BioMarin to Present VOXZOGO® Data on Bone Health and Quality of Life in Achondroplasia at 2024 Conference

BioMarin Pharmaceutical Inc. recently disclosed new data from a Phase 2 study highlighting the effectiveness of VOXZOGO® in children with achondroplasia, a rare genetic condition that impairs bone growth. The findings, presented at the International Conference on Children's Bone Health (ICCBH) in Salzburg, Austria, indicate that VOXZOGO significantly increases bone length while maintaining bone strength over a five-year period. This marks a promising advancement in the treatment of achondroplasia.

Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin, emphasized the comprehensive data collected from trials involving thousands of children since VOXZOGO's approval. The trials reveal that VOXZOGO's safety and efficacy extend across various age groups, including children under five years old, who have been treated for up to four years. Fuchs highlighted that CNP, the active component in VOXZOGO, is emerging as a pivotal regulator of growth, demonstrating positive impacts on bone growth, proportionality, and quality of life. Based on these findings, BioMarin is accelerating the development of VOXZOGO for other growth-related conditions with significant unmet needs.

The Phase 2 111-205 study involved 30 children with achondroplasia who received VOXZOGO. Results showed substantial increases in bone length and metacarpal cortical area, indicating that the bones remained robust as they lengthened. Dr. Cathleen Raggio, a pediatric orthopedic surgeon, noted that this is the first study demonstrating that VOXZOGO treatment allows bones to remain strong while they grow, which is vital for improving daily functioning in children with achondroplasia.

Further data to be presented at ICCBH, also shared at the 2024 Pediatric Endocrine Society Annual Meeting, underscore VOXZOGO's efficacy and safety. Phase 3 studies (111-301 and 111-302) suggest VOXZOGO can enhance health-related quality of life, particularly physical functioning, which is crucial for affected children and their families. Consistent positive effects on linear growth and proportionality improvement have been observed in children of all ages with growth potential, with follow-up extending up to four years. The safety profile of VOXZOGO remains consistent with previous findings.

BioMarin also highlighted the burden of hypochondroplasia, another growth-related condition. A retrospective study using electronic medical records from England revealed higher rates of respiratory, cardiovascular, orthopedic, and mental health-related events among individuals with hypochondroplasia compared to those without the condition. The study also noted increased healthcare utilization, including higher general practitioner visits, hospital admissions, and longer hospital stays. These findings underscore the substantial disease burden and highlight the need for proactive management and care linkage for individuals with hypochondroplasia.

BioMarin is expanding its clinical trials to explore VOXZOGO's potential in treating other growth-related conditions. A multinational observational study in children with hypochondroplasia is currently recruiting participants, with the first patient expected to be dosed in the Phase 3 trial in June 2024. Additionally, an observational study in children with idiopathic short stature (ISS) has begun, with a Phase 2 treatment arm set to commence later this year. Studies targeting other genetic short stature conditions, such as Turner syndrome, SHOX deficiency, and Noonan syndrome, are also planned.

These efforts reflect BioMarin's commitment to addressing the unmet needs of individuals with growth-related conditions through innovative therapies like VOXZOGO, which continues to show promise in improving the lives of affected children.