BioMarin Pharmaceutical Inc. presented new real-world evidence supporting the efficacy and safety of VOXZOGO® (vosoritide) in children with achondroplasia at the 62nd Annual European Society for Paediatric Endocrinology (ESPE) Meeting in Liverpool, England. The data, derived from multiple studies, reinforce the drug's potential to improve height in children with this genetic condition.
Real-World Evidence from European CrescNet Registry
Longitudinal data from the achondroplasia module of the European CrescNet registry, encompassing 452 children across 30 centers in eight countries, demonstrated that VOXZOGO treatment outcomes aligned with previous clinical trials. The median age at enrollment was 6.12 years. Among 143 participants treated with VOXZOGO for 12 months, the average height increase was 6.36 centimeters (cm), accompanied by a height Z-score improvement of 0.7 compared to an achondroplasia reference population. For 73 participants treated for 24 months, the average height increase was 11.86 cm, with a height Z-score improvement of 1.15 compared to the same reference population.
French Real-World Study
A real-world study in France, involving 62 children, showed continued efficacy of VOXZOGO in 17 children above the age of 5. These children experienced an average height increase of 8.76 cm over 18 months. The mean Z-score improvement was 0.56 compared to an untreated natural history population with achondroplasia and 0.44 compared to a general U.S. population. The average annualized growth velocity was 5.85 cm/year, indicating a substantial improvement in growth-related development over time. There were no discontinuations reported, and longer-term safety and effectiveness will continue to be monitored.
BioMarin's Perspective
"These real-world data further reinforce the value of VOXZOGO as the first and only approved treatment for children, including infants, with achondroplasia," said Greg Friberg, M.D., executive vice president and chief research & development officer at BioMarin. "We have now collected more than 6,000 patient-years of safety data through our industry-leading CANOPY clinical program, providing the scientific basis for the rapid development of VOXZOGO in new indications such as hypochondroplasia, where we hope to replicate our success in achondroplasia."
Ongoing Research
BioMarin is also investigating VOXZOGO in other genetic skeletal conditions through the CANOPY clinical program. This includes studies in hypochondroplasia, idiopathic short stature, Noonan syndrome, Turner syndrome, and SHOX deficiency.
