BioMarin Pharmaceutical Inc. 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. The 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.

Results 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.

In both studies VOXZOGO remained well tolerated with no change in its adverse event profile. VOXZOGO addresses the root cause of achondroplasia, the most common form of disproportionate short stature. It is the first U.S. Food and Drug Administration (FDA) and European Medicines Agency (EMA) approved treatment for children with achondroplasia with open epiphyses (bone growth plates).

In January, the EMA validated BioMarin's application for extension of indication for VOXZOGO to treat children with achondroplasia under the age of 2 and earlier this month, the FDA accepted the company's Supplemental New Drug Application for children under 5, setting a PDUFA target action date of October 21, 2023.