BioMarin Says Phase 3 Study of Voxzogo Shows Patient Improvements

Dow Jones

May 21

By Christopher Kuo

BioMarin Pharmaceutical said its phase 3 study of a treatment for a genetic disorder that causes dwarfism showed statistically significant improvements among participants.

The trial evaluated BioMarin's Voxzogo drug among 80 children ages three to 17 with hypochondroplasia, a genetic skeletal disorder that causes short-limbed short stature.

The study met its primary endpoint, demonstrating a statistically significant increase in annualized growth velocity compared with the placebo, BioMarin said. Children who received the drug also showed a statistically significant increase in standing height versus the placebo after a year of treatment as well as improvements in arm span.

The company plans to submit its supplemental new drug application to the Food and Drug Administration in the third quarter, followed by submissions to the European Medicines Agency and other regional health authorities.

Write to Christopher Kuo at chris.kuo@wsj.com

(END) Dow Jones Newswires

May 20, 2026 16:57 ET (20:57 GMT)

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