BioMarin Reports Positive Phase 3 Results For VOXZOGO

September 09, 2026 — 09:45 am EDT

Written by RTTNews.com for RTTNews

(RTTNews) - BioMarin Pharmaceutical Inc. (BMRN) on Wednesday announced positive Phase 3 results for VOXZOGO (vosoritide) in children with hypochondroplasia, with the study meeting its primary endpoint and all secondary efficacy endpoints.

After 52 weeks, VOXZOGO improved annualized growth velocity by 2.33 cm/year versus placebo. Significant improvements were also seen in standing height, height Z-score and arm span.

The treatment was generally well tolerated, with most adverse events mild and no treatment-related serious adverse events reported.

BioMarin recently submitted a supplemental New Drug Application to the FDA seeking approval for VOXZOGO in hypochondroplasia. If approved, it could become the first targeted therapy for the condition, with a potential launch in 2027.

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BioMarin Reports Positive Phase 3 Results For VOXZOGO

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