SAN RAFAEL, Calif., June 16, 2026
BioMarin Pharmaceutical Inc. announced new clinical findings from its approved therapy VOXZOGO® (vosoritide) and investigational candidate BMN 333 at ENDO 2026, the Endocrine Society Annual Meeting in Chicago. The data reinforce BioMarin’s leadership in treating rare skeletal growth disorders, highlighting sustained growth improvements in children with hypochondroplasia treated with VOXZOGO and encouraging early-stage results supporting weekly dosing of BMN 333 for achondroplasia. The presentations further strengthen the company’s expanding portfolio focused on genetically defined skeletal conditions and rare diseases.
Three-Year VOXZOGO Data Show Sustained Growth Improvements
New results from an investigator-sponsored Phase 2 extension study evaluated VOXZOGO treatment in 13 children with hypochondroplasia over three years. The study demonstrated sustained increases in both annualized growth velocity (AGV) and height standard deviation score (SDS), while maintaining a favorable safety profile. Mean AGV increased from 4.27 cm/year at baseline to 7.24 cm/year after one year of treatment, remaining above baseline levels through years two and three. Additionally, mean height SDS improved by 0.72 standard deviations during the study period. The findings build upon recently announced positive topline results from the pivotal CANOPY-HCH-3 Phase 3 trial, which BioMarin plans to include in a supplemental New Drug Application submission to the U.S. Food and Drug Administration during the third quarter of 2026. If approved, VOXZOGO could become the first therapy available for children living with hypochondroplasia, a rare genetic skeletal disorder currently lacking approved treatments.
BMN 333 Demonstrates Potential for Weekly Dosing
BioMarin also presented new Phase 1 clinical data for BMN 333, a long-acting C-type natriuretic peptide (CNP) designed to treat achondroplasia. In healthy adult volunteers, BMN 333 achieved prolonged systemic exposure and sustained pharmacodynamic activity, supporting the possibility of once-weekly dosing. Researchers reported that the highest evaluated dose generated more than 13 times greater free CNP exposure compared with another long-acting CNP therapy, highlighting its potential to deliver stronger and longer-lasting biological activity. Importantly, BMN 333 was well tolerated across all tested dose levels, with no dose-limiting toxicities or treatment-related serious adverse events observed. The promising results support continued advancement of the program as BioMarin seeks to improve treatment convenience and outcomes for patients with achondroplasia..
Advertisement
Late-Stage Development Plans Strengthen BioMarin’s Pipeline
Building on the encouraging early data, BioMarin recently initiated patient enrollment in a registration-enabling Phase 2/3 study of BMN 333, with dose-finding results expected in 2027. Meanwhile, VOXZOGO continues to expand its clinical evidence base beyond its currently approved indication for achondroplasia. Approved in more than 50 countries and used by over 5,000 children worldwide, VOXZOGO remains the only approved medicine specifically developed to support growth in children with achondroplasia from birth. With positive long-term hypochondroplasia data and the advancement of BMN 333 into late-stage development, BioMarin is further strengthening its position as a leader in rare skeletal disorder therapeutics while addressing significant unmet medical needs for patients and families affected by these lifelong genetic conditions.
Source: BioMarin press release
