#USA #San Rafael #BioMarin #VOXZOGO #achondroplasia

BioMarin Unveils New Data for VOXZOGO

BioMarin Pharmaceutical Inc. has recently released new findings from studies on its medication VOXZOGO® (vosoritide), aimed at treating achondroplasia and other skeletal conditions in children. These significant insights were shared during two prominent international conferences: the 2025 Joint Congress of the European Society for Paediatric Endocrinology (ESPE) and the European Society of Endocrinology (ESE) held in Copenhagen, Denmark, followed by the Pediatric Endocrine Society Annual Meeting (PES) in National Harbor, Maryland.

Improvements Noted in Tibial Bowing

One of the standout findings from the studies highlights that treatment with VOXZOGO has resulted in a significant reduction in tibial bowing—a common orthopedic complication in children with achondroplasia. This issue frequently leads to discomfort and at times requires surgical interventions. The analysis represented data from the Phase 2 CANOPY clinical studies specific to younger children. The results indicate that those receiving VOXZOGO experienced a notable improvement in tibial bowing compared to those given a placebo, and this positive effect persisted in children who continued treatment over a number of years.

Michael Hughes, Chair of the Little People of America’s Biotech Industry Liaison Committee, remarked, “The findings underscore a much-needed focus on outcomes beyond just height for the dwarfism community. Addressing conditions like tibial bowing is vital, as it greatly impacts the quality of life for affected children.”

In addition, Dr. Klane White, a pediatric orthopedic surgeon at Children’s Hospital Colorado, emphasized that the observed improvements from VOXZOGO align with his clinical experiences. He noted improvements in proportionality, quality of life, and overall physical well-being for children into adulthood as encouraging outcomes of early treatment.

Sustained Growth Velocity Gains

Further results from the VOXZOGO Phase 3 clinical trial suggest that ongoing and early treatment leads to sustained gains in annualized growth velocity (AGV). The research quantified potential height increases in children treated early and continuously from the age of 6 months until reaching their final adult height. The projections estimate a height gain of about 21.7 cm in girls and 26.4 cm in boys when comparing against untreated children, highlighting the potential long-term benefits of initiating treatment at an early age.

Hypochondroplasia Insights

In addition to findings on achondroplasia, researchers also presented new data regarding hypochondroplasia—a genetically defined skeletal dysplasia. A study utilizing real-world health data from over 600 individuals indicated that those with hypochondroplasia experience higher rates of comorbidities, increased doctor visits, and more frequent surgical interventions compared to the general population. Greg Friberg, MD, BioMarin's Executive Vice President, stressed the necessity for early diagnosis, which allows timely interventions and management for these children and their families.

Future Directions for BioMarin

BioMarin remains dedicated to developing new treatments targeted at various skeletal conditions, including hypochondroplasia and Turner syndrome. The company continues its work across its CANOPY clinical program, focusing on multiple conditions such as idiopathic short stature and SHOX deficiency. Recent efforts have seen completion of enrollment in pivotal Phase 3 studies for VOXZOGO in hypochondroplasia, with plans to disclose topline data by 2026 and a potential launch eyed for 2027.

Presentation Highlights

Key presentations from BioMarin during these meetings included:

• - The long-term effects of VOXZOGO on height growth in children with achondroplasia.
• - Studies examining the medical impact of hypochondroplasia on children and adults based on a 22-year retrospective analysis.

Understanding Achondroplasia and VOXZOGO

Achondroplasia is recognized as the most common form of skeletal dysplasia, stemming from an FGFR3 gene mutation which hinders bone growth. VoXzogo is designed to counter this by encouraging endochondral bone growth, making it a vital treatment for children exhibiting the condition. Currently approved in several countries, VOXZOGO provides hope for many families affected by achondroplasia.

To learn more about VOXZOGO, individuals can reach out to BioMarin's RareConnections® Case Managers for support and additional resources. Monitoring patient safety and treatment efficacy remains a priority as BioMarin pushes forward in its mission to enhance the quality of life for those impacted by skeletal disorders.