BioMarin : Real-World Evidence Supports Safety, Efficacy Of VOXZOGO In Children With Achondroplasia

(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) announced positive and consistent results from multiple real-world evidence studies of VOXZOGO (vosoritide) in children with achondroplasia. The results were presented at the 62nd Annual European Society for Paediatric Endocrinology (ESPE) Meeting in Liverpool, England, November 16-18, 2024.

According to the company, real-world longitudinal data from the achondroplasia module of the European CrescNet registry involving 452 children at 30 centers across eight countries showed outcomes of treatment with VOXZOGO were consistent with previously reported clinical trials. The median age at time of enrollment was 6.12 years. Among 143 participants treated with VOXZOGO for 12 months, the average height increase was 6.36 centimeters (cm), with a height Z-score improvement of 0.7 compared to an achondroplasia reference population. For 73 participants treated for 24 months, the average height increase was 11.86 cm, with a height Z-score improvement of 1.15 compared to the same reference population.

In addition, in a real-world study in France that included 62 children, results from 17 children above the age of 5 (for whom data is available for 18 months after initial treatment) showed continued efficacy of VOXZOGO. The children who received VOXZOGO experienced an 8.76 cm increase in height, on average, and experienced a mean 0.56 improvement in Z-score compared to an untreated natural history population with achondroplasia and a 0.44 improvement in Z-score compared to a general U.S. population, indicating progress in height compared to the untreated population. The average annualized growth velocity was 5.85 cm/year, representing a substantial improvement in growth-related development over time. There were no discontinuations, and longer-term safety and effectiveness will continue to be monitored.

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