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The landscape of pediatric endocrinology is shifting. At the Pediatric Endocrine Society’s (PES) 2026 Annual Meeting in San Francisco, new data presented by BioMarin Pharmaceutical Inc. has provided a clearer picture of how long-term treatment with VOXZOGO® (vosoritide) impacts children with achondroplasia.
While height is often the most visible metric in skeletal dysplasias, the latest research emphasizes that the benefits of VOXZOGO extend far beyond the measuring stick, reaching into the fundamental health of the skeletal system.
Proportional Growth and Arm Span
One of the most significant findings presented at PES involves skeletal proportionality. For children with achondroplasia, limb length often does not keep pace with trunk growth.
The data from three ongoing long-term extension trials revealed:
- Improved Arm Span: Z-scores for arm span improved across all age groups.
- Maintained Ratios: The arm span-to-height ratio remained stable, suggesting that the growth stimulated by VOXZOGO is proportional.
- Cumulative Height Gains: Children who began treatment after age five saw a mean standing height difference of 13.59 cm after eight years of treatment compared to untreated cohorts.
Strengthening the Foundation: Bone Health
A major concern for any long-term pediatric therapy is how it affects bone integrity. A study following 119 children for up to six years used dual X-ray absorptiometry (DXA) to monitor Bone Mineral Content (BMC) and Bone Mineral Density (BMD).
The results are encouraging for parents and clinicians alike. While BMC increased as the children grew, BMD Z-scores remained consistent year over year. This indicates that VOXZOGO supports the development of strong, healthy bones without compromising mineral density, confirming that the “new” bone being created is of high quality.
“With VOXZOGO, we now have a depth and duration of evidence that is unmatched… providing meaningful insight into the broader, sustained impact on a child’s health.”
— Bradley Miller, M.D., Ph.D., University of Minnesota Medical School.
The Next Frontier: Hypochondroplasia
BioMarin is also looking toward the future of VOXZOGO in treating hypochondroplasia, a related but distinct skeletal dysplasia.
A Phase 2 study conducted by Children’s National Hospital showed that children receiving the treatment for 12 months experienced statistically significant improvements in both bone density and mineral content.
What’s next?
- 1H 2026: Topline results from the pivotal Phase 3 CANOPY-HCH-3 trial are expected.
- 2H 2026: If results are positive, BioMarin intends to submit data to health authorities to seek approval for this new indication.
Why Early Intervention Matters
The recurring theme at this year’s PES meeting is the importance of early and sustained treatment. As the only approved treatment for infants starting at birth, VOXZOGO’s decade of clinical research suggests that intervening during the most critical windows of development leads to the best long-term health outcomes.
For the thousands of families navigating achondroplasia, these data points offer more than just statistics—they offer confidence in a treatment path that supports the whole child, from bone strength to physical proportion.