BioMarin Early VOXZOGO Trial Shows −0.53 ULBR Gain and 4.7 cm Height Increase
Children starting VOXZOGO under age two achieved a ULBR improvement of −0.33 at year one and −0.53 at year four versus untreated peers, with a 4.7 cm average height gain and 0.8 Z-score rise after four years. Real-world Japanese patients under age two saw a 9.91 cm height increase at 12 months.
1. Early Treatment Enhances Proportionality
Children who began vosoritide before age two experienced a least-squares mean reduction of 0.33 in upper-to-lower body segment ratio (ULBR) at one year and 0.53 by year four versus untreated peers, indicating improved body proportionality with sustained benefit.
2. Sustained Height Gains and Z-Scores
After four years of treatment, the under-two cohort achieved an average additional height gain of 4.7 cm and a 0.8 improvement in height Z-score compared to reference populations, demonstrating durable growth enhancements alongside stable arm-to-height ratios.
3. International Real-World Growth Data
Real-world evidence from Japan showed a 9.91 cm mean height increase at 12 months in patients starting treatment under two, while European data reported a 16.7 cm gain and 0.75 Z-score rise after 36 months, reinforcing clinical trial outcomes across geographies.