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BioMarin to Share Updated Data at 2023 American College of Medical Genetics and Genomics (ACMG) Meeting Demonstrating Commitment to Understanding Long-Term Benefit of VOXZOGO® in Children with Achondroplasia
New Analyses from Phase 2 and Phase 3 Extension Studies Continue to Demonstrate Sustained Improvement in Growth with no Change in Safety Profile SAN RAFAEL,
About this update from Biomarin Pharmaceutical Inc.
[{"type":"text","content":"New Analyses from Phase 2 and Phase 3 Extension Studies Continue to Demonstrate Sustained Improvement in Growth with no Change in Safety Profile \nSAN RAFAEL, Calif., March 14, 2023 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN), a global biotechnology company dedicated to transforming lives through genetic discovery, will present updated data demonstrating the long-term benefit of treatment with VOXZOGO® (vosoritide) and new observational data on disease burden in children with achondroplasia. These data will be shared this week in an oral presentation and five posters at the 2023 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting in Salt Lake City, Utah.\n\n \n \n \n \n \n \n\n \nThe data will include seven-year results from the open-label, Phase 2 extension study in children with achondroplasia, providing further evidence that improvement in growth velocity with VOXZOGO is sustained as children grow older. Mean age- and sex-specific Annualized Growth Velocities (AGVs) in children treated with VOXZOGO were greater than corresponding mean AGVs in untreated children of the same age and sex and at all ages. When looking across all ages for the entire seven-year period, the mean (SD) difference in AGV versus untreated children was 1.90 (0.51) cm/year for boys and 1.36 (0.63) cm/year for girls in this analysis. A trend toward improvement in body proportion (upper:lower body segment ratio) over time continued to be apparent as children grew older.\nResults from up to 3.5 years of the open-label extension of BioMarin's Phase 3 pivotal study of VOXZOGO also will be presented. Earlier data from this study supported the drug's approval in the United States, Europe, Brazil, Australia and Japan. In children on VOXZOGO, the mean (standard deviation [SD]) AGV improved from 4.26 (1.54) cm/year at baseline to 5.57 (0.76) cm/year after three years of treatment.\nIn both studies VOXZOGO remained well tolerated with no change in its adverse event profile.\n\"We are encouraged to see that as children remain on treatment, the growth promoting effect continues. We are observing sustained increases in growth velocity year on year,\" said Dr. Melita Irving, MBBS, consultant clinical geneticist at Guy's and St. Thomas' NHS Foundation Trust in London. \"The trend toward improving proportionality also ...