Madrid. The findings show that children with achondroplasia treated with VOXZOGO not only gained height but also exhibited significant improvements in health-related quality of life (HRQoL) and bone length without compromising bone strength. These results underscore the potential of VOXZOGO as a groundbreaking treatment for various genetic skeletal conditions, demonstrating its efficacy and safety.

VOXZOGO has now become the standard treatment for achondroplasia, thanks to its proven ability to enhance growth velocity and improve proportionality and quality of life for treated children. Dr. Ravi Savarirayan, an expert in skeletal biology and disease, highlighted the drug's impact on multiple domains of physical, emotional, and social functioning. Caregivers noted that VOXZOGO improved motor skills and self-care abilities, significantly enhancing the daily lives of children with achondroplasia. Psychosocial benefits included increased confidence and improved social interactions.

An investigator-led analysis of BioMarin's Phase 2 111-205 study, presented at the 2024 International Conference on Children's Bone Health, indicated that children who received VOXZOGO experienced significant increases in bone length and metacarpal cortical area after approximately five years of treatment, suggesting that the bone retained its strength as it grew.

Hank Fuchs, M.D., President of Worldwide Research and Development at BioMarin, expressed excitement about the potential of VOXZOGO to treat other genetic skeletal conditions through the CANOPY clinical program. He emphasized the drug's role as a central regulator of growth-related development and its transformative potential for treating achondroplasia and other related conditions.

Additional presentations at the ISDS meeting will reveal more about the efficacy of VOXZOGO in treating conditions beyond achondroplasia, such as hypochondroplasia and Noonan syndrome. Investigator-led studies have shown that VOXZOGO effectively increases annualized growth velocity and height in children with these conditions, with no new safety concerns reported.

Key presentations at ISDS will cover various aspects of VOXZOGO’s efficacy and safety, including its impact on bone growth and quality in osteogenesis imperfecta, growth improvements in children with hypochondroplasia, and preliminary data on its effects in children with RASopathies, ACAN and NPR2 deficiencies.

The CANOPY clinical program aims to explore the potential of VOXZOGO in treating a range of genetic skeletal conditions, including hypochondroplasia, Noonan syndrome, SHOX deficiency, Turner syndrome, and idiopathic short stature. The program includes several ongoing studies designed to address the unmet needs of children and families affected by these conditions.

VOXZOGO is a C-type natriuretic peptide (CNP) analog that acts as a positive regulator of the signaling pathway downstream of FGFR3 to promote endochondral bone growth. This mechanism is particularly vital for children with achondroplasia, where bone growth is inhibited due to a mutation in the FGFR3 gene. VOXZOGO is approved in several countries, including the U.S., Japan, and Australia, for increasing linear growth in children with achondroplasia who have open epiphyses. In the European Union, it is indicated for children aged four months and older with confirmed genetic testing.

To date, approximately 3,500 individuals worldwide have been treated with VOXZOGO, which is available in 44 markets globally. BioMarin continues to explore the drug's potential through its CANOPY clinical program, aiming to expand treatment options and improve the quality of life for children with various genetic skeletal conditions.