San Rafael, California, United States | May 2, 2026
BioMarin Pharmaceutical Inc. has presented new long-term clinical data for VOXZOGO® (vosoritide) demonstrating sustained improvements in growth, bone health, and skeletal proportionality in children with achondroplasia, a rare genetic condition affecting bone development. The findings, shared at the Pediatric Endocrine Society (PES) 2026 Annual Meeting, reinforce the therapy’s position as the only approved treatment targeting the underlying cause of achondroplasia from birth, supported by more than a decade of clinical research.
Long-Term Treatment Improves Growth and Bone Health
New data from ongoing long-term extension clinical trials highlight that VOXZOGO delivers consistent and meaningful improvements across multiple health parameters beyond height, including arm span, bone mineral density (BMD), and bone mineral content (BMC). Patients treated with VOXZOGO showed significant increases in arm span Z-scores across all age groups, while maintaining a stable arm span-to-height ratio, indicating proportional skeletal growth over time.
Children who initiated therapy after age five experienced substantial height gains, with a mean increase of 10.60 cm after six years and 13.59 cm after eight years of treatment, compared to untreated cohorts. In addition, bone health assessments using dual X-ray absorptiometry (DXA) revealed that bone mineral content increased steadily, while bone mineral density remained stable, demonstrating long-term maintenance of skeletal integrity. These findings underscore the importance of early and sustained treatment in improving clinical outcomes for children with achondroplasia.
Expanding Clinical Research into Hypochondroplasia
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BioMarin also presented new research evaluating VOXZOGO in hypochondroplasia, a related skeletal condition. Results from a Phase 2 clinical study demonstrated statistically significant improvements in bone density and bone content after 12 months of treatment, highlighting the therapy’s potential in broader indications.
The company is currently advancing a registration-enabling Phase 3 trial (CANOPY-HCH-3), with topline results expected in the first half of 2026. If successful, BioMarin plans to submit data to regulatory authorities to seek approval for this additional indication, potentially expanding the therapeutic reach of VOXZOGO. This progression reflects ongoing efforts to address unmet needs in rare skeletal disorders through targeted biologic therapies.
Mechanism of Action and Clinical Impact
VOXZOGO is a C-type natriuretic peptide (CNP) analog that acts by counteracting the overactive FGFR3 signaling pathway, which inhibits bone growth in achondroplasia. By promoting endochondral bone growth, the therapy directly targets the underlying genetic mechanism of the disease, offering a disease-modifying approach rather than symptomatic treatment.
Since its initial approval in 2021, VOXZOGO has been used to treat over 5,000 children across more than 50 countries, establishing a strong global clinical footprint. The therapy is administered as a daily subcutaneous injection, with dosing adjusted based on body weight, and is supported by extensive safety monitoring and patient support programs.
The latest clinical data presented by BioMarin reinforce the long-term efficacy and safety of VOXZOGO, demonstrating its ability to improve growth, maintain bone health, and enhance overall patient outcomes in achondroplasia. With ongoing research in additional indications such as hypochondroplasia, VOXZOGO continues to advance as a leading biologic therapy in rare genetic diseases, highlighting the critical role of innovation in GxP-regulated clinical development and rare disease treatment.
Source: BioMarin press release
