BioMarin's Voxzogo Drives Significant Long-Term Growth in Children with Achondroplasia
San Rafael, Saturday 2 May 2026
New 2026 clinical data reveals children treated with BioMarin’s Voxzogo achieved an impressive height increase of 13.59 cm over eight years, maintaining proportional skeletal growth and bone health.
Sustained Growth and Skeletal Proportionality
On 1 May 2026, BioMarin Pharmaceutical Inc. presented highly anticipated clinical data at the Pediatric Endocrine Society’s 2026 Annual Meeting in San Francisco [1]. The findings underscore the efficacy of Voxzogo (vosoritide), which remains the only approved therapeutic intervention for children with achondroplasia that can be administered from birth [1]. The longitudinal data revealed that children who commenced treatment after the age of five achieved a mean height difference of 10.60 cm after six years, a figure that expanded to 13.59 cm after eight years of continuous therapy, when compared to untreated cohorts [1]. This represents an incremental growth advantage of 2.99 cm between the sixth and eighth years of treatment [1].
Bone Density and Structural Integrity
A pivotal concern in accelerating bone growth through pharmacological intervention is the potential compromise of bone density [GPT]. However, a dedicated study tracking 119 children demonstrated that bone mineral content (BMC) steadily increased over time, whilst bone mineral density (BMD) Z-scores remained consistent throughout the long-term administration of the drug [1]. Voxzogo operates as a once-daily injection and functions as an analogue of C-type Natriuretic Peptide (CNP), targeting the underlying pathophysiology of the condition [2]. Dr Greg Friberg, BioMarin’s Executive Vice President and Chief Research & Development Officer, emphasised the company’s commitment to tracking health measures beyond mere height, highlighting the critical nature of these bone density metrics for the thousands of children receiving the therapy globally [1].
Expanding Horizons: Hypochondroplasia and Market Trajectory
Building upon its established efficacy in achondroplasia, BioMarin is aggressively expanding Voxzogo’s clinical applications to encompass hypochondroplasia, a related genetic skeletal dysplasia [1][GPT]. Recent Phase 2 clinical data highlighted a statistically significant improvement in total body (minus head) BMD of 0.03 g/cm² and a staggering BMC increase of 54.84 g after 12 months of treatment [1]. These figures further validate the drug’s mechanism of action in promoting healthy bone mineralisation alongside linear growth [1].