BioMarin Pharmaceutical Inc. (BMRN) has announced positive real-world evidence supporting the safety and efficacy of VOXZOGO® (vosoritide) in children with achondroplasia. The data, presented at the 62nd Annual European Society for Paediatric Endocrinology (ESPE) Meeting in Liverpool, England, included results from the European CrescNet registry and a real-world study in France.
CrescNet Registry Data
The achondroplasia module of the European CrescNet registry, involving 452 children across 30 centers in eight countries, demonstrated that VOXZOGO treatment outcomes were consistent with previous clinical trials. The median age at 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.
French Real-World Study
A real-world study in France, including 62 children, showed continued efficacy of VOXZOGO in 17 children above the age of 5. Data available for 18 months after initial treatment revealed that children who received VOXZOGO experienced an 8.76 cm increase in height, on average, and 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. The average annualized growth velocity was 5.85 cm/year. There were no discontinuations, and longer-term safety and effectiveness will continue to be monitored.
BioMarin's Perspective
"These real-world data further reinforce the value of VOXZOGO as the first and only approved treatment for children, including infants, with achondroplasia," said Greg Friberg, M.D., executive vice president and chief research & development officer at BioMarin. "We have now collected more than 6,000 patient-years of safety data through our industry-leading CANOPY clinical program, providing the scientific basis for the rapid development of VOXZOGO in new indications such as hypochondroplasia, where we hope to replicate our success in achondroplasia."
About VOXZOGO
VOXZOGO (vosoritide) is a C-type natriuretic peptide (CNP) analog that promotes endochondral bone growth by acting as a positive regulator of the signaling pathway downstream of FGFR3. It is approved in the U.S., Japan, and Australia to increase linear growth in children of all ages with achondroplasia with open epiphyses and is indicated in the EU for children 4 months and older whose epiphyses are not closed.
