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BioMarin to Present Data Highlighting Significant Impact of VOXZOGO® (vosoritide) in Bone Health and Health-Related Quality of Life in Achondroplasia at 2024 International Conference on Children's Bone Health
New Investigator-Led Study Shows VOXZOGO Significantly Increased Bone Length While Maintaining Bone Strength Through 5 Years of Observation in Children with
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"New Investigator-Led Study Shows VOXZOGO Significantly Increased Bone Length While Maintaining Bone Strength Through 5 Years of Observation in Children with Achondroplasia\nPhase 2 and 3 Data on VOXZOGO Demonstrate Safety, Efficacy and Impact on Proportionality and Health-Related Quality of Life for Children with Achondroplasia\nNew Research Underscores Unmet Medical Needs of People with Hypochondroplasia\nSAN RAFAEL, Calif., June 17, 2024 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced the presentation of new data from an investigator-led analysis of the Phase 2 111-205 study, which demonstrate that children with achondroplasia treated with VOXZOGO® experienced increases in bone length while maintaining bone strength. The data will be presented at the 11th International Conference on Children's Bone Health (ICCBH) in Salzburg, Austria, June 22-25, 2024. BioMarin will also share the first results from a study underscoring the unmet medical needs of people living with hypochondroplasia.\n\n \n \n \n \n \n \n\n \n\"With thousands of children treated since approval, the breadth and depth of data collected from our trials demonstrate VOXZOGO's safety and benefit in children of all ages, including those under 5 who have received VOXZOGO for up to four years,\" said Hank Fuchs, M.D., president of Worldwide Research and Development at BioMarin. \"CNP is emerging as a natural and holistic regulator of statural development, and safety and efficacy data presented to date illustrate VOXZOGO's positive impact on bone growth, proportionality and quality of life. Based on this extensive evidence, we are accelerating the development of VOXZOGO across a multitude of growth-related conditions with significant unmet need.\"\nSignificant Impact on Bone Length, Strength, Proportionality and Health-Related Quality of Life in Achondroplasia \nResults from an investigator-led analysis of BioMarin's Phase 2 111-205 study found that children who received VOXZOGO (n=30) had significant increases in bone length and metacarpal cortical area after approximately five years of therapy, suggesting that treatment allowed the bone to remain strong as it lengthened.\n\"In order for children with achondroplasia to experience meaningful changes in daily functioning following treatment to enable growth, it is critical that bone robustness...