BioMarin presents new data for Voxzogo in achondroplasia and achondroplasia

BioMarin announced that positive early results from an investigator-sponsored Phase 2 study of Voxzogo in children with hypochondroplasia, will be presented at the 2024 American College of Medical Genetics and Genomics Meeting in Toronto, March 12-16. Researchers will also present data from Phase 2 and Phase 3 studies of the medicine in children with achondroplasia, including results that demonstrate Voxzogo’s positive impact on quality of life. In a Phase 2 study of Voxzogo in children with hypochondroplasia, annualized growth velocity increased from 5.12 cm/year during the observation period to 6.93 cm/year during the treatment period. No new safety signals were reported. A multinational observational study in children with hypochondroplasia is currently recruiting participants, and the company plans to enter the treatment phase, Phase 3, later this year. Data from two studies of Voxzogo in children greater than or equal to 5 years of age with achondroplasia demonstrated a sustained improvement in AGV for each year that the participants were treated. In the Phase 2 study with more than 7 years of follow up, the mean increase in growth across each year of age up to 16 years compared with untreated participants was 1.63 cm/year for boys and 1.33 cm/year for girls. Separate comparative analysis over a 7-year period showed an additional height gain of 11.03 cm with use of Voxzogo compared to matched untreated children over the same period. The same analyses were produced for the Phase 3 trial in which children with achondroplasia have a mean treatment follow-up of 4 years. The mean increase in growth across each year of age up to 17 years was 1.73 cm/year for boys and 1.46 cm/year for girls. Results from a separate analysis of the Phase 3 study showed that Voxzogo improved health-related quality of life among children with achondroplasia, particularly those associated with physical activities. These improvements were even more pronounced in children who grew more.