BioMarin Pharmaceutical Inc. (BMRN) has reported promising and consistent findings from various real-world evidence studies concerning VOXZOGO (vosoritide) for children with achondroplasia. These findings were shared at the 62nd Annual European Society for Paediatric Endocrinology (ESPE) Meeting, held in Liverpool, England, from November 16-18, 2024.
The company highlighted that long-term real-world data from the achondroplasia module of the European CrescNet registry, which involved 452 children across 30 centers in eight countries, demonstrated treatment outcomes with VOXZOGO were in alignment with previously reported clinical trials. The median age at the point of enrollment was 6.12 years. Of the 143 participants who had been treated with VOXZOGO for 12 months, the average height increase was 6.36 centimeters, and there was a 0.7 enhancement in the height Z-score compared to an achondroplasia reference population. For those 73 children treated over 24 months, there was an observed average height increase of 11.86 cm and a height Z-score improvement of 1.15, referencing the same standard population.
Additionally, a real-world study conducted in France including 62 children showed results from 17 children over 5 years old, with data available 18 months post-treatment, continued to demonstrate the efficacy of VOXZOGO. Children on VOXZOGO treatment experienced, on average, an 8.76 cm height increment and a mean Z-score improvement of 0.56 relative to an untreated achondroplasia population. This was complemented by a 0.44 Z-score enhancement compared to a standard United States population, indicating notable growth progress in comparison with untreated counterparts. The average yearly growth rate was calculated at 5.85 cm/year, showcasing a marked improvement in growth development over time. No discontinuations were reported, and continuous monitoring of long-term safety and efficacy is planned.