BioMarin (BMRN) Releases 10-Year Long-Term Growth and Bone Density Improvement Data for VOXZOGO
10-Year Long-Term Data Demonstrates Sustained Treatment Efficacy
BioMarin Pharmaceutical presented 10-year long-term clinical extension study data for VOXZOGO (vosoritide), a treatment for achondroplasia, at the 2026 Pediatric Endocrine Society (PES) Annual Meeting. This data demonstrates that long-term administration of VOXZOGO, a C-type natriuretic peptide (CNP) analog, consistently improves arm span Z-score and maintains balanced body proportions in pediatric patients. Specifically, in children who started treatment after the age of 5, a significant average height increase of 10.60 cm was achieved after 6 years of administration compared to the control group, and 13.59 cm after 8 years. This provides strong medical evidence that the treatment effect is stably maintained until adulthood, and is not limited to temporary growth.
Improved Bone Density and Real-World Impact in Clinical Practice
A notable aspect of this presentation is that long-term administration significantly improved bone mineral density (BMD) and bone health indicators in pediatric patients. Patients with achondroplasia are not only short in stature but are also constantly at risk of serious complications such as spinal stenosis and fractures due to skeletal deformities. VOXZOGO is the only targeted therapy approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for this indication, and the long-term bone density improvement data demonstrates that it also improves the structural health of the bones. In clinical practice, this data is expected to contribute to reducing the frequency of surgeries due to skeletal complications and improving the overall quality of life for pediatric patients.
Solidifying a Leading Market Position with Annual Sales Approaching $1 Billion
VOXZOGO has become a key growth driver for BioMarin, with annual sales of $927 million in 2025, representing a robust 26% increase year-over-year. The company expects VOXZOGO's annual sales to exceed $975 million to $1 billion in 2026. At this point, the released 10-year long-term data increases treatment continuity, prevents patient attrition, and provides a stable basis for long-term prescriptions. BioMarin is further enhancing market confidence by raising its overall annual revenue guidance from $3.825 billion to $3.925 billion through acquisitions such as Amicus Therapeutics.
Competition from Pipeline Drugs and Expansion of Indications
Currently, strong follow-up competitor drugs, including Ascendis Pharma's once-weekly CNP analog, navepegritide, and BridgeBio Pharma's oral FGFR3 inhibitor, infigratinib, are in Phase 3 clinical trials. In response, BioMarin is proactively expanding its market share by recently achieving the primary endpoint in a Phase 3 clinical trial to expand VOXZOGO's indication to hypochondroplasia. The long-term safety data released this time will serve as a difficult barrier for competitors to overcome in the short term, and will undoubtedly act as a strong defense mechanism against competitors' market entry.
BioMarin's VOXZOGO's 10-year long-term clinical data demonstrates safety and efficacy, solidifying its market position as a blockbuster drug poised to exceed $1 billion in sales in 2026. The data, which showed an additional growth of up to 13.59 cm compared to the control group, maximizes clinical credibility, leading to new patient acquisition and high prescription maintenance rates. This serves as a strong defense against follow-up competitor drugs in Phase 3 clinical trials, such as Ascendis Pharma's navepegritide and BridgeBio's infigratinib. In synergy with the recent successful Phase 3 clinical trial for hypochondroplasia, the long-term safety validation will accelerate the global expansion of the drug's prescription by easing the scope of reimbursement coverage by major national health insurance companies.
Source: PR Newswire Biotech (rss_filter)