BioMarin Pharmaceutical Inc. reports new long-term data for VOXZOGO showing improved arm span, stable bone health, and early hypochondroplasia results, supporting broader clinical impact beyond growth.
Written By: Pharmacally Medical News Desk
BioMarin Pharmaceutical Inc. has reported new clinical findings for VOXZOGO (vosoritide), expanding its clinical profile beyond previously established growth benefits to include improvements in skeletal proportionality, bone health, and emerging evidence in hypochondroplasia.
Building on the 12 March 2026 update demonstrating durable height gains with early treatment in achondroplasia, the latest data extend these findings across broader age groups with longer-term follow-up.
Long-term extension analyses show that VOXZOGO drives consistent improvements in arm span across all age groups, with rising arm span Z-scores and a stable arm span-to-height ratio, reinforcing proportional skeletal growth.
Additional data in 119 children treated for up to six years show sustained increases in bone mineral content, while bone mineral density Z-scores remained stable, confirming preservation of bone health alongside continued growth.
BioMarin also reported new data in hypochondroplasia from the Phase 2 CANOPY-HCH-1 study, demonstrating statistically significant gains in bone mineral density and bone mineral content after 12 months of treatment.
Bradley Miller M.D., Ph.D., pediatric endocrinologist at the University of Minnesota Medical School emphasized that the growing long-term evidence for VOXZOGO shows sustained benefits beyond height, strengthening confidence in early treatment to improve overall health outcomes in children with achondroplasia.
Greg Friberg M.D., Executive Vice President and Chief Research & Development Officer at BioMarin highlighted that over a decade of research consistently demonstrates the importance of early and long-term therapy, with meaningful impacts on measures such as arm span and bone health for patients worldwide.
These findings provide early clinical validation that VOXZOGO’s mechanism may extend to other FGFR3-driven skeletal dysplasias. The company expects topline results from the Phase 3 CANOPY-HCH-3 trial in the first half of 2026 and plans regulatory submissions in the second half of 2026 if positive, positioning VOXZOGO for potential expansion into a broader patient population.
Reference